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Gilead and Kite to Share Latest Scientific Advances in Hematologic Malignancies at ASH 2020 – Investing News Network

November 11th, 2020 11:54 am

16 Abstracts, Including Three Oral Presentations, Highlight Breadth of Companys Innovation in Immuno-Oncology for Patients with Blood Cancers

Kite Data Highlight Yescarta Long-Term Efficacy in Relapsed/Refractory Large B-Cell Lymphoma, its Potential as An Earlier Line of Therapy in DLBCL, as well as Results in Other Cancers, and One-Year Follow-up Results for Tecartus in Relapsed Mantle Cell Lymphoma

Magrolimab Demonstrates Continued Response Rates in Updated Results of Phase 1b Study of Acute Myeloid Leukemia Patients, Including Those with TP53 Mutation

Gilead Sciences, Inc. (Nasdaq: GILD) and Kite, a Gilead Company, today announced that 16 abstracts, including three oral presentations from the companies combined immuno-oncology research and development programs, have been accepted for presentation at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition. The meeting, which is being held virtually on December 5-8, 2020, will feature presentations on Yescarta (axicabtagene ciloleucel), Tecartus (brexucabtagene autoleucel, KTE-X19) and other ongoing research from Kites chimeric antigen receptor (CAR) T cell therapy development program, as well as magrolimab, Gileads first-in-class, investigational anti-CD47 monoclonal antibody.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201105005130/en/

The evidence supporting our innovation in hematologic malignancies continues to grow, providing assurance of the lasting and positive impact our diverse oncology pipeline could achieve over time, said Merdad Parsey, MD, PhD, Chief Medical Officer, Gilead Sciences. We continue to see broad potential across our oncology portfolio anchored by Kite in cell therapy and Gileads anti-CD47 monoclonal antibody to transform care for patients with hard-to-treat blood cancers.

New Long-Term Efficacy Data and the Potential of CAR T Therapy for More Patients

Building on three-year data presented at ASH 2019, overall survival results at four years from the pivotal ZUMA-1 trial of Yescarta in patients with refractory large B-cell lymphoma will be presented (Abstract #1187). Additionally, new data include one-year follow-up results from the ZUMA-2 study evaluating KTE-X19 in relapsed or refractory mantle cell lymphoma (Abstract #1120), as well as several studies evaluating the potential of Yescarta in new indications include an interim analysis of ZUMA-12 in first-line large B-cell lymphoma among patients with high-risk features (Abstract #405) and the ZUMA-5 primary analysis in relapsed or refractory indolent non-Hodgkin lymphoma (NHL), including follicular lymphoma (FL) and marginal zone lymphoma (MZL; Abstract #700).

Data from the ZUMA-5 primary analysis form the basis for the supplemental Biologics License Application (sBLA) for Yescarta currently under review by the U.S. Food & Drug Administration (FDA). Yescarta has previously been granted a Breakthrough Therapy Designation by the FDA for relapsed or refractory FL or MZL after at least two prior therapies and has been granted a Priority Review with a target action date, under the Prescription Drug User Fee Act (PDUFA), of March 5, 2021.

Our data at ASH build on the established strengths of our CAR T franchise, including practice-changing potential in new cancers, said Ken Takeshita, MD, Kites Global Head of Clinical Development. As we become the first company to present four-year CAR T data from a pivotal study in large B-cell lymphoma and continue to expand our leadership in cell therapy across different hematologic malignancies and into earlier lines of therapy, we remain committed to bringing the benefits of cell therapies to as many patients as possible.

Harnessing Potential First-in-Class Anti-CD47 Antibody in Difficult-to-Treat Malignancies

Researchers will give an oral presentation of updated results from the Phase 1b study of magrolimab in patients with previously-untreated acute myeloid leukemia (AML) who cannot undergo treatment with intensive chemotherapy, including patients with TP53 -mutant AML (Abstract #330). The FDA recently assigned Breakthrough Designation to magrolimab, in combination with azacitidine for the treatment of adult patients with newly-diagnosed MDS including intermediate-, high-, or very high-risk tumor types to expedite the development and regulatory review of this investigational treatment. Magrolimab also received PRIME Designation for treatment of MDS from the European Medicines Agency (EMA).

Dates and times for all accepted abstracts are as follows:

Area of Focus, Presentation Number and Date/Time

Abstract Title

Oral Presentations

Acute Myeloid Leukemia

Abstract #330

Sunday, Dec 6

(12:30pm ET / 9:30am PT)

The First-in-Class Anti-CD47 Antibody Magrolimab Combined with Azacitidine Is Well-Tolerated and Effective in AML Patients: Phase 1b Results

Large B-cell Lymphoma

Abstract #405

Sunday, Dec 6

(4:15pm ET / 1:15pm PT)

Interim Analysis of ZUMA-12: A Phase 2 Study of Axicabtagene Ciloleucel (Axi-Cel) as First-Line Therapy in Patients (Pts) with High-Risk Large B Cell Lymphoma (LBCL)

Non-Hodgkin Lymphoma Abstract #700

Monday, Dec 7

(4:30pm ET / 1:30pm PT)

Primary Analysis of ZUMA-5: A Phase 2 Study of Axicabtagene Ciloleucel (Axi-Cel) in Patients With Relapsed/Refractory (R/R) Indolent Non-Hodgkin Lymphoma (iNHL)

Poster Presentations

Follicular Lymphoma

Abstract #1145

Saturday, Dec 5

(10:00am ET / 7:00am PT)

Safety Profile of Idelalisib in Patients with Refractory Follicular Lymphoma: Interim Analysis of a Noninterventional Study

Large B-cell Lymphoma Abstract #1187

Saturday, Dec 5

(10:00am ET / 7:00am PT)

Long-Term Survival and Gradual Recovery of B Cells in Patients With Refractory Large B Cell Lymphoma Treated With Axicabtagene Ciloleucel (Axi-Cel)

Large B-cell Lymphoma Abstract #2100

Sunday, Dec 6

(10:00am ET / 7:00am PT)

Outcomes of Patients (Pts) in ZUMA-9, A Multicenter, Open-Label Study of Axicabtagene Ciloleucel (Axi-Cel) in Relapsed/Refractory Large B Cell Lymphoma (R/R LBCL) for Expanded Access and Commercial Out-of-Specification (OOS) Product

Large B-cell Lymphoma

Abstract #1224

Saturday, Dec 5

(10:00am ET / 7:00am PT)

The First Retrospective Commercial Claims-Based Analysis of CAR T Treated Patients With Relapsed or Refractory Large B-Cell Lymphoma (R/R LBCL)

Large B-cell Lymphoma

Abstract #2500

Sunday, Dec 6

(10:00am ET / 7:00am PT)

Cost and Healthcare Utilization in Relapsed/Refractory Diffuse Large B-Cell Lymphoma: A Real-World Analysis of Medicare Beneficiaries Receiving Chimeric Antigen Receptor T-Cell Vs. Autologous and Allogeneic Hematopoietic Stem Cell Transplants

Large B-cell Lymphoma

Abstract #2548

Sunday, Dec 6

(10:00am ET / 7:00am PT)

Burden of Illness and Outcomes in the 2nd Line Treatment of Large B-Cell Lymphoma: A Real-World Comparison of Medicare Beneficiaries with and without Stem Cell Transplants

Large B-cell Lymphoma

Abstract #1646

Saturday, Dec 5

(10:00am ET / 7:00am PT)

Lines of Therapy in Patients with Relapsed or Refractory Large B-Cell Lymphoma and Stem Cell Transplant-Intended Treatment

Mantle Cell Lymphoma

Abstract #1120

Saturday, Dec 5

(10:00am ET / 7:00am PT)

One-Year Follow-Up of ZUMA-2, the Multicenter, Registrational Study of KTE-X19 in Patients With Relapsed/Refractory Mantle Cell Lymphoma

Mantle Cell Lymphoma

Abstract #1126

Saturday, Dec 5

(10:00am ET / 7:00am PT)

Pharmacological Profile and Clinical Outcomes of KTE-X19 by Prior Bruton Tyrosine Kinase Inhibitors (BTKi) Exposure or Mantle Cell Lymphoma (MCL) Morphology in Patients With Relapsed/Refractory (R/R) MCL in the ZUMA-2 Trial

Non-Hodgkin Lymphoma

Abstract #2036

Sunday, Dec 6

(10:00am ET / 7:00am PT)

Retreatment With Axicabtagene Ciloleucel (Axi-Cel) in Patients With Relapsed/Refractory Indolent Non-Hodgkin Lymphoma in ZUMA-5

Trials-In-Progress

Acute Lymphoblastic Leukemia & Non-Hodgkin Lymphoma

Abstract #1896

Sunday, Dec 6

(10:00am ET / 7:00am PT)

ZUMA-4: A Phase 1/2 Multicenter Study of KTE-X19 in Pediatric and Adolescent Patients With Relapsed/Refractory B Cell Acute Lymphoblastic Leukemia or Non-Hodgkin Lymphoma

Large B-cell Lymphoma

Abstract #2103

Sunday, Dec 6

(10:00am ET / 7:00am PT)

ZUMA-19: A Phase 1/2 Multicenter Study of Lenzilumab Use with Axicabtagene Ciloleucel (Axi-Cel) in Patients (Pts) With Relapsed or Refractory Large B Cell Lymphoma (R/R LBCL)

Online Publication

Follicular Lymphoma

Efficacy Outcomes of Treatments for Double Relapsed/Refractory Follicular Lymphoma (R/R FL): A Systematic Literature Review

For more information, including a complete list of abstract titles at the meeting, please visit: https://ash.confex.com/ash/2020/webprogram/start.html .

Yescarta was the first CAR T cell therapy to be approved by the FDA for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, and high grade B-cell lymphoma and DLBCL arising from FL. Yescarta is not indicated for the treatment of patients with primary central nervous system lymphoma. In July, Tecartus became the first CAR T cell therapy to receive accelerated approval from the FDA for the treatment of relapsed or refractory mantle cell lymphoma, based on overall response rate and durability of response. Continued approval for this indication may be contingent upon additional data from a confirmatory trial. The U.S. Prescribing Information for Yescarta and Tecartus each have BOXED WARNINGS for the risks of CRS and neurologic toxicities, and Yescarta and Tecartus are each approved with a risk evaluation and mitigation strategy (REMS) due to these risks; see below for Important Safety Information.

The uses of Yescarta in relapsed or refractory FL or MZL or as a first-line treatment for patients with large B-cell lymphoma and high-risk genetics are investigational and not approved anywhere globally. Its efficacy and safety have not been established for these indications.

Magrolimab is investigational and not approved anywhere globally. Its efficacy and safety have not been established. More information about clinical trials with magrolimab is available at http://www.clinicaltrials.gov .

ABOUT MAGROLIMAB

Magrolimab is a first-in-class, investigational monoclonal antibody against CD47 and macrophage checkpoint inhibitor that is designed to interfere with recognition of CD47 by the SIRP receptor on macrophages, thus blocking the dont eat me signal used by cancer cells to avoid being ingested by macrophages. Magrolimab is being developed in several hematologic and solid tumor malignancies, including MDS. Magrolimab has been granted Fast Track Designation for the treatment of MDS, AML, DLBCL and FL. Magrolimab has also been granted Orphan Drug Designation by the FDA and EMA for treatment of MDS and AML.

About Yescarta

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Gilead and Kite to Share Latest Scientific Advances in Hematologic Malignancies at ASH 2020 - Investing News Network

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The great reset: new danger on the horizon – Amandala

November 11th, 2020 11:54 am

Belize City, Nov. 2, 2020 Most people in Belize are either taken up these days with finding a job/income, with fears of COVID-19, or with anticipation of the General Elections of Nov. 11, 2020. But lurking in the shadows is a much more dangerous foe.

In the past it was called The New World Order, but that has been so discredited, that the wizard behind the curtain had to change the name to The Great Reset. What is this Great Reset?

The Great Reset is a new social contract that ties you to it through an electronic ID linked to your bank account and health records, and a social credit ID that will dictate every facet of your life. While the COVID-19 pandemic is being used as a justification for the Great Reset movement, the agenda has nothing to do with health and everything to do with a long-term plan to monitor and control the world through digital surveillance and artificial intelligence.

The Great Reset and the Fourth Industrial Revolution are rebranded terms for the old New World Order, melded with the trans-humanist movement. Technocracy (which is the new name for Fascism) is an economic system of resource allocation that revolves around computer technology in particular artificial intelligence, digital surveillance, and Big Data (5G) collection and the digitization of industry and government, which in turn allows for the automation of social engineering and social rule, thereby doing away with the need for democratically elected leadership.

While the real plan is to usher in a tech-driven dystopia free of democratic controls, the elites speak of this plan as a way to bring us back into harmony with nature the Green New Deal. Importantly, the pandemic is being used to destroy local economies around the world, which will then allow the World Economic Forum to come in through the IMF and rescue debt-ridden countries through facilitated financial bailouts.

However, the price for this salvation is your personal freedom and liberty. And, again, one of the aspects of the Fascist plan is to eliminate national borders and nationalism in general.

Who are the main actors behind the Great Reset?

Bill Gates and the World Economic Forum, along with the United Nations (which keeps a relatively low profile), appear to be at the heart of the big boys agenda. Gates is also the largest money-bag for the World Health Organization the medical branch of the U.N. Other key partners that play important roles in the implementation of the elites/globalists agenda include foundations such as the Rockefeller Foundation, the Rockefeller Brothers Fund, the Ford Foundation, Bloomberg Philanthropies, the UN Foundation, and George Soros Open Society Foundation; companies such as: Avanti Communications, a British provider of satellite technology with global connectivity, and 2030 Vision, a partnership of technology giants that is aimed at providing the infrastructure and technology solutions needed to realize the U.N.s 2030 Sustainable Development Goals. 2030 Vision is also partnered with Frontier 2030, which is a partnership of organizations under the helm of the World Economic Forum.

These organizations include the major Wall Street bankers/financiers; Google, the No. 1 Big Data collector in the world and a leader in AI services; MasterCard, which is leading the globalist charge to develop digital IDs and banking services, and Salesforce, a global leader in cloud computing, the internet of things and artificial intelligence.

Incidentally, Salesforce is led by Marc Benioff, who is also on the World Economic Forums board of directors, and Professor Klaus Schwab, chairman of the World Economic Forum.

Most Belizeans know little or nothing about the trans-humanist movement, or Human 2.0, which is geared at transcending biology through computer technology. Or, as Dr. Carrie Madej of USA explains in a blog, their goal is to meld human biology with computer technology and artificial intelligence. Two visible proponents of trans-humanism are Ray Kurzweil (director of engineering at Google since 2012) and Elon Musk (founder of SpaceX, Tesla and Neuralink). According to Dr. Madej, today we may be standing at the literal crossroads of trans-humanism, thanks to the fast approaching release of one or more mRNA COVID-19 vaccines.

Many of the COVID 19 vaccines https://articles.mercola.com/sites/articles/archive/2020/05/22/coronavirus-vaccine-timetable.aspx are not conventional vaccines. Their design is aimed at manipulating your very biology, and therefore have the potential to alter the biology of the entire human race. Conventional vaccines train your body to recognize and respond to the proteins of a particular virus by injecting a small amount of the actual viral protein into your body, thereby triggering an immune response from your body and the development of antibodies.This is not what happens with an mRNA vaccine. The theory behind these vaccines is that when you inject the mRNA into your cells, it will stimulate your cells to manufacture their own viral protein. The mRNA COVID-19 vaccine will be the first of its kind. No mRNA vaccine has ever been licensed before. And, to add insult to injury, theyre forgoing all animal safety testing.

Madej has reviewed the background of certain individuals participating in the race for a COVID-19 vaccine, which include Moderna co-founder Derrick Rossi, a Harvard researcher who successfully reprogrammed stem cells using modified RNA, thus changing the function of the stem cells. Moderna was founded on this concept of being able to modify human biological function through genetic engineering.

The mRNA vaccines are designed to instruct your cells to make the SARS-CoV-2 spike protein, the glycoprotein that attaches to the ACE2 receptor of the cell. The idea is that by creating the SARS-CoV-2 spike protein, your immune system will mount a response to it and begin producing antibodies to the virus.

However, as we now know, Moderna is having problems, because both the CEO and CFO have, according to the Wall Street Journal, dumped their shares and sold everything, making some $350 million + dollars.

But the biggest insult by the globalists to our intelligence is the censorship of the news about the research done by genetic analysis using the Oak Ridge National Lab supercomputer called the Summit which has revealed an interesting new hypothesis that helps explain the disease progression of COVID-19. A September 1, 2020 Medium article1 by Thomas Smith reviewed the findings of what is now referred to as the Bradykinin hypothesis.

As reported by Smith, the computer crunched data on more than 40,000 genes obtained from 17,000 genetic samples.

Summit is the second-fastest computer in the world, but the process which involved analysing 2.5 billion genetic combinations still took more than a week. When Summit was done, researchers analysed the results. It was, in the words of Dr. Daniel Jacobson, lead researcher and chief scientist for computational systems biology at Oak Ridge, a eureka moment.

Bradykinin is a chemical that helps regulate your blood pressure and is controlled by your renin-angiotensin system (RAS). As explained in the Academic Press book on vitamin D (which has a significant impact on the RAS):

The renin-angiotensin system (RAS) is a central regulator of renal and cardiovascular functions. Over-activation of the RAS leads to renal and cardiovascular disorders, such as hypertension and chronic kidney disease, the major risk factors for stroke, myocardial infarction, congestive heart failure, progressive atherosclerosis, and renal failure.

The Bradykinin hypothesis provides a model that helps explain some of the more unusual symptoms of COVID-19, including its bizarre effects on the cardiovascular system. It also strengthens the hypothesis that vitamin D plays a really important role in the disease.

Your ACE2 receptors are the primary gateways of the virus, as the virus spike protein binds to the ACE2 receptor. As explained2 by Smith:

COVID-19 infection generally begins when the virus enters the body through ACE2 receptors in the nose The virus then proceeds through the body, entering cells in other places where ACE2 is also present But once Covid-19 has established itself in the body, things start to get really interesting The data Summit analysed shows that COVID-19 isnt content to simply infect cells that already express lots of ACE2 receptors. Instead, it actively hijacks the bodys own systems, tricking it into up-regulating ACE2 receptors in places where theyre usually expressed at low or medium levels, including the lungs.

In this sense, COVID-19 is like a burglar who slips in your unlocked second-floor window and starts to ransack your house. Once inside, though, they dont just take your stuff they also throw open all your doors and windows so their accomplices can rush in and help pillage more efficiently.

The end result is a Bradykinin storm, and according to the researchers, this appears to be an important factor in many of COVID-19s lethal effects, even more so than the Cytokine storms associated with the disease. As Bradykinin accumulates, the more serious COVID-19 symptoms appear. Mounting clinical data suggest COVID-19 is actually primarily a vascular disease rather than a respiratory one, and runaway Bradykinin build-up help explain this.

The good news is that since Bradykinin storms are to blame, there are a number of already existing drugs (Icatibant, Danazol, Stanozolol) that can help prevent Bradykinin storms, and there are many other safe, inexpensive strategies like nebulized peroxide, ozone, molecular hydrogen, steroids, exogenous ketones, and Quercetin with zinc, vitamin D, and high-dose vitamin C.

And there are two reports by the American CDC. One says that 70.6% of COVID-19 patients always wore a mask3. The other says only 6% of all COVID-19 deaths were due ONLY to coronavirus4. And yet another said that the common seasonal flu caused more deaths than COVID-19.

So, if COVID-19 deaths are not what is being reported by the mass media, if the SAR CoV-2 virus is not as deadly to humans, then why the lockdowns, the face masks, the social distancing, the destruction of the way we live, of our economies? Why? Why?

But not all men are blind. On Oct 25, 2020, the Archbishop of Ulpiana, former Apostolic Nuncio to the United States of America, Carlo Maria Vigano, wrote an open letter (which over 100 million Americans have read) to President Donald Trump. The letter is long and is all over the internet. This is some of it:

at this hour in which the fate of the whole world is being threatened by a global conspiracy against God and humanityin the midst of the silence of both civil and religious authoritiesthis historical moment sees the forces of Evil aligned in a battle against the children of Lightwe see heads of nations and religious leaders pandering to this suicide of Western culture and its Christian soul, while the fundamental rights of citizens and believers are denied in the name of a health emergency that is revealing itself more and more fully as instrumental to the establishment of an inhuman faceless tyranny.

A global plan called the Great Reset is underway. Its architect is a global lite that wants to subdue all of humanity, imposing coercive measures with which to drastically limit individual freedoms and those of entire populations Behind the world leaders who are the accomplices and executors of this infernal project, there are unscrupulous characters who finance the World Economic Forum and Event 201, promoting their agenda.

The purpose of the Great Reset is the imposition of a health dictatorship aiming at the imposition of liberticidal measures, hidden behind tempting promises of ensuring a universal income and cancelling individual debt. The price of these concessions from the International Monetary Fund will be the renunciation of private property and adherence to a program of vaccination against Covid-19 and Covid-21 promoted by Bill Gates with the collaboration of the main pharmaceutical groups. Beyond the enormous economic interests that motivate the promoters of the Great Reset, the imposition of the vaccination will be accompanied by the requirement of a health passport and a digital ID, with the consequent contact tracing of the population of the entire world. Those who do not accept these measures will be confined in detention camps or placed under house arrest, and all their assets will be confiscated.

Mr President, I imagine that you are already aware that in some countries the Great Reset will be activated between the end of this year and the first trimester of 2021. For this purpose, further lockdowns are planned, which will be officially justified by a supposed second and third wave of the pandemic. But this world, Mr. President, includes people, affections, institutions, faith, culture, traditions, and ideals: people and values that do not act like automatons, who do not obey like machines, because they are endowed with a soul and a heart, because they are tied together by a spiritual bond that draws its strength from above, from that God that our adversaries want to challenge, just as Lucifer did at the beginning of time with his non serviam.

Until a few months ago, it was easy to smear as conspiracy theorists those who denounced these terrible plans, which we now see being carried out down to the smallest detail. No one, up until last February, would ever have thought that, in all of our cities, citizens would be arrested simply for wanting to walk down the street, to breathe, to want to keep their business open, to want to go to church on Sunday. Yet, now it is happening all over the world.

Mr. President, you have clearly stated that you want to defend the nation One Nation under God, fundamental liberties, and non-negotiable values that are denied and fought against today. It is you, dear President, who are the one who opposes the deep state, the final assault of the children of darkness.

For this reason, it is necessary that all people of goodwill be persuaded of the epochal importance of the imminent election Your adversary is also our adversary: it is the Enemy of the human race, He who is a murderer from the beginning (Jn 8:44).

And yet, in the midst of this bleak picture, this apparently unstoppable advance of the Invisible Enemy, an element of hope emerges. The adversary does not know how to love, and it does not understand that it is not enough to assure a universal income or to cancel mortgages in order to subjugate the masses and convince them to be branded like cattle. This people, which for too long has endured the abuses of a hateful and tyrannical power, is rediscovering that it has a soul; it is understanding that it is not willing to exchange its freedom for the homogenization and cancellation of its identity; it is beginning to understand the value of familial and social ties, of the bonds of faith and culture that unite honest people.

This Great Reset is destined to fail because those who planned it do not understand that there are still people ready to take to the streets to defend their rights, to protect their loved ones, to give a future to their children and grandchildren. The levelling inhumanity of the globalist project will shatter miserably in the face of the firm and courageous. To be an instrument of Divine Providence is a great responsibility, for which you will certainly receive all the graces of state that you need, since they are being fervently implored for you by the many people who support you with their prayers.

Meanwhile, here in Belize, we kill our so-called COVID-19 patients. Ventilators will kill you. Doctors of Belize, read the report of the US Oak Ridge National Lab on COVID-19. NO one needs to die anymore from COVID-19. US President Trump, who is 74 years old, was cured after 3 days of COVID-19.

And by the time you read this article, the world will know who won the elections in the United States.

Curfew on Nov. 11, election night in Belize, is part of the Globalist agenda. Let the people celebrate their victory. Open the churches, the schools, the bars; open the society. Send the globalist/elites back to Hell with Lucifer.

(Footnotes)1https://elemental.medium.com/a-supercomputer-analyzed-covid-19-and-an-interesting-new-theory-has-emerged-31cb8eba9d63

2ibid3https://www.cdc.gov/mmwr/volumes/69/wr/mm6936a5.htmRead the table at the end.4https://www.cdc.gov/nchs/nvss/vsrr/covid_weekly/index.html

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The great reset: new danger on the horizon - Amandala

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Teladoc Is A Strong Buy: A Radical Healthcare Change Will Come – Seeking Alpha

November 11th, 2020 11:54 am

Introduction

Last week, the Teladoc (TDOC) and Livongo (LVGO) merger was completed. That means that Livongo doesn't trade anymore. If you still had your Livongo shares, you got (or will get if your broker is a bit slower) 0.5920 per share of Livongo in Teladoc shares plus cash of $11.33, paid with a special dividend of $7.09 on October 29th, and the rest when your shares were changed to Teladoc shares.

(Source)

As a former shareholder of Livongo, you may not be completely familiar with how Teladoc is positioned now. If there is a buy-out or a merger, that always generates mixed feelings, or at least it should. If it doesn't, it means that you had a bad stock in your portfolio.

With Potential Multibaggers, my marketplace service here on Seeking Alpha, I try to find multibaggers early on. I picked Livongo as a Potential Multibagger on December 26, 2019. The stock then traded at $24.86 and had a market cap of just $2.5B. The stock returned 462.2%, so it's more than a fivebagger in less than a year. But still, quite a few shareholders, from both companies, didn't feel great about the merger.

I think Teladoc could still be a multibagger at this point for patient investors. I think most investors underestimate that this combined company could represent the future of our healthcare system. In this article, written from a bird's eye perspective, I will try to explain why.

I think a lot of people know that the American healthcare system (and that of most Western countries) is unsustainable. It's too expensive but nobody seems to find a way out to cut costs.

The problem is that our healthcare is one of the last sectors that has not been disrupted by tech yet. The system originates from a time when bigger was better because it was more affordable to have standard procedures. Long ago, there was a family doctor and he knew you and you knew him and you had a personal relationship with that man (female doctors almost didn't exist back then).

After the Second World War, two evolutions emerged that made this system unsustainable: people reached a higher and higher age and the Baby Boomers were born. That put pressure on both ends of the healthcare system and the solutions were more scale and introducing standard procedures, so the productivity of healthcare workers became higher. Specialized care also contributed to more efficiency. And it worked.

But there was a side effect. People don't feel connected to the people that should care for them. They often feel treated like numbers, like patients at best, but mostly not like individual people. I'm not throwing a stone here at doctors, nurses and other healthcare workers. They often share that feeling. They don't have the time to deeply care about people. Their time is limited, they have to reach the quota. A doctor is not paid for listening to you. He's paid per patient that he handles. In other words, the less he or she listens, the more the doctor is incentivized. And that wears out a lot of healthcare workers.

Normally, such a market would correct itself. If you are not properly served in Lowe's (LOW), you go to Home Depot (HD) or the other way around. If one is really not giving enough attention to its clients, the company will go bankrupt eventually. That's where the efficiency of the market plays its role.

But in healthcare, the patients are not the customers. They are the goods, as it were. Customers are the paying party. And who pays for healthcare costs? Exactly, the insurance companies. Their only objective is as little costs as possible and that's why they pay per visit, for example.

But this creates the strange effect that a doctor that treats you very well is only paid once, while a bad doctor, who follows the wrong procedure and has to repair the damage or gives the wrong diagnosis, is rewarded each time he or she treats you and so earns more money than the good doctor.

To fix the healthcare system, a little reparation here and there won't help. We saw in the last decades that tech has entered almost every industry and has disrupted industries completely. Think of how Amazon (AMZN) made Sears obsolete. The same thing should (and probably will) happen in healthcare.

Disruption comes from the Latin verb 'disrumpere', which means to break apart and that is what healthcare needs: breaking things apart to build them up again. The bottom line of every healthcare reform should be to use tech and turn the system upside down. Health should be rewarded and paid for, not sickness.

All insurance is meant for emergency cases, except for healthcare insurance. Doctors are incentivized to do as many consults and tests as possible because someone makes money on that: the doctor himself or the hospital, mostly both. A hospital, for example, makes 10 or 20 times more money if you go to the emergency room than if you use an online platform to talk to a telehealth doctor.

As a patient, if you see a doctor, most of that little time you spend with him or her is dedicated to tests, collecting data. But suppose the doctor already would have all the data when you come in and he or she has already been able to look into your case, your history, and tens of up-to-date data points before you came in, that doctor could have time for that which we all crave when we visit a doctor: talking about what you exactly have and what it means, what we can do to get better, a discussion about what the underlying cause could be, talking about the psychological effects that your condition brings with it, what the best plan of action would be for you, etc. In other words, the doctor could become some kind of health coach, a professional that, with the help of precise data, could prescribe a trajectory to better health, hold you accountable, help you when sticking to the plan is tough etc. A doctor could partly become a real healthcare worker, not just a sickcare worker.

That would mean that you wouldn't have to visit a doctor as often. If you have a chronic condition, you could be monitored 24/7 by sensors, assisted by AI, as Livongo does for diabetes. You are not only monitored but you also get health nudges. That means that you would know exactly what is the right path for you. And that path is much more individualized than most people can imagine.

Hemant Taneja, a venture capitalist of General Catalyst who founded Livongo with Glen Tullman, calls this new space in healthcare 'health assurance'.

(Hemant Taneja, right, together with Livongo founder Glen Tullman, source)

Taneja is convinced that health assurance industry will see several $100B+ companies. I suspect that he was the great driving force behind the Livongo/Teladoc merger, together with Glen Tullman. They realized that Livongo can become so much more combined with Teladoc than on its own.

This is how health assurance is defined:

Health assurance is an emerging category of consumer-centric, data-driven healthcare services that are designed to bend the cost curve of care and help us stay well. Built on the principles of open technology standards, these services employ empathetic user design and responsible AI. This is the future of your health experience.

If you want to know more about this, you could read Taneja's interesting book Unhealthcare.

(Source)

Now that you know the basics of the concept of health assurance, I think you can see the potential for Teladoc and Livongo in this market. There is no company that is as well-positioned as the combination of Teladoc and Livongo. Livongo brings measuring, data collection and especially AI to the table, Teladoc has a worldwide network of telehealth.

Investors who just see what there is now could get scared away from Teladoc. They look at the number of telehealth visits that could slow down after the peak of the coronavirus and they are afraid that Teladoc will just have been a COVID-19-induced fad. But if you look at the future, you see that this company could be in the sweet spot when healthcare will be disrupted over the next decade.

Insurance companies will help them with this. As we have already seen, they just want to pay as little as possible. Livongo saved insurance companies $88 per patient per month. The reason: the monitoring of diabetes patients, the fact that they have a diabetes coach that is always available and the health nudges reduce the medical costs dramatically. $88 per patient per month means more than $1,000 per year per patient. I think you see the potential.

Besides that, the patients also feel freer than before. Their diabetes doesn't control their lives as much. They need less medication and if they need it, the data will indicate it before the attack.

This is just for diabetes. That's already a big market. But Livongo doesn't just focus on diabetes but also on hypertension and obesity. Those are two huge markets as well. The hypertension market is estimated to be $23B in 2026 and the obesity treatment market is estimated to be around $20B in 2026. But suppose you add the weight loss market to this, which is worth about $70B in the US alone, and you can see the potential.

The obesity-related healthcare costs are estimated at around $147B annually, so this might mean big savings for healthcare spending.

There will certainly be other companies in this space than just Livongo and Teladoc, companies that will also focus on other domains, but so far, I don't see any competitor that is as advanced as Livongo/Teladoc is.

When I pick a Potential Multibagger, I turn a company inside out and that means that I know Livongo, its founders Glen Tullman and Hemant Taneja and what the company exactly does really well.

I didn't know Teladoc as well before the merger was announced. I had it on my watchlist, but I had not done a really deep dive. When I did, I found a lot that I liked. This statement by Teladoc's CEO Jason Gorevic on the closing of the merger is worth reading word for word if you want to understand the combination of the companies:

Both Teladoc Health and Livongo were founded with the same mission: to create a new kind of healthcare experience, one that empowers people everywhere to live their healthiest life. Today's news (the merger, FGTV) dramatically accelerates our ability to make this a reality for the tens of millions of consumers and healthcare professionals we serve around the world. Together, our team will achieve the full promise of whole-person virtual care, leveraging our combined applied analytics, expert guidance and connected technology to deliver, enable and empower better health outcomes."

There are a few critical phrases here. Let's look at them one by one.

"a new kind of healthcare experience" This is the disruption that I mentioned. Not just fine-tuning the current system, but a completely fresh start.

"one that empowers people everywhere to live their healthiest life".

This is the health assurance that I was talking about. Helping people to live their healthiest life is the reverse of what the current healthcare system thrives on. Empowerment means that people will be able to decide for themselves and take their health into their own hands. The 'everywhere' in this phrase refers to the global footprint Teladoc has.

"whole-person virtual care"

This refers to everything from health assurance to data, treatment plans, health nudges, and specialized diagnosis and surgery. Teladoc/Livongo will be the only one-stop-shop for taking your healthcare into your own hands. Healthcare is one of the few paternalistic sectors left. Paternalistic here means: "We know everything, you don't. Your only function is to give us the money and shut up."

Again, that's not throwing a stone to healthcare workers. I have a really, really deep respect for the people working in healthcare and this pandemic has highlighted even more how crucial they are. Several of my friends work in the sector.

They actually often feel the same frustration as patients. They don't have enough time to really listen, which would help them to diagnose more accurately, they have to do too much administration, they lose precious time gathering simple data and there are no efficient follow-up programs. Most healthcare workers would love to have the time to establish a human connection with each of their patients and listen to every detail that could count. But there is just no time. That's why a lot of people seek help from all kinds of coaches.

This is Teladoc's representation of whole-person care:

(Source)

Let's look into this in a bit more detail.

As you see from the graph, and I think this is really important, the category Wellness and Prevention is included in that care too. It is explained as 'Complete regular screenings and improve nutrition, exercise and well-being'. That means planning your health.

No company flies blindly and every company has a clearly-defined goal for the future often with step-by-step roadmaps. But for health, the advice is often: "Eat well and exercise." That's like saying to a company: "Execute well and make money." In other words, although it's true, it's too general.

A personal note here to illustrate what I mean. For years, I was very tired and I had trouble staying awake after meals or after drinking coffee. The advice I got from my doctors? Eat healthily, do regular exercise.

I found out 5 years ago that I have a milk allergy but I had to find out all about what it meant for my life (dairy-free cooking, avoiding almost all cookies, but also potato chips etc.) on my own. It would have been great to have a specialist that could have coached me there.

The example of my own life is just to illustrate the truth about healthcare that we all intuitively know but that is not acknowledged enough under this system: we are all individuals, with our differences, our unique needs. In stock terms, we are as different as a steel producer stock and a SaaS stock. I'm a man of 6 feet and 5 inches and almost always, I get the same dose of medicine as a woman of barely 5 feet high.

We are on the brink of other breakthroughs in healthcare: stem cell therapy, gene therapy, cheap genome sequencing, CRISPR, and many more. All these trends point in one direction: individualized healthcare. Medicine will not be a mass-produced, mass-prescribed drug anymore. We will evolve to personalized medicine.

Initially, people will be split into different groups based on certain data points (age, weight, condition...) and later it will be really about you, the individual. Your genome, your microbiome, your allergies, your reactions to certain drugs, everything will be known and taken into account for your prescription. Lots of medicines contain milk, for example, as a filling agent. Each time again, I have to say this to a doctor and sometimes there are even no medicines on the market without milk. These will be produced in the individualized healthcare that will come, if only for me. Medicines will be prepared on who you are, not on who the masses are.

But it will be much more than just medicines. Which supplements should you take? What is the perfect exercise regimen for your type of body? How could you build up your condition for that marathon or triathlon you always dreamed of without the risk of an injury because of your specific body composition? What are the best shoes for your feet so they can be 3D-printed? What is the best diet for the specific microbes that you have in your gut? What are the diseases you are genetically susceptible to and what can you change in your lifestyle to prevent them?

Don't get me wrong, I'm not a pie-in-the sky thinker. This will not be for the first years, of course not. And at first, it could be unevenly distributed, as a lot has been throughout healthcare's history. The first individualized programs could come with a hefty price ticket. But probably that will democratize, as a lot of procedures have in healthcare. Or maybe it will democratize from the start but with different degrees of quality, a bit like smartwatches.

The medical know-how of Teladoc, its wide network of doctors and health specialists, combined with Livongo's AI, data gathering and processing capabilities, make that this company is, like no other that I know, prepared for the future of healthcare.

I haven't seen any other AI healthcare platform that even comes close to that of Livongo at this point. With its AI+AI approach (aggregate, interpret + apply, iterate) it has already learned a lot, both from the whole pool of patients as from individual patients. With more patients because of the merger, there will be more data and more data means more insights and new products over time.

And Livongo will double down on its AI and data analysis. Revealera.com is a website that looks at jobs, job openings and it tries to find relevant information from these data.

It showed that of all publicly-traded companies, Livongo had the highest percentage of job openings that require data science and machine learning. 16% of Livongo's jobs openings ask for experience in those fields.

This clearly shows to me that Teladoc/Livongo is skating where the puck will be, not just where it is.

If you look at the combination of the two companies that merged, you can see that they are very complementary. These are Teladoc's key growth strategies and in blue, Livongo could help to accelerate Teladoc's strategy:

(Source)

The companies estimate that there will be $500M in synergies.

(Source)

Now, I know that it's all too common in an acquisition or a merger to overestimate these synergies by a wide margin. But in this case, I think the synergy opportunities are actually very conservative. The companies even acknowledge that in their merger presentation:

There will be a lot of cross-selling, as there is only a 25% overlap in the customers of the companies. Even before the merger officially was closed, Livongo was cross-sold in two deals already by Teladoc.

The first deal was Fresenius Medical Care, a company specialized in working with patients that suffer from CKD (chronic kidney disease). Partnerships and distribution are quintessential in healthcare and in its field, Fresenius is a big player. It provides dialysis for 347,000 kidney patients. The press release of Fresenius explains:

This marks the first time Livongo will use its robust virtual care solutions to specifically support those with CKD and is a significant step forward in Fresenius Medical Care's efforts to provide a more coordinated care experience. With earlier intervention, Fresenius Health Partners also seeks to increase optimal dialysis starts, as well as offer earlier evaluation of transplantation and home dialysis options.

This shows that the possibilities for Livongo to branch out are numerous.

The second deal was with Florida Blue, part of GuideWell Mutual Holding. Together with its merging partner Teladoc, Livongo will offer Florida Blue members with diabetes virtual care, including connected devices, advanced data science, and telehealth.

Being the only one-stop-shop for digital healthcare will provide Teladoc/Livongo with a competitive advantage that others simply don't have at the moment. The companies shared an example about Claire, an imaginary future client. You can see the different stages and situations in which she can be helped by Teladoc after the merger with Livongo:

As you can see, there is not a single moment in the whole process that Claire has to leave the platform. In this way, Teladoc and Livongo show that they are very complimentary. And the data component of Livongo, combined with preventive healthcare, gives Teladoc a lot of flexibility to introduce even more products, each one more and more targeted and eventually personalized.

There are risks to every investment, of course, although I generally believe that too many investors overemphasize risk. What is risk? Risk is not the same as volatility, no matter what some want you to believe. Volatility is risky if you are a short-term investor. If you need the money in 2 years, volatility is a risk. But if you need the money in, let's say, 20 years, why would it matter if a certain stock is up or down 50% this year or the next?

If you read the great book 100 baggers by Chris Mayer, you'll see that ALL (!) of the 100 baggers (stocks that turn your $10K into $1M) saw drops of 50% and more at least once. Most several times, and often they dropped substantially more than 50%.

Risk is the chance that you will lose your money permanently, not volatility. That also means that you should look at risks in their context. All companies make mistakes and if you sell because of a mistake, you'll never find multibaggers. Do I need to remind you of the Amazon Fire phone, the Netflix Quikster failure, the Windows phone, Google Plus and so many more mistakes? Don't let one failed product mislead you. The company as a whole is much more important.

Having said that, what will I keep my eyes on for Teladoc?

First, I want to see that Livongo really has an impact on Teladoc because health assurance is more a part of what Livongo does right now. There is a risk that Teladoc doesn't leverage Livongo's capacities enough.

The second element that I will watch is how the two companies work together when it comes to company culture. Teladoc has a good tracking record when it comes to acquisitions and giving them a place where they feel good inside of the company but this merger with Livongo is on a whole different level. I see some good signs because Teladoc CEO Jason Gorevic and Livongo's founder and executive chairman have already come out together several times and the two seem to share the same vision.

The third and final element of risk that I want to touch on is competition. At this moment, I don't see any competitor that is even close to Teladoc after the merger with Livongo but that can always change fast, of course. On the other hand, this market is so big that there will be several winners. And the size of the market, that's the next topic of this article.

The market cap of the combined company is around $30B at this moment. For Potential Multibagger stocks, I want to see the possibility that the stock could be a tenbagger in the next 10 years. For Teladoc, I think this is still possible, despite its already substantial market cap. The company has everything it needs to start a new era in healthcare as I showed, and it's in a gigantic market. This is the title of recent research:

Global Digital Health Market was Valued at USD 111.4 billion in 2019 and is Expected to Reach USD 510.4 billion by 2025, Observing a CAGR of 29.0% during 2020-2025

Teladoc operates in a TAM (total addressable market) of $510B in 2025 and at this moment it is the only 360 digital health company. That's a great position to be in. For those who wouldn't know: TAM is the yearly total addressable market. The fact that Teladoc projects a CAGR (compound annual growth rate) of 30% to 40% seems conservative to me. If you would add the synergies, it will be at least to the higher end of that margin, in my opinion.

If Teladoc could just bring in 3% of that TAM of 2025, that would already mean $15.3B. If you slap a P/S ratio of 20 on that, you already have a company with a market cap of more than $300B. A P/S of 20 might seem aggressive but for a company growing at more than 30% per year and gross margins which will probably be in the mid-70s, I think it's very reasonable. You can tinker with the numbers but the conclusion to me is always that if Teladoc executes well, it has the potential to become a giant.

I'm not saying that the company will already have 3% of the global digital health market by 2025, mind you. I think revenue of 1% of the TAM, about $5.1B, is possible at that moment, though, and much more growth will be in the pipeline.

There are always a lot of ifs but when I look at Teladoc, I can see the potential to become really big, ten times or more bigger than today.

With a lot of healthcare disruption knocking at the door, such as cheap genome sequencing, CRISPR, personalized medicines and much more, data will become more and more important for healthcare. Livongo's AI will add that to Teladoc.

The combination of Teladoc and Livongo definitely has the first-mover advantage in a very important and big emerging market because it can provide a 360 degrees one-stop-shop for personalized digital healthcare.

If you have enjoyed this article, feel free to hit the "Follow" button next to my name.

In the meantime, keep growing!

Potential Multibaggers focuses on finding multibaggers early on.

Potential Multibaggers is not for those who trade in and out of stocks but for long-term investors who want life-changing returns.

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How genetics can help predict risks of cancer recurrence and improve treatment – Euronews

November 11th, 2020 11:53 am

A biobank is a storage facility for biological samples including blood, human tissue and/or DNA. They can then be used at any time for future medical research or pioneering methods.

The Managing Director of Estonian Biobank, Andres Metspalu, gives us some insight:

"I started the Estonian Biobank about 20 years ago. Our biobank is pretty large for a small country. We have around 20% of the entire Estonian population over the age of 18 included in our biobank; which equates to more than 200,000 individuals.

"They have all been analysed genetically, which is really remarkable. That is why we can do this genetic medicine not only for cancer, but also for other diseases.

"More than 3,000 people have already received their genetic risk (result) from the biobank.

"This is what keeps me busy every day, doing research and also facilitating the use of this information in healthcare".

"We are mainly talking about (predicting the risks of developing diseases like) cancer, cardiovascular diseases and type-2 diabetes. We also study melanoma, prostate cancer and lung cancer.

"We are also doing pharmacogenomics, drug response (how our bodies respond to drug intake).

"Not all drugs work on everyone as (pharmaceutical) companies believe or expect. Some drugs (can be) pretty harmful. You (can) get reactions and you (can) get side effects. You may end up in hospital after taking prescription drugs.

"Genetics can predict some serious events. It (genetics) should be used. This is what we are trying to introduce into everyday medical practice in Estonia".

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Biopharma Money on the Move: November 4-10 – BioSpace

November 11th, 2020 11:53 am

Here's a look at which companies are raking in cash this week in the biopharma industry.

Apollomics, Inc.

Committed tocombatting cancer with precision,Apollomics plans to use the$124.2 million Series Cfinancing to focusclinicalefforts on its lead programs: APL-101 and APL-106. APL-101 is an oral c-MET inhibitorcurrently involved in several ongoing clinical trials. TheSPARTA trialis in Phase II, targeting non-small cell lung cancer,glioblastomamultiforme and solid tumors with MET amplifications.APL-106 is a first-in-class targeted inhibitordesignedtoblockE-selectin, an adhesion molecule on cells in bone marrow,from binding with blood cancer cells.It has received Breakthrough Therapy Designation from the FDA in relapsed and refractory acute myeloid leukemia.In 2019,Apollomicsraised$100 million in a Series B round.

Decibel Therapeutics

Decibel Therapeutics made some noise this week with an oversubscribed Series D financing, raking in$82.2 million. The company will use the funds to advance DM-OTO, a gene therapy to restore hearing in children with congenital deafness due to a deficiency in the otoferlin gene.Clinical testing is expected to initiate in2022.DB-020 is already in a phase Ib study with cancer patients as apreventative treatment for the ototoxicityassociated with cisplatin-based chemotherapy.Hearing and balance disorders have historically been overlooked by the biopharma industry, even though they exact a devastating toll on the lives of hundreds of millions of people around the globe. At Decibel, we are dedicated to restoring hearing and balance with precision therapeutics designed to deliver the right genetic medicine specifically to the right cells in the ear, said Laurence Reid, Ph.D., Chief Executive Officer of Decibel.

Adagio Therapeutics

After launchingin Junewith $50 million, Adagio has pocketed another$80 million in a Series Bfinancing round to take its COVID-19 antibody into the clinic next year. Adagios neutralizing monoclonal antibodies are expected to provide broad protection against not onlyagainst SARS-CoV-2 and SARS-CoV-1, but also additional bat coronaviruses that have yet to cross the species barrier. Adagio got the green light from the FDA to proceed with their first-in-human study in early 2021.We were impressed by the thoughtful approach that Adagio took. By dealing with the broader coronavirus problem, we expect ADG20 to be more resistant to escape mutations and potentially cover future coronavirus pandemics, said Krishna Yeshwant, Managing Partner at GV. As a preventative agent, ADG20 holds the promise of providing the efficacy necessary to deliver greater protection against COVID-19. Given its unique combination of attributes, ADG20 could complement and supplement vaccines by providing rapid, durable antibody protection against current and future coronaviruses.

InmageneBiopharmaceuticals

Drug development companyInmagenehas its eye on being number one in immunology in China.The$21 million Series Bclosed this week will be pumped into conducting global clinical trials, research and development, and product in-licensing activities.Currently candidate IMG-020 is in a Phase II psoriasis trial, with a strong safety profile and clear clinical benefits giving it best-in-class potential. Manufactured inan E. coli system, IMG-020 is less than 1/20thof the average manufacturing cost of a typical antibody drug.The candidate is about to enter global registration trials formultiple indications.

Locus Biosciences

Its been a busy season for this CRISPR-engineer.In September,Locussigneda$144 million contract with BARDAto develop theirproduct targeting E. coli bacteria causing recurrent urinary tract infections. This week they closed a$14.4 million deal with CARB-Xto advance development of LBP-KP01, another CRISPR Cas3-enhanced bacteriophage (crPhage) product, targeting K. pneumoniae.The initial indication will be to target recurrent UTIs, then development for targeting lung infections (pneumonia), intra-abdominal infections (IAIs) and bacteremia.Together, the two cocktails have the potential to treat more than 90% of UTIs.Auniquedual mechanismof bacteria-hunting bacteriophages along with the DNA-targetingCRISPR-Cas3makesLocuscandidates significantly more effective at killing the targeted bacteria cells, regardless of whether they are resistant to antibiotics.Both the U.S. Centers for Disease Control and Prevention (CDC) and World Health Organization (WHO) have identified antibiotic-resistant K. pneumoniaeas an urgent and serious public health threat requiring development of new treatments.

Memo Therapeutics

Swiss innovator Memo Therapeutics has raised over$15.3 million in a Series Bprimarilyto advance its COVID-19 antibody treatment.The company entered into a partnership with NorthwayBiotechpharmain August to manufactureMTX-COVAB, which is currently going through a fast-tracked development path as an immunotherapy and a preventative of the novel coronavirus. Memo plans to begin clinical studies in 2021. Proceeds will also be used to advance its neutralizing antibody MTX-005 against BK virus infection in renal transplant patients into Phase II studies."We believe Memo Therapeutics AG has taken innovation in the field of antibody discovery to the next level. Their ability to exploit the power of microfluidic single-cell molecular cloning could not only serve to move one step closer to conquer the COVID pandemic but also potentially other infectious diseases and cancer, said Dr. Robert Schier, Investment Director atSwisscantoInvest.

Trailhead Biosystems

Pushing the boundaries of dimensional testing, Trailhead is increasing the speed, lowering the cost and reducing the risk of developing cell therapies. A$6.6 millioninfusion of cash will expand the companys High Dimensional Design of Experiments platform (HD-DoE) to support the generation of multiple specialized human cells with therapeutic properties and theirpilotscalemanufacturing.Trailheads aim is to rapidly develop the capability to create highly pure, specialized human cell types for regenerative medicine and therapeutic purposes at an industrial scale. "Biology is complex, but conventional science is not," saysJan Jensen, Ph.D., Chief Executive Officer and founder of Trailhead Biosystems. "We created Trailhead Biosystems to address key limitations in the scientific process, unlocking a deeper understanding of biology that will enable us to better control it."

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Biopharma Money on the Move: November 4-10 - BioSpace

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Yale scientists discover genes that could be COVID treatment targets – Yale Daily News

November 11th, 2020 11:52 am

Eric Wang, Senior Photographer

Researchers at the Yale School of Medicine have identified genes that could be future targets for COVID-19 treatments.

In a partnership with the Broad Institute of MIT and Harvard, researchers at the School of Medicines Department of Immunology performed a genome-wide CRISPR screen, which evaluated each of the 20,000 preselected genes in the African green monkey genome that could affect coronavirus infections. This technique allowed researchers to quickly and effectively evaluate the genetic information from over a million modified cells.

According to assistant professor of laboratory medicine and immunobiology Craig Wilen, using a genetically modified virus called a CRISPR library, certain genes of interest were knocked out in the monkey cells in order to stop their products from being made and used in the cell. The cells were then infected with the coronavirus, and those that survived were analyzed to detect what genes were knocked out and could be affecting viral infection. The results pointed to over 25 possible host genes related to infection, but two specific hits for receptor and enzyme encoding genes seemed most promising as treatment targets.

We think its possible that you could develop drugs that affect human targets, Wilen said. And the advantage there is it would be conserved and function across different coronaviruses.

Jin Wei, the studys primary author and a postdoctoral associate at Wilens lab, explained that he was directly involved in identifying the host genes critical to coronavirus infections.

According to Wei, the lab had prior experience in studying the modes of infections of RNA viruses such as MERS and other coronaviruses. This previous work meant they were uniquely prepared to study the genes that affect the SARS-CoV-2 virus infection which had never been done before.

We found there is no CRISPR screens for host genes for any coronaviruses, which may reveal novel therapeutic targets and inform our understanding of COVID-19 pathogenesis, Wei wrote in an email to the News. We leveraged our expertise with RNA virus pathogenesis and CRISPR screening to identify the host factors that are essential for SARS-CoV-2 infection.

Mia Madel Alfajaro, another postdoctoral associate at Wilens lab, explained that they found two important genes during their screening process that, when absent, helped cells survive the virus infection. One of them encodes the SARS-Cov-2 receptor, while the other is translated into an enzyme that aids the coronavirus in entering the cell.

Scientists at the Broad Institute provided the Yale researchers with the CRISPR library to be used in the monkey cells and the analyses they ran on the surviving cells genetic material.

Our group has significant expertise and capacity in terms of making CRISPR libraries, turning them from an idea into an actual test tube of particles, John Doench, an institute scientist at the Broad Institute, said.

Wei and Doench believe one of the main findings of the study comes from the comparison between SARS-CoV-2 and another coronavirus, MERS-CoV. These genetic hits that affect coronaviruses in general could be useful in finding pan-coronavirus treatments, according to Wilen.

According to Alfajaro, one of the limitations of this study is that there is no way to mimic exactly the behavior of a human beings lung cells, which means there are still many steps to be taken before a treatment is developed.

If we have [found] molecules, peptides or chemical inhibitors or COVID-19, that would be great, Alfajaro said. It will take time because some of the hits need to be developed.

Alfajaro believes drugs that are already approved by the FDA could be a possible focus for future research, since some of the drugs already on the market could affect the molecules found during the screening.

Doench does not believe that the main goal of the study was finding a drug that would end the pandemic. He argued that a future drug may be able to target the genes they found to create therapeutics for COVID-19, but that more work needs to be done.

From doing a genetic screen in a cell line in a monkey to having a drug target, there is so much science that needs to happen, he said.

According to Doench, the only way to stop the pandemic is through social distancing, wearing masks and eventually developing a vaccine.

The Broad Institute of MIT and Harvard was founded in 2004.

Beatriz Horta | beatriz.horta@yale.edu

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Generation Bio Reports Third Quarter 2020 Business Updates and Financial Results – GlobeNewswire

November 11th, 2020 11:52 am

CAMBRIDGE, Mass., Nov. 10, 2020 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO) is an innovative genetic medicines company creating a new class of non-viral gene therapy. Today the company reported recent business highlights and third quarter financial results.

2020 continues to be a year of progress and execution for Generation Bio as we advance our non-viral gene therapy approach, said Geoff McDonough, M.D., president and chief executive officer of Generation Bio.Despite the challenges of the COVID-19 pandemic, we remain on-track to advance our lead programs into IND-enabling preclinical development next year. We believe our strong cash balance positions us well to execute on our ambitions into 2023.

Recent Business Highlights

This period marks an expansion of our focus beyond our platform to include preclinical development and readiness for the clinic. To support this effort, I am pleased to announce the appointment of Tracy Zimmermann to chief development officer. Tracy will lead our pre-clinical development programs across the portfolio, building on the excellent foundation she has created since joining Generation Bio in 2018. Tracys new role allows for Doug Kerr to focus on building our clinical development capabilities as chief medical officer. Together with Matt Stanton, our chief scientific officer, Tracy and Doug make a terrific, complementary leadership team for our R&D work, Dr. McDonough said. A summary of the leadership appointments follows.

Dr. McDonough continued, Separately, Mark Angelino, our chief operating officer and co-founder, will undertake a planned transition from Generation Bio to return to early stage company formation work in early 2021. Although too soon for farewells, we are indebted to Mark for his vision and leadership in forming and building our community here.

Selected Anticipated Company Milestones

Upcoming Investor Conference Presentations

Management will present at two upcoming investor conferences:

Live webcasts of the presentation and the fireside chat will be available in the investor section of the company's website atwww.generationbio.com. The webcasts will be archived for 60 days following the presentations.

Financial Results

About Generation Bio

Generation Bio is an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases. The companys non-viral platform incorporates a proprietary, high-capacity DNA construct called closed-ended DNA, or ceDNA; a cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed. The ctLNP is designed to deliver large genetic payloads, including multiple genes, to specific tissues to address a wide range of indications. The companys efficient, scalable manufacturing process supports Generation Bios mission to extend the reach of gene therapy to more people, living with more diseases, in more places around the world.

For more information, please visit http://www.generationbio.com.

Forward-Looking Statements

Any statements in this press release about future expectations, plans and prospects for the Company, including statements about its strategic plans or objectives, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities, the initiation and completion of preclinical studies and clinical trials and clinical development of the Companys product candidates; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; expectations for regulatory approvals to conduct trials or to market products; challenges in the manufacture of genetic medicine products; the Companys ability to obtain sufficient cash resources to fund the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements; the impact of the COVID-19 pandemic on the Companys business and operations; as well as the other risks and uncertainties set forth in the Risk Factors section of the Companys most recent quarterly report on Form 10-Q, and in subsequent filings the Company may make with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Companys views as of the date hereof. The Company anticipates that subsequent events and developments will cause the Companys views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Companys views as of any date subsequent to the date on which they were made.

Contacts:

InvestorsChelcie ListerTHRUST Strategic Communicationschelcie@thrustsc.com910-777-3049

MediaStephanie SimonTenBridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

GENERATION BIO CO.CONSOLIDATED BALANCE SHEET DATA(unaudited)(in thousands)

GENERATION BIO CO.CONSOLIDATED STATEMENTS OF OPERATIONS(unaudited)(in thousands, except share and per share data)

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Generation Bio Reports Third Quarter 2020 Business Updates and Financial Results - GlobeNewswire

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Exploring Genetic Variation and COVID-19 Clinical Variability – Technology Networks

November 11th, 2020 11:52 am

One of the biggest challenges that scientists and healthcare professionals are facing during the COVID-19 pandemic is the high rate of clinical variability. Whilst some patients present as asymptomatic, others are developing more severe symptoms such as pneumonia, and some cases are ultimately proving fatal. Why?The answer remains elusive; however, extensive research is exploring the possible contribution our genetics may be having. Genetic variation differences in the DNA sequences that make up our genome can impact our response to infectious diseases.

GoodCell uniquely measures and monitors inherited and acquired genetic variations in stem cells and other nucleated cells in our blood over time. Technology Networks recently spoke with Dr Salvatore Viscomi, chief medical officer at GoodCell, and attending physical at Baystate Health, to explore factors that might influence COVID-19 risk, and to discuss how the company is working to identify at-risk individuals through genetic variation analysis.

Molly Campbell (MC): For our readers that may be unfamiliar, can you discuss why medicine is moving towards a personalized approach, and why this is important considering genetic variation?Salvatore Viscomi (SV): Healthcare has traditionally taken the approach of one size fits all in defining individual risk for a disease and prescribing therapy for it. Understanding the differences between individuals on a molecular level optimizes assessment of an individuals susceptibility to a certain disease and predicting response to pharmacological therapy. Genomics plays the most important role in the emergence of personalized therapy. Identifying the inherited and acquired genetic variation will direct personalized screening and prevention plans and inform bespoke medical therapies.

MC: We know that there is high clinical variability across COVID-19 patients. How might genetic variation be contributing here, and what published evidence exists to support this?SV: Understanding immune response is critical to identifying individuals at high risk of severe morbidity and mortality. Emerging research suggests that accumulated genetic variation in our blood cells may be associated with a dysfunctional inflammatory response to COVID-19 leading to its pulmonary, cardiac and coagulopathic complications.

In a recent study published by JAMA Cardiology, researchers demonstrated an association between the presence of accumulated genetic change in our blood cells and a pro-inflammatory immune response that resembles the exaggerated cytokine release syndrome (CRS) manifested in COVID-19-positive patients. Direct evidence has emerged more recently; a study published in Cancers examined patients hospitalized with COVID-19 and found a significantly higher prevalence of accumulated genetic variation in all age groups compared to age-matched control groups.

MC: What impact might genetic variation in COVID-19 patients have on efforts to develop therapeutics or preventives, such as vaccines?SV: Identifying highly susceptible individuals through blood testing could have many applications. As an initial wave of vaccines move through Phase III trials and potentially come to market, we would have the data to determine prioritization of vaccinations when one is available. Business and government sectors need insight into risk factors that can inform inoculation strategies for societys most vulnerable, inform decisions around who should and should not be on the front lines, and give people more control when making personal decisions about how to mitigate individual risk. The broader field of genetics offers a window into the potential to correlate inherited and acquired gene mutations with immune response for the betterment of society, providing a more robust and accurate set of risk factors unique to every individual.

Furthermore, in high-risk individuals, targeting inflammation may be a clinical strategy to mitigate its clinical consequencesin COVID-19. For example, we may identify patients who are most responsive to pro-inflammatory inhibitors. Implementing measures intended to reduce subjects exposure to the infection or likelihood of contracting such infection through self-isolation, quarantine or social distancing may be advised.

MC: Can you explain the aims of GoodCell, and what the company does in terms of "banking blood for life"?SV: GoodCells mission is to extend and improve the quality of life through technology powered by our own cells. Blood is the author of our bodies, and can both cure as well as cause disease. Through our proprietary data aggregation and analytics technology platform, which aims to decode our blood cells and harness their insights to advance population and personal health, we empower individuals to identify, track and mitigate health risks. By getting ahead of their health risks, we enable the potential for a better life. In addition, through our personal biobanking service, long-term storage of your healthiest cells provides the opportunity for potential use in future therapeutics if you need them you are your best donor.

MC: Does GoodCell measure other "omics" parameters outside of genomics (DNA measurements and analysis), such as proteomics or metabolomics?SV: GoodCells platform leverages the power of blood to assess risk as such, we of course look at acquired and inherited genetic changes, but there are many more opportunities afforded by blood to understand and assess risk including routine blood chemistry tests, tests for biomarkers of disease, including emerging capabilities in liquid biopsy for earlier detection of solid tumor cancers. Ultimately, we are always looking to incorporate novel health and data insights into our product platform to better inform both an individuals health, as well as population-based health. Transcriptomics, epigenomics and metabolomics are but a few of the opportunities we are evaluating.

MC: What work is GoodCell currently conducting in the COVID-19 space?SV: GoodCell is currently engaged in a research collaboration with the New York Blood Center to evaluate how specific acquired and inherited genetic variation contribute to COVID-19 severity and recovery. We are analyzing genetic variation in asymptomatic/mildly symptomatic patients compared to hospitalized/ICU patients. GoodCell will evaluate the genetic variation in the collected samples using our proprietary assay platform to identify and validate their association with COVID-19 morbidity and mortality.

Salvatore Viscomi was speaking to Molly Campbell, Science Writer, Technology Networks.

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Flaws emerge in modeling human genetic diseases in animals – The Conversation US

November 11th, 2020 11:52 am

My lab, based at the University of Southern California Keck School of Medicine, uses zebrafish to model human birth defects affecting the face. When I tell people this, they are often skeptical that fish biology has any relevance to human health.

But zebrafish have backbones like us, contain by and large the same types of organs, and, critically for genetic research, share many genes in common. My group has exploited these genetic similarities to create zebrafish models for several human birth defects, including Saethre-Chotzen Syndrome, in which the bones of the skull abnormally fuse together, and early-onset arthritis.

Similar to fish, our bodies develop under the control of about 25,000 genes. The trick is finding out what each gene does. Stunning advances such as CRISPR-based molecular scissors, for which the Nobel Prize in chemistry was just awarded, allow us to precisely change genes, and designer chemicals can silence particular genes. In a recent study from our group published in Nature, however, we find that these tools are still far from perfect. Although CRISPR now allows us to efficiently generate lab animals that can pass human disease mutations onto the next generation, claims that simply injecting CRISPR into embryos or silencing genes with designer chemicals can accurately model human genetic disease are being questioned.

Finding the precise mutation that causes a particular birth defect or a late-onset disease can be tedious work. The human genome is made up of 3 billion building blocks called DNA nucleotides, and changing just one of these can cause devastating birth defects.

To figure out if we have identified the right disease-causing mutation in humans, we typically engineer the same change into the genome of a lab animal. We then breed these animals to generate babies with the disease mutation and look for the appearance of defects similar to those in human patients.

We study zebrafish because they are small, which means we can grow thousands of different genetically modified animals. We routinely use CRISPR to engineer fish that pass on a gene-breaking mutation to the next generation.

We then study the appearance of defects similar to those in humans lacking these genes in essence creating personalized zebrafish avatars of genetic disease. As zebrafish embryos are transparent and develop rapidly outside the mother, they are particularly useful for understanding how human disease mutations disrupt normal development.

Even in zebrafish, engineering animals to lack particular genes can be a time-consuming process. In my lab, we first create gene mutations in embryos, grow these fish to adulthood and then breed fish together to look at defects in the next generation.

This whole process can take a year or longer. Unsurprisingly, many labs are attempting shortcuts. Some are injecting large quantities of CRISPR molecular scissors into animals and then looking for defects in these same animals. Others are using chemicals to turn off, or silence, genes in the embryo rather than permanently changing the genes.

More and more frequently studies like this are calling into question the accuracy of these shortcuts. In animals that have been injected with CRISPR molecular scissors, not every cell is changed in the same way. And the chemicals used to silence genes appear to have unintended consequences, poisoning the embryo in a generic way.

For example, researchers in Spain recently reported that a gene called prrx1a was critical for the proper development of the heart. To figure this out, they silenced prrx1a in zebrafish with chemicals. Then, in a second experiment, they injected CRISPR molecular scissors into zebrafish embryos and examined them just one day later for heart defects.

In contrast, we completely removed the prrx1a gene and looked at generations of fish lacking this gene. Hearts in these mutant fish developed perfectly normally, showing that prrx1a was not critical for heart development. Instead, we showed that the heart defects seen upon chemical treatment in the Spanish study were due to a general poisoning of the embryos unrelated to the prrx1a gene. Animals simply injected with CRISPR also showed defects not seen upon complete removal of the prrx1a gene, although the exact reasons for these differences remain a source of active debate.

And not just our group has noticed these flaws. Using similar gene removal as we reported, the group led by Didier Stainier refuted a study that had used CRISPR injection and gene silencing to link the tek gene to blood vessel development. Given the number of studies relying on gene silencing in lab animals, as opposed to engineering the DNA mutations, the causative genes for many human diseases may need to be reevaluated.

The desire for speed in research must not come at a cost of accuracy and reproducibility.

The good news is that, with the ease of CRISPR, we now know how to engineer the right types of mutations in lab animals to validate human disease mutations. By creating lab animals such as zebrafish that have the mutations engineered into their genomes and then observing whether their offspring develop the same diseases as patients with the mutations, we can be confident in having identified the right human disease gene.

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Getting it right is important for accurately counseling prospective parents of their genetic risks for certain birth defects, as well as identifying the relevant genes that can be targeted to prevent or even reverse disease.

Science is constantly evolving. While the ability to engineer the genome with CRISPR is opening up endless possibilities for human genetics, researchers must also recognize the limitations of new technologies. Although rapid, directly injecting CRISPR or silencing genes with chemicals gives misleading results too often. In order to confidently identify causative mutations linked to human disease, we will need to continue to study lab animals engineered to carry and pass on the same DNA changes as found in human patients.

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Why Its a Big Deal If the First Covid Vaccine Is Genetic – WIRED

November 11th, 2020 11:52 am

On Monday morning, when representatives from the drug company Pfizer said that its Covid-19 vaccine appears to be more than 90 percent effective, stocks soared, White House officials rushed to (falsely) claim credit, and sighs of relief went up all around the internet. Dear World. We have a vaccine! Best news since January 10, tweeted Florian Krammer, a virologist and vaccinologist at the Mount Sinai School of Medicine (who also happens to be a participant in the Pfizer Covid-19 vaccine trial).

Here's all the WIRED coverage in one place, from how to keep your children entertained to how this outbreak is affecting the economy.

But having a press release from a pharmaceutical company saying a vaccine works is very different from actually having a vaccine that works. Pfizer, and its German partner on the vaccine, BioNTech, have yet to release any data from their Phase III trial. The findings this week are based on the trials first interim analysis, conducted by an outside panel of experts after 94 of the 43,538 participants contracted the coronavirus. That analysis suggests that most of the people who became ill had received a placebo, instead of the vaccine. But it doesnt say much beyond that. (More on why that matters, later.)

And logistically, theres still a lot that has to happen before people who arent study subjects can start rolling up their sleeves. Pfizer researchers are now collecting at least two months worth of safety follow-up data. If those findings raise no red flags, the company could then apply for an emergency use authorization from the US Food and Drug Administration. Only then could execs start doling out the 50 million or so doses they expect to make by the end of the year, a process complicated by the fact that until its ready to be shot into someones arm, Pfizers vaccine needs to be kept at temperatures downwards of -80 degrees Fahrenheit, which is way colder than the usual vaccine cold chain. Completing the immunization also requires two doses given three weeks apart. Oh yeah, and states that at this moment are trying to do all the other things you have to do to prepare for such a complicated immunization pushhiring vaccinators, setting up digital registries, deciding who will get vaccine priorityare doing so without any extra money dedicated to the effort.

Those are a lot of caveats. But still, theres reason to be hopeful. If the results hold up, a Covid-19 vaccine thats 90 percent effective will have vastly exceeded the efficacy bar set by the FDA. That level of protection would put it up there with the measles shot, one of the most potent vaccines developed to date.

The arrival of an effective vaccine to fight SARS-CoV-2 less than a year after the novel coronavirus emerged would smash every record ever set by vaccine makers. Historic isnt even the right word, says Larry Corey of the Vaccine and Infectious Disease Division at the Fred Hutchinson Cancer Center. A renowned virologist, Corey has spent the last three decades leading the search for a vaccine against the virus that causes AIDS. Hes never seen an inoculation developed for a new bug in under five years, let alone one. Its never happened before, never, not even close, he says. Its just an amazing accomplishment of science.

And perhaps even more monumental is the kind of vaccine that Pfizer and BioNTech are bringing across the finish line. The active ingredient inside their shot is mRNAmobile strings of genetic code that contain the blueprints for proteins. Cells use mRNA to get those specs out of hard DNA storage and into their protein-making factories. The mRNA inside Pfizer and BioNTechs vaccine directs any cells it reaches to run a coronavirus spike-building program. The viral proteins these cells produce cant infect any other cells, but they are foreign enough to trip the bodys defense systems. They also look enough like the real virus to train the immune system to recognize SARS-CoV-2, should its owner encounter the infectious virus in the future. Up until now, this technology has never been approved for use in people. A successful mRNA vaccine wont just be a triumph over the new coronavirus, itll be a huge leap forward for the science of vaccine making.

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Genosity and Igentify Announce Strategic Collaboration to Integrate Their Offerings, Enabling Comprehensive and Holistic Precision Medicine Solution…

November 11th, 2020 11:52 am

NEW YORK--(BUSINESS WIRE)--Genosity, Inc., an innovative biotechnology company that provides comprehensive software and laboratory solutions to enable precision medicine, announced today that it has entered into a strategic collaboration agreement with Igentify, a digital health technology company that has developed a digital genetic counselor assistant to improve provider interaction with patients for onboarding, enrollment, consenting, as well as facilitating counseling for personalized genetic testing results. Under the terms of agreement, Igentify will integrate its digital platform with Genositys Integrated Genomic Toolkit (IGT), and both companies will comarket the combined solution.

The integrated product, with an ability to interface with traditional EMR systems, is a complete solution for any health system offering genetic testing. This comprehensive platform enables a genomic testing order for a patient to be originated by a third-party health system, explained to the patient by a personalized and dynamic multimedia presentation which takes the patient through a genetic testing consent process and literacy assessment; processed in LIMS upon receipt of the patients specimen; the genomic data analyzed, interpreted and reported, and a full medical report communicated to the patient with customized, patient friendly PDF and video reports.

Our goal is to enable health systems to fully embrace precision medicine to the benefit of their patients which requires more than just performing or sending out a test. It is about enabling those who most directly touch patient lives. Our integrated system not only provides a solution for the operational aspects of all genetic testing, from patient enrollment to result report, but also enables our clients to build their data driven knowledge base that can help them advance research, improve patient care and engage in commercial collaborations. It is our pleasure to work with Igentify. Both executive teams have worked on solving operational challenges in genomics for a long time; this collaboration is the result of our work and experience in this critically important field, said Dr. Marc Grodman CEO and Cofounder of Genosity.

Dr. Doron Behar, CEO and Cofounder of Igentify, said, Genomic testing is a pillar of precision medicine which will affect the health of each individual worldwide and shape healthcare policies and prevention medicine practices. Demand for genetic testing is rising quickly, but a shortage of genetic counselors makes it impossible to scale up personal genetic counseling services, leaving health care providers unable to fulfill patient needs. Igentify is proud to combine our efforts with Genosity to allow the first of its kind comprehensive software solution that enables healthcare providers to establish genomic core centers of excellence comprising patient counseling, bioinformatics and a laboratory information management system for better patient care.

About Genosity:Genosity is a life science biotechnology company that employs its expertise, novel software solutions and laboratory services for both somatic and germline applications to enable its strategic partners to fully realize the value of precision medicine for both the research and clinical markets. For more information, please visit us at https://genosity.com.

About Igentify:Igentify is a digital health technology company with expertise in analyzing, interpreting and transforming complex genomic molecular results into medically supervised genetic reports. Igentify enables personalized genetic counseling services at scale. The mission of Igentify is to create accessible and actionable genomic data for all. For more information, please visit us at https://igentify.com.

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Passage Bio and Invitae Announce Collaboration to Facilitate Genetic Testing to Support Early Diagnosis and Greater Awareness of Clinical Trials for…

November 11th, 2020 11:52 am

PHILADELPHIA, Nov. 09, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, and Invitae (NYSE: NVTA), a leading medical genetics company, announce a collaboration to facilitate genetic testing and support early identification of GM1 gangliosidosis (GM1) through Invitaes Detect Lysosomal Storage Disorders (Detect LSDs). The two companies also are partnering to provide educational clinical trial information to clinicians and patients.

Early identification and intervention are crucial steps in providing effective treatment to patients with GM1, particularly in the infantile form where onset occurs within the first six months of a patients life, said Bruce Goldsmith, Ph.D, president and chief executive officer of Passage Bio. By combining Invitaes no-charge testing and counseling with information on clinical trials, clinicians will be able to intervene sooner and more efficiently, shortening the timeline to reliable diagnosis. As we plan to initiate our Phase 1/2 trial for PBGM01 soon, this partnership will be an important part of our support for patients and will also serve as a key resource to patients with GM1 and their families.

The Detect LSDs program offers genetic testing and genetic counseling at no charge to patients to encourage earlier diagnosis of lysosomal storage disorders like GM1 and, as a result, earlier access to clinical trials. In addition, Invitae provides clinical trial information and education to clinicians and patients who may benefit. Currently, the Detect LSDs program is available to patients within the United States and Canada.

Increasing access to genetic testing supports earlier diagnosis, enables clinical trials to develop new treatments and helps clinicians provide precision therapies sooner for better overall outcomes, said Robert Nussbaum, M.D., chief medical officer of Invitae. Were pleased Passage Bio has joined us in this effort.

Additional details, as well as terms and conditions of the Detect LSDs program, can be found at https://www.invitae.com/en/detectLSDs/.

About GM1

GM1 gangliosidosis (GM1) is a rare and often life-threatening monogenic recessive lysosomal storage disease caused by mutations in the GLB1 gene, which encodes lysosomal acid beta-galactosidase (-gal). Reduced -gal activity results in the accumulation of toxic levels of GM1 ganglioside in neurons throughout the brain, causing rapidly progressing neurodegeneration. GM1 manifests as a continuum of disease and is most severe in the Infantile form, which is characterized by onset in the first 6 months of life with hypotonia (reduced muscle tone), progressive CNS dysfunction, and rapid developmental regression. Life expectancy for infants with GM1 is two to four years, and infantile GM1 represents approximately 62.5% of the incidence of 0.5 to 1 in 100,000 live births. Currently, there are no approved disease-modifying therapies available.

About Passage Bio

At Passage Bio (Nasdaq: PASG), we are on a mission to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvanias Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with unparalleled access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at http://www.passagebio.com.

About Invitae

Invitae Corporation (NYSE: NVTA) is a leading medical genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website atinvitae.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including our planned IND submissions, initiation of clinical trials and the availability of clinical data from such trials; our expectations about our collaborators and partners ability to execute key initiatives; our expectations about manufacturing plans and strategies; our expectations about cash runway; and the ability of our lead product candidates to treat the underlying causes of their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as aim, anticipate, believe, could, estimate, expect, forecast, goal, intend, may, might, plan, potential, possible, will, would, and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

For further information, please contact:

Investors:Sarah McCabe and Zofia MitaStern Investor Relations, Inc.sarah.mccabe@sternir.com and zofia.mita@sternir.com

Media:Media:Gwen FisherPassage Bio215-407-1548gfisher@passagebio.com

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Beam Therapeutics Announces Business and Pipeline Progress and Reports Third Quarter 2020 Financial Results – GlobeNewswire

November 11th, 2020 11:52 am

BEAM-201, an Off the Shelf Allogeneic CD7-Targeting CAR-T, Named as Development Candidate for Treatment of T-ALL; First Cell Therapy Featuring Four Simultaneous Genetic Edits; Demonstrates 96-99% On-target Editing and In Vivo Proof of Concept of Tumor Clearance

Multiple Upcoming Data Presentations Demonstrate Strength and Breadth of Base Editing Platform, Including First Preclinical Data from GSDIa Program

$135 Million in Capital Raised through Successful Follow-on Offering

CAMBRIDGE, Mass., Nov. 10, 2020 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported pipeline updates, recent business highlights and third quarter 2020 financial results.

2020 has been a year of significant progress for Beam, said John Evans, chief executive officer of Beam. Since the start of the year, weve named three development candidates from our portfolio, now including BEAM-201, our multiplex editing program for the treatment of T-cell acute lymphoblastic leukemia. We are also pleased to report that were on track to submit our first IND in the second half of 2021, with BEAM-101 for the treatment of sickle cell disease. The continued advancement of our pipeline is a testament to both the strength and breadth of our base editing platform and our exceptional team. Combined with the capital weve added to our balance sheet, we are well positioned to continue our strategy of advancing multiple programs to the clinic in parallel, in the hope of providing much-needed new treatment options for patients with serious diseases.

Base Editing Progress

Upcoming Base Editing Data Presentations

Beam will also report data during an oral presentation at AASLD from its Alpha-1 Antitrypsin Deficiency (Alpha-1) program. Details of the presentation are as follows:

Recent Business Highlights

Upcoming Investor Conference Presentation

John Evans, chief executive officer, will participate in a fireside chat during the Jefferies Virtual London Healthcare Conference on Thursday, November 19, 2020 at 4:25 p.m. GMT/11:25 a.m. ET.

The live webcast will be available in the investor section of the company's website at http://www.beamtx.com. The webcast will be archived for 60 days following the presentation.

Third Quarter 2020 Financial Results

About Beam Therapeutics

Beam Therapeutics(Nasdaq: BEAM) is a biotechnology company developing precision genetic medicines through the use of base editing. Beams proprietary base editors create precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases. For more information, visitwww.beamtx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: the expected timing of filing investigational new drug applications; our ability to advance programs to the clinic; the sufficiency of our cash position; expected presentations at upcoming conferences; and the therapeutic applications and potential of our technology, including our ability to develop precision genetic medicines for patients through base editing. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the potential impact of the COVID-19 pandemic; that preclinical testing of our product candidates and preliminary or interim data from preclinical and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that enrollment of our clinical trials may take longer than expected; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; and the other risks and uncertainties identified under the heading Risk Factors in our Annual Report on Form 10-K for the year ended December 31, 2019, our Quarterly Report on Form 10-Q for the quarters ended March 31, 2020, June 30, 2020, and September 30, 2020, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.

Contacts:

Investors:Chelcie ListerTHRUST Strategic Communicationschelcie@thrustsc.com

Media:Dan Budwick1ABdan@1abmedia.com

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Homology Medicines Announces Presentation of Positive Data from the Dose-Escalation Phase of the pheNIX Gene Therapy Trial for Adults with PKU | DNA…

November 11th, 2020 11:52 am

DetailsCategory: DNA RNA and CellsPublished on Friday, 06 November 2020 17:53Hits: 894

- Marked Reductions in Phe Observed at Two Doses -

- Achieved Goal with Plans to Advance to Randomized, Concurrently Controlled Expansion Phase of Trial -

BEDFORD, MA, USA I November 06, 2020 I Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the presentation of positive data from the dose-escalation portion of the Phase 1/2 gene therapy pheNIX clinical trial for adults with phenylketonuria (PKU). The results showed that product candidate HMI-102 was generally well-tolerated, and resulted in marked reductions in phenylalanine (Phe) and the Phe-to-tyrosine (Tyr) ratio (Phe/Tyr ratio) at two doses. Phe is a registrable endpoint in PKU, and the Phe/Tyr ratio is a clinically relevant diagnostic measurement for PKU. With these positive results, Homology is progressing to the randomized, concurrently controlled expansion phase of the trial, which has the potential to be converted to a registrational trial.

The data were presented today in an oral presentation by Olaf Bodamer, M.D., Ph.D., FACMG, FAAP, Park Gerald Chair in Genetics & Genomics and Associate Chief of Genetics & Genomics at Boston Childrens Hospital, and principal investigator of the pheNIX trial, during the New England Consortium of Metabolic Programs (NECMP) annual meeting, which is focused on new research in metabolic disorders. NECMP includes metabolic clinics, healthcare providers, patient organizations and others dedicated to increasing knowledge of metabolic disorders and improving delivery of healthcare to patients.

This is the first-ever PKU gene therapy clinical trial, and I am excited to share these data with the PKU community as I believe they demonstrate the potential of HMI-102 to treat the underlying genetic cause and reduce the therapeutic burden for patients and their families, stated Dr. Bodamer. PKU is a challenging condition, and a treatment that establishes normal metabolism could change the prognosis for patients with this rare genetic disorder. We look forward to participating in the next phase of the study.

We are pleased to have met the goals of the dose-escalation portion of the trial, which were evaluation of safety and efficacy of a single I.V. administration of HMI-102 and dose determination for the expansion phase of the trial, stated Gabe Cohn, M.D., Chief Medical Officer of Homology Medicines. Even as many patients self-liberalized their diets, there were patients at the mid- and high-doses with plasma Phe values below 360 mol/L and/or 600 mol/L, and one of these patients achieved a Phe level within the normal range. This is the first time a genetic medicines approach has achieved these results in patients with PKU. We have learned a tremendous amount in the dose-escalation phase and are applying these learnings to the expansion phase of the trial, which we anticipate initiating in early 2021.

Dr. Cohn continued, We greatly appreciate the PKU community of patients, clinicians and caregivers who have participated in this first phase of the pheNIX trial, and we look forward to working together during the next phase.

As of the data cutoff date of October 19, 2020, six patients in the dose-escalation phase of the pheNIX trial had received gene therapy product candidate HMI-102 across three dose cohorts (low-dose Cohort 1, n=2; mid-dose Cohort 2, n=2; high-dose Cohort 3, n=2). Cohorts included males and females, with an age range of 21-49 and time in study ranging from 13 weeks to 52 weeks (end of study).

Safety ObservationsHMI-102 was generally well-tolerated, and there were no treatment-related serious adverse events (SAEs). There were no clinically significant changes in ECG or vital signs and no clinical signs of complement activation. The Grade 1 and 3* alanine aminotransferases (ALTs) observed in Cohorts 2 and 3, which is common in AAV-based gene therapy, were managed with increased steroids when necessary. The patients who experienced Grade 3 ALTs had pre-existing underlying immune conditions. An independent data monitoring committee, which provided guidance throughout the pheNIX trial, concluded that there were no safety concerns related to bilirubin, and that ALT elevations may be associated with reduced efficacy.

Updates to the expansion phase of the pheNIX trial, including key learnings related to patient selection, monitoring and steroid regimen, are being incorporated.

Efficacy Observations

Cohort 1 (Low-Dose)Through 52 weeks, patients in Cohort 1 continued to show no meaningful reductions in Phe.

Cohorts 2 and 3 (Mid- and High-Dose)The mean percent change from baseline in Phe observed in patients in Cohorts 2 and 3 were significant, compared to Cohort 1**. These Phe reductions occurred while patients self-liberalized their diets.

Through 48 weeks, one patient in Cohort 2 had Phe levels of <360 mol/L and/or <600 mol/L*** at multiple timepoints and had reached a minimum Phe level of 42 mol/L, compared with a baseline level of 1,010 mol/L. Through 13 weeks, one patient in Cohort 3 had a Phe level <360 mol/L and several Phe levels <600 mol/L at multiple timepoints and had reached a minimum Phe level of 303 mol/L, compared with a baseline level of 1,060 mol/L.

In Cohorts 2 and 3, Phe reductions were greater among patients with Grade 1 ALTs compared to patients with Grade 3 ALTs****; ALT elevations were managed with increased steroids when necessary. It appears higher ALT elevations may limit therapeutic activity, but can be managed with a modified steroid regimen, which is being incorporated into the expansion phase.

Expansion PhaseBased on the safety and efficacy results observed in the dose-escalation phase, Homology is advancing to the randomized, concurrently controlled, dose expansion phase of the pheNIX trial, which has the potential to be converted to a registrational trial.

All cohorts in the dose-escalation phase showed an acceptable safety profile and certain patients in Cohorts 2 and 3 showed marked Phe reductions. Based on these collective data, Homology has selected two doses for the expansion phase: the mid-dose from Cohort 2 and a dose between the doses in Cohorts 2 and 3. The Company believes the latter dose has the potential to improve Phe reductions while reducing steroid exposure that was required at the high-dose. The Company believes that advancing two doses in parallel provides the potential to convert to a registrational trial quickly with the optimal dose as the expansion phase does not include staggered dosing between patients.

Webcast/Conference Call Homology management and Dr. Bodamer will host a conference call and webcast today, Friday, November 6 at 4:30 p.m. ET. The webcast will be accessible on Homologys website in the Investors section, and the webcast replay will be available on the website for 90 days following the presentation. To access using the conference call line, dial (866) 244-8091 (U.S./Canada toll-free) or (602) 563-8623, with Conference ID 7394503.

About HMI-102HMI-102 is an investigational gene therapy in clinical development for the treatment of phenylketonuria (PKU) in adults. HMI-102 is designed to encode the PAH gene, which is mutated in people with PKU, delivered via the liver-tropic AAVHSC15 vector. Homology has received Fast Track Designation and orphan drug designation for HMI-102 from the U.S. Food and Drug Administration (FDA), and orphan drug designation from the European Medicines Agency (EMA).

About Phenylketonuria (PKU)PKU is a rare inborn error of metabolism caused by a mutation in thePAHgene. PKU results in a loss of function of the enzyme phenylalanine hydroxylase, which is responsible for the metabolism of phenylalanine (Phe), an amino acid obtained exclusively from the diet. If left untreated, toxic levels of Phe can accumulate in the blood and result in progressive and severe neurological impairment. Currently, there are no treatment options for PKU that target the underlying genetic cause of the disease. According to the National PKU Alliance, PKU affects nearly 16,500 people in the U.S. with approximately 350 newborns diagnosed each year. The worldwide prevalence of PKU is estimated to be 50,000 people.

About Homology Medicines, Inc. Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homologys proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicinesin vivoeither through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visitwww.homologymedicines.com.

*ALT Grades based on Common Terminology Criteria for Adverse Events (CTCAE) Version 5**P<0.004; Post-hoc comparison of Cohort 1 vs Cohorts 2&3 using repeated measures MANOVA/regression analysis***U.S. and EU PKU treatment guidelines described in: Vockley J et al. Phenylalanine hydroxylase deficiency: diagnosis and management guideline. Genetics in Medicine 2014;16: 188-200.van Spronsen FJ et al. Key European guidelines for the diagnosis and management of patients with phenylketonuria. Lancet Diabetes Endocrinol 2017; 5: 74356.****P<0.05; Post-hoc comparison of Patients 3&6 vs Patients 4&5 using repeated measures MANOVA/regression analysis

SOURCE: Homology Medicines

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LogicBio Therapeutics Reports Third Quarter 2020 Financial Results and Provides Business Update – GlobeNewswire

November 11th, 2020 11:52 am

- IND for LB-001 in methylmalonic acidemia (MMA) cleared in August 2020, with first patient in Phase 1/2 SUNRISE trial expected to be enrolled in early 2021- Fast Track designation for LB-001 in MMA received in November 2020- First follow-on offering post-IPO closed in October 2020

LEXINGTON, Mass., Nov. 09, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc.(Nasdaq:LOGC) (LogicBio or the Company), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, today reported financial results for the quarter endedSeptember 30, 2020 and provided a business update.

LogicBio has recently marked several important achievements, which could set the stage for exciting news from our company in the quarters to come, said Frederic Chereau, President and CEO. Over the last several months, LogicBio made significant advances in its LB-001 program in methylmalonic acidemia (MMA), with the clearance of the IND and the receipt of Fast Track designation for LB-001 in MMA. Following our $48.3 million follow-on public offering in early October, we believe we are well-positioned financially to deliver on our upcoming milestones. Mr. Chereau continued, The Phase 1/2 SUNRISE trial is designed to treat MMA patients at a young age when gene editing could potentially make a meaningful, life-long difference. We continue to anticipate the enrollment of our first patient in the SUNRISE trial in early 2021, and we eagerly look forward to updating both the clinical community and investors of our progress as 2021 unfolds. Mr. Chereau concluded by saying, In addition to our exciting clinical program, we have extended our collaboration with the Childrens Medical Research Institute of Australia to continue to develop our Next Generation Capsid platform, which has already yielded novel liver-tropic capsids that we believe are superior to ones that are currently used in the clinic. We also anticipate sharing further data on our novel capsids in early 2021.

Anticipated LogicBio Milestones for 2021:

LB-001 for MMA

Pipeline

Third Quarter 2020 Financial Results

Three Months Ended September 30, 2020 and 2019

About LogicBio Therapeutics

LogicBio Therapeuticsis dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms. LogicBios proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is expected to begin in early 2021. In addition, LogicBio has a collaboration withTakedato research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.

LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples. Top-tier capsid candidates from this effort demonstrated significant improvements over benchmark AAVs currently in clinical development. LogicBio is developing these highly potent vectors for internal development candidates and potentially for business development collaborations.

LogicBio is headquartered inLexington, Mass. For more information, please visitwww.logicbio.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the federal securities laws, including those related to the Companys plans to initiate, advance and complete its planned Phase 1/2 SUNRISE clinical trial of LB-001 in MMA and the potential benefits to patients of LB-001; the timing, progress and results of the Companys research and development activities, including those related to the GeneRide technology platform and Next Generation Capsid Program; its plans for LB-301 in Crigler-Najjar; and the sufficiency of our cash on hand to fund our operating expenses and capital expenditures. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. In particular, the impact of the COVID-19 pandemic on the Companys ability to progress with its research, development, manufacturing and regulatory efforts, including the Companys plans to initiate, advance and complete its Phase 1/2 clinical trial for LB-001 in MMA, and the value of and market for the Companys common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements inthe United Statesand in other countries, and the effectiveness of actions taken globally to contain and treat the disease. These risks are discussed in the Companys filings with theU.S. Securities and Exchange Commission(SEC), including, without limitation, the Companys Annual Report on Form 10-K filed onMarch 16, 2020with theSEC, the Companys Quarterly Report on Form 10-Q filed onMay 11, 2020, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with theSEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

LogicBio Therapeutics, Inc.Condensed Consolidated Balance Sheets(In Thousands)(Unaudited)

LogicBio Therapeutics, Inc.CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS(In thousands, except share and per share amounts)(unaudited)

Contact:

Matthias Jaffe Chief Financial Officer mjaffe@logicbio.com (617) 245-0399

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Prostate Cancer Foundation, Dana-Farber Cancer Institute and Oregon Health & Science University Partner to Adv – Prostate Cancer Foundation

November 11th, 2020 11:52 am

LOS ANGELES, Calif.,November 11, 2020 In recognition of Veterans Day, the Prostate Cancer Foundation (PCF) today announced its new $5 million commitment to launch two new Centers of Excellence in collaboration with the VA Boston Healthcare System (VABHS), Dana-Farber Cancer Institute, the VA Portland Health Care System (VAPORHCS) and Oregon Health & Science University (OHSU) to deliver best-in-class precision oncology treatments to Veterans with prostate cancer.

PCF is committed to continuing to expand our partnership with the VA to advance cutting-edge research and care for Veterans with prostate cancer, said Jonathan W. Simons, MD, PCF president and CEO. Partnering with Dana-Farber and Oregon Health & Science University brings top expertise to our precision oncology efforts serving our nations Veterans. It is our duty to ensure that no Veteran is left behind when a medical breakthrough comes to the clinic.

More than 15,000 men in the Veterans Affairs (VA) health system are newly diagnosed with prostate cancer each year, making it the most frequently diagnosed cancer among Veterans. One in nine men and one in six African American men are diagnosed with prostate cancer each year, and four million men in the U.S. are living with the disease.

Precision medicine is individualized, based on gene sequencing a patients tumor, allowing for custom-tailored treatment that targets an individuals cancer by its unique biology and genetic signature. It is the key to ending deaths from prostate cancer. More than half the funds the PCF has committed have been used to launch its collaborative COE network at the forefront of precision oncology for prostate cancer. The PCF has also dedicated funding to the research of numerous VA physician scientists. The platform the PCF and VA have created is being used to build centers of excellence for other cancers, as well as to launch clinical studies in the fight against COVID-19.

With the addition of the new centers, the PCF has established 12 COEs to date, executing the ambitious mission of improving patient care for U.S. Veterans with prostate cancer. Located inBoston, MA, and Portland, OR, these new COEs join 10 other established Centers in cities across the U.S. in delivering advanced precision oncology treatments to save the lives of Veterans battling prostate cancer. The other centers are based inPhiladelphia, PA,Washington, DC,Durham, NC, Tampa Bay, FL, Seattle, WA,Chicago, IL,Bronx, NY,Los Angeles, CA,Manhattan, NY,andAnn Arbor, MI.

We are proud to partner with the PCF and the VA to launch this first PCF-VA Center of Excellence in New England, said Dana-Farber President and CEO Laurie H. Glimcher, MD. Our partnership will create better access to clinical trials and genomics for Veterans across all VA systems, and deliver the best possible care for Veterans diagnosed with prostate cancer.

The PCFs Veterans Health Initiative, which was established in 2016, is committed to investing$50 millionto deliver innovative, best-in-class prostate cancer care to Veterans, which includes expanding genomic data banking to provide improved prostate cancer treatment, greater access to clinical trials, and resources to develop better precision oncology care.

This award will make it possible for us to reach out to veterans throughout Oregon and Southwest Washington to offer genetic testing and sequencing for all patients with metastatic prostate cancer, said Julie Graff, M.D., Section Chief of Hematology/Oncology, VA Portland Health Care System and Associate Professor, Hematology & Medical Oncology, OHSU Knight Cancer Institute. Knowing more about each patients specific mutation(s) gives us the knowledge we need to explore more treatment and clinical trial options. Ultimately, we want Veterans to know they have access to leading treatment and clinical trials through the VA Portland Health Care System.

The Portland and Boston COEs were established with funding made possible through the Blavatnik Family Foundation and Rob and Cindy Citrone.

Members of the Blavatnik Family Foundation Precision Oncology

Center of Excellence (COE) at VABHS-Dana-Farber Boston include: Chong-xian Pan, MD, PhD, MS, Medicine faculty, Brigham and Womens Hospital, Harvard Medical School, staff physician, hematology/oncology, VABHS; Himisha Beltran, MD, Associate Professor of Medicine, Lank Center for Genitourinary Oncology Division of Molecular and Cellular Oncology, Dana-Farber, Director of Translational Research-Medical Oncology, Dana-Farber.

Members of the PCF Precision Oncology Center of Excellence (COE) at VAPORHCS-OHSU Portland team include: Julie Graff, MD, oncology (VAPORHCS, OHSU); Tomasz Beer, MD, oncology (OHSU, VAPORHCS); Jeremy Cetnar, MD, oncology (VAPORHCS, OHSU); Ryan Kopp, MD, urology (VAPORHCS); Mark Garzotto, MD, urology (VAPORHCS, OHSU); Amy Moran, PhD, immunology (OHSU); Reid Thompson, MD PhD, radiation oncology (VAPORHCS, OHSU); Rajan Kulkarni, MD PhD, dermatology (VAPORHCS, OHSU); Dean Fong, DO, pathology (VAPORHCS).

To learn more about clinical trials offered by the PCF-VA Centers of Excellence, Veterans should call (206)-277-3621. Information is also available atwww.PCF.org/veterans.

About the Prostate Cancer Foundation

The Prostate Cancer Foundation (PCF) is the worlds leading philanthropic organization dedicated to funding life-saving prostate cancer research. Founded in 1993 by Mike Milken, PCF has raised more than $830 million in support of cutting-edge research by more than 2,200 research projects at 220 leading cancer centers in 22 countries around the world. Thanks in part to PCFs commitment to ending death and suffering from prostate cancer, the death rate is down by 52% and countless more men are alive today as a result. The Prostate Cancer Foundation research now impacts more than 70 forms of human cancer by focusing onimmunotherapy, the microbiome, and food as medicine. Learn more at http://www.pcf.org.

Media Contact: Staci Vernick Prostate Cancer Foundationsvernick@pcf.org610-812-6092

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The Prime Editing market to witness elegant elevation in the next decade – Eurowire

November 11th, 2020 11:52 am

Prime editing is the gene-editing method that can insert, delete and do base swapping accurately. Prime editing also termed as genetic word processor precisely select the target DNA and replace genetic code. Targeting 75,000 different mutations and correcting 89% of genetic defects will drive the demand for prime editing. In 2017, the first gene editing in the human body was attempted. Gene editing in a patient with Hunters syndrome was tested for safety and concluded reliable shreds of evidence. Superior target flexibility and editing precision with minimal errors make Prime editing first preference over the other conventional technique such as CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats). Application of prime editing in reversing Genetic disease will be a milestone in gene editing.

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Increasing prevalence of genetic disease creates a huge opportunity for prime editing market. Successful preliminary results with a genetic disease like Tay Sachs and Sickle cell anaemia will drive the prime editing market. Technological advancements providing minimal error with this technique will fuel the growth of prime editing. Decreased cost of DNA sequencing will propel prime editing market for research and commercialization. Arising ethical and safety concerns will make prime editing highly regulated sector. This may limit the scope and can restraint the growing market. Detrimental effect on Genetic diversity due to genetic engineering in one way may limit the market scope.

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The global Prime Editing market is classified on the basis of application and end user:

Based on application, Prime Editing Market is segmented into following:

Based on end user, Prime Editing Market is segmented into following:

To receive extensive list of important regions, Request TOC here @https://www.persistencemarketresearch.com/toc/31691

Prime Editing is the most recent invention has created a buzz in the market. Firms accessing conventional genome engineering technologies have rolled plans of transitioning to this new technology. The restructuring by the firms is either by building upon the technological capabilities or by merging or acquiring the firms which hold expertise in prime editing. Inscripta, one of the most innovative company has launched the worlds first benchtop platform for digital genome engineering. Inscriptas Onyx device that was launched in October 2019, will enable genome editing at an unprecedented scale and cheaper rate. In 2019, Beam Therapeutics collaborated with a premium start-up in prime editing segment Prime Medicine for Prime Editing Technology. Beam therapeutics holds expertise in precision genetic medicine using base editing technology. The market consolidation activities my giants depict that genome editing will be the largest revenue-generating segment for prime editing market.

North America will drive the market for Prime Editing due to high prevalence of genetic disease and technological advancement in the U.S. and Canada. One in every 27 Jews, is carrying Tay Sachs disease gene. After North America, Europe is leading in patient pool for genetic diseases such Hemophilia and Cystic fibrosis. The genetic disease pool will drive the adoption for Prime editing treatments in this region. Asia-Pacific will remain at steady growth for Prime Editing market due less disease prevalence and focus on other therapies. Latin America and Middle East and Africa region will boost the market owing to the disease prevalence.

Examples of some of the market participants in Prime Editing market identified across the value chain Beam Therapeutics Inc., Precision BioSciences, Inscripta, Inc, Horizon Discovery Ltd., Sangamo Therapeutics, Inc., CRISPR Therapeutics., Intellia Therapeutics, Inc.

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New Data Show Importance of Vectra Testing and Biomarkers in Predicting Risk for Cardiovascular Events in Patients with Rheumatoid Arthritis -…

November 11th, 2020 11:52 am

SALT LAKE CITY, Nov. 11, 2020 (GLOBE NEWSWIRE) -- At the annual meeting of the American College of Rheumatology (ACR), Myriad Genetics, Inc. (NASDAQ: MYGN), a leader in molecular diagnostics and precision medicine, shared new data further demonstrating that Vectra testing and three additional biomarkers, combined with traditional risk factors, can predict the risk of cardiovascular (CV) events in patients with rheumatoid arthritis (RA). Also, presented at the meeting was a study showing that Vectra is a significant predictor for joint damage, and is a better predictor than several tested subjective measures. Vectra is an advanced blood test that objectively measures inflammation caused by RA.

Due to inflammatory processes, patients with RA have approximately 50% greater risk for cardiovascular disease (CVD), the leading cause of mortality among patients with RA, which accounts for 30-40% of deaths. said Elena Hitraya, M.D., Ph.D., rheumatologist and chief medical officer at Myriad Autoimmune. The data shows that a newly developed Multi-Biomarker Based CVD Risk Score can accurately predict the risk of major cardiovascular events over the next three years across various subgroups of RA patients. Knowing a patients future risk of potential joint damage and cardiovascular events, clinicians can make more informed treatment decisions with the goal of achieving better health outcomes.

Vectra Posters at ACRTitle: External Validation of a Multi-Biomarker-Based Cardiovascular Disease Risk Prediction Score for Rheumatoid Arthritis PatientsVirtual Poster Location: https://acrabstracts.org/abstract/external-validation-of-a-multi-biomarker-based-cardiovascular-disease-risk-prediction-score-for-rheumatoid-arthritis-patients/ This validation study assessed the performance of the Multi-Biomarker-Based Cardiovascular Disease Risk Prediction Score in a non-Medicare patient population. The goal of the study was to validate the risk score in a cohort (N=44,379) with median age of 54 (46-60) years that was younger than, and independent of, the Medicare cohort used for test development. The study found that the Vectra-based CVD risk score (mean 3.3, IQR 2.8-3.8) was a significant predictor of CVD risk, with hazard ratio (HR) = 3.99 (95% CI: 3.52-4.51, p=4.410-95); i.e., for every 1-unit increase in the score, the CVD event rate was ~4 times higher.

Title: Performance of the MBDA-based CVD risk score in RA patient groups of clinical interestVirtual Poster Location: https://acrabstracts.org/abstract/performance-of-the-mbda-based-cvd-risk-score-in-ra-patient-groups-of-clinical-interest/ The purpose of this study was to evaluate the ability of a risk score that combines Vectra, TNF-R1, MMP-3, leptin, age and traditional risk factors (diabetes, hypertension, smoking, history of CVD) to predict 3-year risk for myocardial infarction (MI), stroke, or fatal CVD for RA patients in subgroups of interest. The study found that, in a cohort of 10,275 Medicare patients, with high prevalence of comorbidities, such as diabetes (40%) and hypertension (79%), the Vectra-based CVD risk prediction score had good accuracy overall and in subgroups based on level of a Vectra score, sex, and statin use.

Title: Comparison of MBDA Score, Patient Global Assessment, and Evaluator Global Assessment for Predicting Risk of Radiographic ProgressionVirtual Poster Location: https://acrabstracts.org/abstract/comparison-of-mbda-score-patient-global-assessment-and-evaluator-global-assessment-for-predicting-risk-of-radiographic-progression/ In a cohort of 766 patients from one registry and two clinical trials, this study compared Vectra, patient global assessment (PGA), and evaluator global assessment (EGA) in terms of their abilities to predict risk of radiographic progression (RP) (i.e. new joint damage). The study found Vectra significantly predicted risk for RP (Figure 1A in the poster), with univariate OR=1.53, p=5.3x10-8. In contrast, neither PGA nor EGA predicted RP (p=0.38 and 0.47, respectively). Vectra predicted RP regardless of whether PGA and EGA were concordant or discordant.

RA affects more than one million people in the United States. Lost productivity associated with RA is substantial, with approximately 20-70% of individuals working at the time of their RA diagnosis being disabled after seven to 10 years. RA is an inflammatory autoimmune disease that attacks a patients joints and often affects other organ systems, contributing to increased disability, significant morbidity, increased mortality and financial burden. The risk of RP, defined as change in total Sharp score >5 units per year, is a function of Vectra score. Increased risk of RP means greater irreversible joint damage.

Three out of four rheumatologists have used Vectra and have ordered more than one million tests for their RA patients. The ACR includes Multi-Biomarker Disease Activity Score (Vectra) as a disease activity measure that meets the minimum standard for regular use for patients with rheumatoid arthritis. Those recommendations were published in the journal Arthritis Care & Research.

About VectraVectra is a multi-biomarker molecular blood test that provides an objective and personalized measure of inflammatory disease activity in patients with rheumatoid arthritis. Vectra demonstrates unsurpassed ability to predict radiographic progression and can help guide medical management decisions to improve patient outcomes. Vectra testing is performed at a state-of-the-art CLIA (Clinical Laboratory Improvement Amendments) facility. Test results are reported to the physician five to seven days from shipping of the specimen. Physicians can receive test results by fax or the private provider web portal, VectraViewTM. For more information on Vectra, please visit: VectraScore.com.

About Myriad GeneticsMyriad Genetics Inc., is a leading personalized medicine company dedicated to being a trusted advisor transforming patient lives worldwide with pioneering molecular diagnostics. Myriad discovers and commercializes molecular diagnostic tests that: determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across six major medical specialties where molecular diagnostics can significantly improve patient care and lower healthcare costs. Myriad is focused on three strategic imperatives: transitioning and expanding its hereditary cancer testing markets, diversifying its product portfolio through the introduction of new products and increasing the revenue contribution from international markets. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.

Myriad, the Myriad logo, BART, BRACAnalysis, Colaris, Colaris AP, myPath, myRisk, Myriad myRisk, myRisk Hereditary Cancer, myChoice, myPlan, BRACAnalysis CDx, Tumor BRACAnalysis CDx, myChoice CDx, Vectra, Prequel, Foresight, GeneSight, riskScore and Prolaris are trademarks or registered trademarks of Myriad Genetics, Inc. or its wholly owned subsidiaries in the United States and foreign countries. MYGN-F, MYGN-G.

Safe Harbor StatementThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements related to Vectra-related posters at the annual meeting of the American College of Rheumatology (ACR); and the Companys strategic directives under the caption "About Myriad Genetics." These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by forward-looking statements. These risks and uncertainties include, but are not limited to: uncertainties associated with COVID-19, including its possible effects on our operations and the demand for our products and services; our ability to efficiently and flexibly manage our business amid uncertainties related to COVID-19; the risk that sales and profit margins of our molecular diagnostic tests and pharmaceutical and clinical services may decline; risks related to our ability to transition from our existing product portfolio to our new tests, including unexpected costs and delays; risks related to decisions or changes in governmental or private insurers reimbursement levels for our tests or our ability to obtain reimbursement for our new tests at comparable levels to our existing tests; risks related to increased competition and the development of new competing tests and services; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and pharmaceutical and clinical services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and pharmaceutical and clinical services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and pharmaceutical and clinical services and any future tests and services are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with operating our laboratory testing facilities and our healthcare clinic; risks related to public concern over genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of the healthcare system or healthcare payment systems; risks related to our ability to obtain new corporate collaborations or licenses and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we license or acquire; risks related to our projections about our business, results of operations and financial condition; risks related to the potential market opportunity for our products and services; the risk that we or our licensors may be unable to protect or that third parties will infringe the proprietary technologies underlying our tests; the risk of patent-infringement claims or challenges to the validity of our patents or other intellectual property; risks related to changes in intellectual property laws covering our molecular diagnostic tests and pharmaceutical and clinical services and patents or enforcement in the United States and foreign countries, such as the Supreme Court decisions in Mayo Collab. Servs. v. Prometheus Labs., Inc., 566 U.S. 66 (2012), Assn for Molecular Pathology v. Myriad Genetics, Inc., 569 U.S. 576 (2013), and Alice Corp. v. CLS Bank Intl, 573 U.S. 208 (2014); risks of new, changing and competitive technologies and regulations in the United States and internationally; the risk that we may be unable to comply with financial operating covenants under our credit or lending agreements; the risk that we will be unable to pay, when due, amounts due under our credit or lending agreements; and other factors discussed under the heading "Risk Factors" contained in Item 1A of our most recent Annual Report on Form 10-K for the fiscal year ended June 30, 2020, which has been filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.

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New Gene Therapy Approach Prevents Toxicity Tied to AAV Vectors – Clinical OMICs News

November 11th, 2020 11:52 am

A new modified gene therapy approach avoids a toxicity seen in some nonhuman primate studies using adeno-associated viral (AAV) vectors to treat neurological disorders. Penn Medicine researchers developed the technique, which uses a modified transgene with a microRNA target designed to reduce the level of transgene expression in the dorsal root ganglia (DRG). They report that the alteration lowered transgene expression in the DRG by more than 80 percent and reduced toxicity in primates, which is considered a major hurdle to gene therapy for central nervous system conditions. The Penn groups findings were reported online this week in Science Translational Medicine.

We believe it is a safe, straightforward way to ameliorate the safety of AAV therapy for the central nervous system, said first author Juliette Hordeaux, DVM, PhD, senior director of Translational Research in Penns Gene Therapy Program. This approach could be used to design other gene therapy vectors to repress transgene expression in the cell types that are affected by the toxicity and not others, which is critical, because you need the expression everywhere else to effectively treat the disorder.

Transgene overexpression in the DRG is thought to cause axonal degeneration in spinal cord tracts and peripheral nerves. Although this side effect has only been seen in primates so far, it represents a major technical challenge to the field. There are already several gene therapy trials ongoing in CNS conditions such as spinal muscular atrophy and Parkinsons, and many more potential CNS-related targets for gene therapy exist.

This side effect was first seen in nonhuman primate studies using AVV vectors to deliver corrected genes via the spinal cord fluid and intravenously. Those studies reported problems of axonal degeneration in some tracts of the spinal cord and peripheral nerves. The cause was traced back to the DRG, a cluster of neural cells on the outside of the spinal cord responsible for transmission of sensory messages.

For their studies, the Penn researchers first documented DRG toxicity in nonhuman primates, then they devised a way to overcome it. Though its asymptomatic in primates, the damage is clear under close study of CNS histopathology. Its already established that damage to the DRG in humans can lead to the breakdown of axons responsible for delivering impulses from nerves to the brain. Numbness and weakness in limbs, among other side effects, follow suit.This observed toxicity prompted the U.S. Food and Drug Administration to recently place a partial hold on human trials administering a gene therapy vector into the spinal cord to treat spinal muscular atrophy, a genetic disease that severely weakens muscles and causes problems with movement.

To overcome this toxicity, the Penn researchers injected vectors with and without a microRNA target, first in mice and then primates. microRNA regulates gene expression and makes for an ideal target in the cells. They chose microRNA-183 because it is largely restricted to the neurons in the DRG.

They found that the unmodified AAV vectors resulted in robust delivery of the new gene into target tissue and toxicity in DRG neurons. Vectors with the miRNA target, on the other hand, reduced transgene expression significantly, as well as the toxicity of DRG neurons, without affecting transduction elsewhere in the primates brain, based on histological analyses of the specimens up to 90 days later. The researchers also examined whether the DRG toxicity was caused by an immune response. They carried out experiments that showed immunosuppressants and steroids were unsuccessful at alleviating the toxicity.

According to the authors, toxicity of DRGs is likely to occur in any gene therapy that relies on high doses of a vector or direct delivery of a vector into the spinal cord fluid.

We were concerned about the DRG pathology that was observed in most of our [non human primate] NHP studies, Wilson said. This modified vector shows great promise to reduce DRG toxicity and should facilitate the development of safer AAV-based gene therapies for many CNS diseases.

Originally posted here:
New Gene Therapy Approach Prevents Toxicity Tied to AAV Vectors - Clinical OMICs News

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Verve Therapeutics Announces Presentations on its Gene Editing Approach to Develop Transformative Medicines for Coronary Heart Disease at the American…

November 11th, 2020 11:52 am

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Verve Therapeutics, a biotech company pioneering gene editing medicines to treat cardiovascular disease, today announced two presentations highlighting gene editing as a potentially transformative therapeutic approach at the American Heart Association (AHA) Scientific Sessions 2020, taking place virtually November 13-17, 2020. Verve is developing one-time gene editing medicines to safely and precisely turn off a gene in the liver to permanently lower LDL cholesterol or triglyceride levels and thereby treat adults with coronary heart disease, the leading cause of death worldwide.

Sekar Kathiresan, M.D., co-founder and chief executive officer of Verve Therapeutics, will present an overview of Verves approach to develop medicines that safely edit the adult human genome and mimic naturally occurring protective gene variants to confer resistance to coronary heart disease. He will also highlight preclinical data in non-human primates demonstrating the successful use of base editing to knock out PCSK9 or ANGPTL3 in the liver and substantially reduce blood levels of LDL cholesterol or triglycerides. Coronary heart disease occurs when cholesterol-laden plaque builds up in the arteries of the heart, which can restrict blood flow and lead to a heart attack.

Human genetics has provided the blueprint for how to treat coronary heart disease by revealing healthy individuals who have protective genetic mutations that confer resistance to heart attack, said Dr. Kathiresan. With advances in gene editing, we now have the ability to edit the adult genome to treat this disease at a genetic level, opening the door to create a potential once-and-done treatment for patients. We have made significant progress toward our goal of developing gene editing medicines for adults with coronary heart disease and plan to select our lead program by the end of this year. We look forward to presenting an overview of our therapeutic approach and proof-of-concept data in non-human primates utilizing base editing at the upcoming AHA annual meeting.

Dr. Kathiresans presentations will be available for OnDemand viewing on the AHA Scientific Sessions 2020 virtual platform throughout the meeting. Presentation details are as follows:

Presentation Title: Manipulating the Genome for TherapySession Title: Best Science in Cardiovascular Genetics and GenomicsSession Date & Time: Available OnDemand beginning Friday, November 13, 2020 at 9:00 a.m. CT through Tuesday, November 17, 2020 at 8:30 p.m. CTSession Number: GE.CVS.639

Presentation Title: Coronary Heart Disease Prevention in 2050: Reading the Genome for Risk and Rewriting It for HealthSession Title: 20:20 Vision for 2050: Predicting the Future of Cardiovascular Health and MedicineSession Date & Time: Available OnDemand beginning Friday, November 13, 2020 at 9:00 a.m. CT through Tuesday, November 17, 2020 at 8:30 p.m. CTSession Number: EP.HL.729

About Verve Therapeutics

Verve Therapeutics is a biotechnology company created with a singular focus: to protect the world from heart disease. The company brings together human genetics analysis and gene editing two of the biggest breakthroughs in 21st century biomedicine to develop transformative therapies for coronary heart disease. Verve is developing medicines, administered once in life, to safely edit the genome of adults and mimic naturally occurring gene variants to permanently lower LDL cholesterol and triglyceride levels and thereby treat coronary heart disease. Founded by world-leading experts in cardiovascular medicine, human genetics and gene editing, Verve is backed by a top-tier syndicate of investors, including GV (formerly Google Ventures), ARCH Venture Partners, F-Prime Capital, Biomatics Capital, Wellington Management, Casdin Capital, and Partners Innovation Fund. Verve is headquartered in Cambridge, Massachusetts. For more information, visit http://www.VerveTx.com.

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Verve Therapeutics Announces Presentations on its Gene Editing Approach to Develop Transformative Medicines for Coronary Heart Disease at the American...

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