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Archive for the ‘Gene therapy’ Category

Taysha Gene Therapies Expands Leadership Team to Deepen Manufacturing and Communications Capabilities – Business Wire

Wednesday, December 30th, 2020

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced new additions to its leadership team with the appointments of Greg Gara as Senior Vice President of Manufacturing and Kimberly Lee, D.O., as Senior Vice President of Corporate Communications and Investor Relations.

We are excited to welcome Greg and Kim to Tayshas leadership team, said RA Session II, President, Founder and CEO of Taysha. They each bring significant domain experience and their contributions will be invaluable as we continue our mission of eradicating monogenic CNS diseases. Gregs technical expertise in AAV gene therapy manufacturing along with his proven success in constructing several cGMP gene therapy facilities and Kims deep experience across capital markets and corporate communications will add tremendous value to the team. Importantly, both share our unrelenting, patient-first focus and passion for bringing new cures to life.

Mr. Gara has over 25 years of experience in designing, constructing, and starting up large- and small-scale manufacturing facilities for biotechnology companies globally. Prior to joining Taysha, he served as Vice President of Pharmaceutical Engineering at Sarepta, where he led and managed manufacturing operations for all gene therapy products. Before Sarepta, he served as Vice President of Technical Operations and Engineering at AveXis, a Novartis company, where he led the design, construction, and startup of the Libertyville facility and the new facilities in Research Triangle Park and Colorado. Mr. Gara also led the team for the facility expansion in North Carolina and the renovation of the Colorado site. Prior to AveXis, he led the facilities and engineering organization at Hospira prior to the companys acquisition by Pfizer. Before joining Hospira, he spent 15 years at Amgen, holding positions of increasing responsibility, and was part of the Cork, Ireland, construction project. Mr. Gara received a B.A. in Biology and Environmental Science from Augustana College.

Tayshas dedication to the development and commercialization of potentially transformative gene therapy treatments and its innovative and pioneering spirit is truly inspiring and I am excited to contribute in a meaningful way, said Mr. Gara. I look forward to playing an instrumental role in the companys growth and expansion of its manufacturing capabilities.

Dr. Lee joins Taysha with over 20 years of capital markets, strategic corporate finance, and communications experience from prior roles as a biotech equity research analyst on Wall Street and corporate strategy, communications, and investor relations professional. She most recently served as Head of Corporate Strategy and Investor Relations at Lexicon Pharmaceuticals and previously as Vice President of Corporate Strategy, Corporate Communications and Investor Relations at Raptor Pharmaceuticals until its acquisition by Horizon Pharma. Prior to joining Raptor, Dr. Lee was a biotechnology sell-side analyst at investment banks, including Jefferies and Wedbush Securities, covering biotechnology companies across all market capitalizations, multiple therapeutic areas, and modalities. Dr. Lee received a B.S. in Biological Sciences from Stanford University and a D.O. from Kirksville College of Osteopathic Medicine.

In less than one year, Taysha has made extraordinary progress in developing and funding its elegant platform and rapidly advancing its product candidates for the betterment of patients and I am thrilled and grateful to be a part of this journey, said Dr. Lee. I am eager to learn from and work alongside this team of gene therapy experts at this exciting stage of our companys lifecycle and I look forward to making lasting contributions.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at

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Taysha Gene Therapies Expands Leadership Team to Deepen Manufacturing and Communications Capabilities - Business Wire


A Look Back at the Past Decade of European… –

Wednesday, December 30th, 2020

As we prepare to enter a new decade, we look back on the major milestones and blunders within European biotech over the last 10 years.

Over the last decade, we have seen many biotech breakthroughs to come from Europe, including first-in-class therapies for cancer, the first approved in vivo gene therapy, as well as notable efforts to combat Covid-19. Some other areas have proven to fall behind expectations, such as Alzheimers or microbiome research, and will need a push over the next decade. Lets have a look back at what the biotech industry has accomplished in the decade were now leaving behind.

Few would disagree that this has been a game-changing decade within oncology. Weve seen the arrival of checkpoint inhibitor drugs, the first oncolytic viral therapy, and the approval of the first CAR-T cell therapies.

For Alexandra Bause, who leads a venture creation program at the investment firm Apollo Health Ventures, immuno-oncology was one of the most exciting things to happen during the last decade. Its not just one cancer and one drug for that specific cancer anymore, we can now activate, reactivate or replenish the bodys own immune response and we can potentially target malignant cells throughout the whole body.

This decade has witnessed the advent and establishment of checkpoint inhibitor drugs. These immunotherapies consist of therapeutic antibodies that block immune checkpoints proteins on the surface of immune cells that tumors use to evade the immune system.

The first checkpoint inhibitor, ipilimumab, was approved in 2011. Since then, six other checkpoint inhibitor drugs belonging to a new generation that blocks the PD-1/PD-L1 immune checkpoint have been approved. These drugs have made a big difference for a certain percentage of patients with difficult-to-treat forms of cancer.

Many good things came to cancer patients in the last decade, said Martin Bonde, CEO of Inthera Biosciences in Switzerland. For instance, we are now much better at treating multiple myeloma and malignant melanoma than we were 10 years ago. At the start of the decade, malignant melanoma had a 5% 5-year survival rate, now its over 50 % thanks to drugs like Opdivo (nivolumab) and Keytruda (pembrolizumab).

Since the initial approval of Opdivo and Keytruda as treatments for advanced melanoma in 2015, their indications have been expanded to include a range of blood and solid cancers. They are also used in combination with other checkpoint inhibitors as well as conventional chemotherapeutic agents. Sales-wise, Keytruda takes the lead as one of the worlds best-selling drugs.

The year 2015 also brought the approval of Imlygic, the first viral therapy for cancer in the Western world. The treatment acts by injecting melanoma tumors with a virus that infects and destroys cancerous cells. While Imlygic hasnt proved to be a blockbuster, viral therapies for cancer going forward could find a niche when combined with other cancer treatments such as checkpoint inhibitors.

In 2017, we saw the US approval of Kymriah, the worlds first CAR-T cell therapy. This treatment consists of engineering the patients own immune cells to make them better at identifying and attacking cancer cells. That same year, a second CAR-T therapy, Yescarta, was approved and a third one, Tecartus, received approval this year.

Despite concerns regarding severe side effects and prohibitive pricing, CAR-T cell therapy has made a big difference for patients with blood cancer that had not responded to other treatments, with remission rates above 90% in some cases. There are now over 1,000 clinical trials testing different forms of CAR-T technology aiming to improve its efficacy and safety and to extend its use to other forms of cancer, such as solid tumors.

The future of cancer treatment seems to be going in the direction of combining different approaches and selecting the most suitable for each patient. According to Bonde, combination therapy will continue to be refined with genomics technology.

I think its difficult to see combination therapy going away anytime soon because cancer is so complex and still difficult to treat, so we need to attack it from multiple angles. I think we will continue to see the search for new mechanisms of action, and research will help us to understand how we can best tackle a particular cancer in relation to its genetic makeup.

The last decade brought gene therapy to the market, offering a one-off treatment for patients suffering from genetic disorders. In Europe, the first gene therapy, Glybera, was approved in 2012. Although the treatment was withdrawn after a commercial failure, it set a precedent; there are now a total of 11 cell and gene therapies approved in the EU.

In 2018, the EMA approved Luxturna, a gene therapy developed by Novartis to treat blindness caused by a genetic mutation. This made Europe the first to approve an in vivo gene therapy, in which the genetic modification happens directly within the body rather than in cells extracted from the patient.

Despite these breakthroughs, the number of approved gene therapies in Europe is very modest compared to the over 1,200 gene therapy trials taking place across Europe. Meaning there is still a lot of space to grow.

Today we can get the entire human genome sequence in 24 hours for $500. This has opened up the possibility to design drugs using genetic information this is just starting and will be important in the next decade in gene therapy for cancer and elsewhere, said Bonde.

The gene therapies approved to date have mostly consisted of replacing a faulty gene with a functional copy delivered on viruses. Rapid technical advances within gene-editing technologies such as CRISPR-Cas9 are now making it possible to make precise edits to the genome, and many such therapies have entered clinical trials in Europe.

The orphan disease space has become much more crowded in recent years, with companies trying to target specific gene mutations behind a given rare disease, many of which incorporate gene therapy or gene-editing technologies, while others target downstream pathways with small molecules, said Bause.

The last decade has primarily been about tool-building within the gene therapy area and with the advent of CRISPR-Cas9 and related technologies, we are really only at the beginning of this era.

A therapeutic area that has seen huge investment, particularly in the second half of the last decade, is aging. Companies in this field seek to tackle aging-related diseases.

Why is this so important? According to Alexandra Bause, a wide range of diseases can be attributed to aging. In our 20s, 30s, and 40s, most of us are healthy, and then disease may creep up from the age of 50 years. The underlying aging process is causing the majority of known diseases that make up the major markets, such as Alzheimers, cancer, heart disease, chronic kidney disease, type 2 diabetes, metabolic disorders, and more.

Right now, much of the focus is on reverting age-related physiological damage. As research sheds light on the mechanisms underlying aging, many strategies to combat aging are being explored. Senolytics are a prominent example. These are small molecules that can simultaneously eliminate aged cells and promote tissue rejuvenation.

In recent years weve seen the creation of companies developing senolytics, including Senolytx in Barcelona and Velabs Therapeutics in Helsinki. However, a senolytic candidate developed by the US company Unity Biotechnology failed a key phase II trial in osteoarthritis earlier this year and the company is still suffering the fallout. Evidently, a lot more progress is still needed in the senolytics field before it can produce any marketed drugs.

Other strategies include drug repurposing, stem cells, and genomics. A major study called TAME is looking at whether the drug metformin can extend longevity and delay the onset of age-related chronic diseases such as heart disease, cancer, and dementia.

Metformin is widely used to treat type 2 diabetes but it also seems to have properties that may reduce Alzheimers and cancer risk. The TAME study will evaluate whether metformin doesnt just increase healthy lifespan by diminishing the risk of Alzheimers and cancer, but actually affects cellular pathways of aging as well. This is an important drug and an important study because it demonstrates how drugs can be repurposed to directly impact aging, said Greg Bailey, CEO of anti-aging company Juvenescence.

The organizers of the TAME trial also plan to launch a study into what biomarkers can best assess biological age, since its currently hard to measure how much a drug has slowed the aging process. Going forward, these trials will be a huge help to those working within aging, which is not currently seen as an official disease by the EMA or FDA. This means that companies are limited to targeting a specific age-related disease each time they want to test new treatments in clinical trials.

I think genetic modification of cellular pathways and epigenetics will play an enormous role. There is incredible ongoing work with Yamanaka factors; those transcription factors and gene modifications can reset cells to embryonic stage, potentially erasing the epigenetic changes of aging. Clearly the control of these factors and genes would be hugely transformational, said Bailey.

The field is coming fast and furious. We have the opportunity and ability to turn science fiction into science. In the last 10 years, scientists have truly begun to understand the cellular pathways involved with aging. And when we understand a cellular pathway, we can manipulate it. This was made possible by unlocking the human genome with computational biology, and advances in machine learning have literally opened the floodgates.

Although Covid-19 only appeared at the end of the decade, it has already made a huge impact on the biotech industry. If there is a positive in this crisis, the speed at which the biotech, pharma and research communities have come together in the face of Covid-19 is truly remarkable. It has probably accelerated scientific knowledge by years, said Bailey.

Covid-19 has focused the attention on biotech and healthcare and I believe that biotech is the ultimate superhero. It is the Modernas and the BioNTechs of this world that will kill Covid, said Antoine Papiernik, Managing Partner at life sciences VC firm Sofinnova Partners.

There are currently over 50 Covid-19 vaccines in clinical trials, with 12 of these in late-stage testing. One area in particular that has seen a big push because of the pandemic has been RNA therapeutics. In partnership with Pfizer, BioNTech in Germany has obtained approval in the UK and US for a Covid-19 vaccine, making it the first medicine using messenger RNA technology.

Although European diagnostics and vaccine development have seen a boost in funding this year thanks to Covid-19, funding for infectious diseases in general will still be an uphill battle. Its almost impossible to get funding for infectious diseases nowadays. Why? Because there are few patients in need, and a lot of drugs already out there, and treatment lasts a maximum of 2 weeks. How much can you charge for that compared to a cancer treatment? said Bonde.

There have been some efforts to boost the development of new antibiotics, such as the launch of the Antimicrobial Resistance Action Fund this year. Additionally, the UK began trialling a new payment policy to incentivize antibiotics development in 2019. However, this may still not be enough.

Its a broken business model, and its going to hurt us because an estimated 10 million people will die over the next 10 years or so, from multi-resistant bacteria. But theres no incentive in the market for companies to go after it. I think this should and will end as a state matter. The state will make sure that we have enough options to deal with deadly bacteria.

The last decade saw hundreds of clinical trials for Alzheimers disease, yet not a single drug is able to stop or slow down its progression. Clinical trials are failing one after the other, in most cases because of a lack of convincing therapeutic efficacy when tested in large groups of patients.

For the last decade, research and clinical strategies within the Alzheimers field have largely focused on the beta amyloid protein, which accumulates in the brains of Alzheimers patients years before they experience cognitive symptoms. These findings led to the hypothesis that these beta amyloid plaques were responsible for cognitive decline, but the failure of clinical trials targeting beta amyloid seems to indicate the solution may lay elsewhere. The biggest challenge is that no consensus exists on the underlying mechanisms of the disease, which is now the 6th leading cause of death worldwide.

Poor disease models and an incomplete understanding of the mechanisms of disease are a big part of the problem. Beta amyloid might be more of a biomarker or a symptom, than the mechanism, said Bause.

Companies dont talk to each other enough to share knowledge about what doesnt work. How many amyloid drugs do you have to put into trials before you realize that this isnt working? This is one of the biggest blunders of the last decade from my perspective, added Bailey.

Even for companies following other approaches, the results have been mixed so far. One example is an antibody drug developed by the Swiss company AC Immune and US partner Genentech to tackle Alzheimers disease by blocking a protein called tau. However, this drug proved a dud on a debut phase II trial this year.

However, the French company AB Science provided a glimmer of hope at the end of the decade. Its drug designed to reduce inflammation in the brain reportedly reduced the number of mild Alzheimers cases that progressed to being severe cases in a phase IIb/III trial this month.

Alzheimers isnt the only field to suffer from inadequate animal models. Other notable disease areas that lack translatable animal models include infectious diseases, bacterial sepsis, psychiatric disorders and immunological disorders.

Alexandra Bause points out that the problem with animal studies is not only due to an incomplete understanding of the underlying mechanisms of disease, but also to the fact that there are intrinsic problems with the way animals are tested. Most companies or most research programs are looking at young animals, and theyre artificially making these young animals sick. Then they are giving them the drug to target whatever made them sick, and they recover. But that doesnt mean that an old animal can recover equally.

Other factors that limit the predictive power of animal models are assumptions that animals and humans use the same or highly similar cellular pathways in response to specific diseases, as well as gender-biases and the use of germ-free animals that dont reflect the potential impact of our microbiome on health and disease.

For the next decade, the biotech industry will have to face the challenge of improving animal models or even replacing them with alternatives such as organs on chips or tissue bioprinting.

In the last decade, the study of the microbiome has gained a lot of attention. The human microbiome, which comprises the collection of microorganisms that live in and on our bodies has been linked to almost every disease imaginable. There are currently more than 1,000 clinical trials listed worldwide testing microbiome-related therapies.

This created huge expectations that are taking longer than expected to pan out. Technological advances have resulted in the generation of mountains of data that is often extremely complex and difficult to interpret. We still dont really understand the dynamic complexity of the microbiome or how to manipulate it for therapeutic benefit.

I think theres a long way to go, said Bonde. Im not sure were ever going to fully understand it.

We aggressively looked at a number of microbiome companies and its fascinating but chaotic. We know what we can do with lactobacilli, but what do we do with everything else? You can change one factor, but what does it do to the other billion or trillion entities that constitute the microbiome? said Bailey.

Overall, the biotech industry in Europe has matured over the last decade and strengthened its position in the global market. If the biotech market in Europe was born around 25 years ago, then by now we have learned from the best during our childhood as well as our adolescent years. Today, as an industry, we are now young adults in our prime, ready to forge our own paths, said Antoine Papiernik, Managing Partner at the French life sciences investment firm Sofinnova Partners.

During this pandemic, Europe has demonstrated its strength, resilience, and scientific prowess, he added. Europe has strong scientific and technological output, it has cultivated talent and built highly experienced management teams, and more than ever before, we are capable of funding young biotechs and medtech to success.

The last decade has brought incredible levels of progress to the biotech industry, while also opening up new challenges to tackle in the coming years. Stay tuned for part two next week, where well look in more detail into what the next decade has in store.

A Look Back at the Past Decade of European... -


5 Medical Innovations You Probably Didn’t Notice Happened in 2020 – Healthline

Wednesday, December 30th, 2020

Even in the midst of the COVID-19 pandemic, the medical community made advances throughout 2020. Here are 5 of the years most impactful innovations.

Overall, 2020 has been a tumultuous year. From a health perspective, its been one turned upside down with a deadly global pandemic reorienting how we live our lives and relate to others.

The COVID-19 pandemic has justifiably dominated headlines and attention from media, policymakers, and health officials alike.

While its clearly the defining public health, cultural, economic even political event of the year, the pandemic shouldnt obscure the fact that 2020 was also a time of great medical innovation.

From breakthroughs in oncology, gene therapies, and heart health, to the development of COVID-19 vaccines that are now being administered domestically and around the world, theres a lot that the medical community can be proud of in 2020.

Healthline touched base with leading experts about some of the most impactful medical advances of the year and how they hint at a more hopeful tomorrow.

Almost every expert Healthline interviewed agreed that gene editing was one of the big stories of the year.

In October, Emmanuelle Charpentier and Jennifer A. Doudna were awarded the Nobel Prize in Chemistry for discovering the CRISPR/Cas9 genetic scissors. (Just five other women have won this prize before).

This gene scissor tool is what it sounds like enzymes snip out pieces of DNA to restore them to their normal function, Dr. William Morris, executive medical director of Cleveland Clinic Innovations, told Healthline.

Charpentier and Doudna showed that these genetic tools could be controlled to cut any kind of DNA molecule at a designated location not just distinguish DNA from viruses, as these scissors exist in their natural form.

Essentially, it means we can rewrite the code of life, according to the Nobel Prizes official announcement.

Morris said that this innovation has wide-ranging ramifications for people who have a wide range of genetic conditions.

He cited sickle cell disease, a condition where malformed sickle-shaped red blood cells cause blockages in blood flow, preventing the protein hemoglobin from effectively ferrying needed oxygen through the body.

Morris said these microscopic tools can cut out these genetic errors.

There have only been a handful of drugs to treat these kinds of conditions in the past.

Now, this kind of development allows you to remove the error and replace the [genetic] code, kind of like in your computer or your iPhone if you downloaded a patch for new software to repair an app that always crashes thats what this is, Morris explained.

Its so earth-shatteringly amazing to tell these patients who otherwise faced an entire lifetime of pain and suffering. You can now use the word cure, which is unbelievable to think about, he said.

Olivier Elemento, PhD, director of the Englander Institute for Precision Medicine at Weill Cornell Medicine in New York City, told Healthline that 2020 is the year of the genetic code.

Were really able to use the genetic code in humans and viruses to help humanity in ways we were not equipped to before, in ways we couldnt do before, Elemento said.

He added that gene therapy in general, along with this CRISPR technology, is pretty extraordinary.

This more comprehensive understanding of genetics extends beyond the Nobel Prize.

For instance, Elemento said Weill Cornell Medicine, where he currently works, along with New York-Presbyterian Hospital and Illumina Inc. recently announced an initiative to sequence the genome of thousands of patients.

The more we understand about genetics and gene therapies, the more improved our precision medicine capacity will be opening up the possibilities of creating targeted therapies for all kinds of conditions.

Recently, the American Heart Association released its own list of innovations in medical treatments.

The spotlight includes a new phase 3 study that could change the way hypertrophic cardiomyopathy (when the heart muscle thickens and can stiffen) is treated.

It also highlights new treatments that might change up the first-line treatment for atrial fibrillation (AFib) a new minimally invasive surgery to prevent stroke and a new trial that reveals more treatment might not necessarily mean better treatment for coronary heart disease.

American Heart Association President Dr. Mitchell S.V. Elkind, MS, FAAN, FAHA, wrote in an email to Healthline that all of these advances over the past year reflect connections between seemingly disparate areas of medicine and the fact that we are most successful when we break down the barriers between fields.

In order to tackle an issue as wide ranging as heart disease, for instance, it takes an interdisciplinary, comprehensive approach.

For example, we learned more this year about the unexpected ways in which medicines designed to treat diabetes, the sodium glucose transporter 2 inhibitors, or SGLT2 inhibitors, help patients with heart failure, even those without diabetes, he added.

Elkind also cited our growing understanding of how connections between infectious diseases like the flu and COVID-19 are tied to greater risk for heart disease and stroke.

Often, the most important advances occur when experts from different areas work together in creative ways to solve a difficult problem, he wrote.

When asked if there was one particular heart health innovation that stood out the most to him, Elkind said that what resonated with him was something not tied to fancy medications or groundbreaking research.

An analysis of people from across the U.S. showed that rates of blood pressure control have begun to decline in the U.S., after almost two decades of better control. High blood pressure is one of the most important and easily treated risk factors for stroke and heart disease, and so this backsliding is especially alarming, he added.

He stressed that the study also pointed to the impact that having health insurance has on controlling ones blood pressure.

Those with some form of health insurance had blood pressure control rates of 4354 percent, while for those without insurance, it was only 24 percent, Elkind explained.

Improving access to quality care is one of the best ways we have to improve health, and that is where we at the American Heart Association will be placing our efforts in the coming years, he said.

Benjamin Neel, MD, PhD, director of the Perlmutter Cancer Center at NYU Langone Health, said 2020 has been a year thats seen cancer research push forward on multiple fronts.

He said technologies are in development for early detection of cancer by way of blood tests.

Its been known for quite some time that tumors release DNA into the blood stream, we have technology developing from the standpoint of monitoring tumors, conducting sensitive tests for tumors, for tests for recurrence of cancers and protein-based tests, Neel told Healthline, outlining current research.

He also cited technology that modulates the regulatory DNA sequence patterns which refers to the part of the DNA molecule that can change the way a gene expresses itself in a living thing to pinpoint when methylation patterns might point to the development of cancer.

Among other research highlights over the past year, Neel said researchers have been developing new ways of drugging genetic mutations.

He mentioned work being done in developing a compound to degrade the androgen receptor for prostate cancer cells what allows these cancer cells to grow.

One of the biggest changes this year came in the form of how our new normal work-from-home lifestyle has impacted medicine.

As more and more people stay away from offices and public spaces, theyre turning to telemedicine. The Zoom screen is the new doctors office.

Morris said that, while this isnt a medical discovery per se, its a crucial in some ways life-saving development for how we relate to healthcare in our lives.

Out of this whole pandemic, one of the things weve discovered as clinicians is that we need to see patients where they are and not force them to cross state lines, Morris said. While we had telemedicine, we had some patients over Skype and video visits, there were clear disincentives and policies in place against people easily crossing state lines to seek medical care, of receiving care remotely.

He said the pandemic facilitated a push at the government level and with state and federal regulators to reduce barriers to these tools that are critical lifelines for patients.

Even when healthcare professionals couldnt always see patients in person this year, the embrace of telemedicine has resulted in unprecedented increases in the adoption and use of these tools and seeking care, Morris added.

This pandemic has challenged us to question old perceptions and policies, so that was a very positive thing, he stressed.

Innovation doesnt necessarily have to be an aha moment in a lab or something right in front of us, he said. Its unfortunate we needed a pandemic or a challenge to sometimes see a barrier, and sometimes that barrier is us.

This year, a possible breakthrough in Alzheimers disease research and treatment came in the form of a blood test that can diagnose this progressive form of dementia.

While the news is huge, the test is still in the trial phase.

If ultimately approved, a simple test for the condition would be a game changer.

There are as many as 5 million people living with Alzheimers in the United States, a number that will likely triple by 2060, according to the Centers for Disease Control and Prevention (CDC).

While this test has yet to go through all the proper approvals, a company distributed the first publicly available Alzheimers blood test this fall.

As 2020 comes to a close, experts are looking to a more hopeful new year.

For its part, Cleveland Clinic, which enters its centennial next year, released a list of the predicted top 10 innovations of 2021.

Morris said that while many think of this year as fraught and divisive full of tragedy and setbacks looking back at these innovations shows theres always something to be grateful for and look forward to.

Elemento said that he expects a biotech and pharma boom in the next few years.

Citing the breakthroughs in gene therapies and genetic manipulation indicate what will be a continual embrace of this kind of medical technology.

All these technologies, now everyone knows they exist and that they can be used for good, it will be a big boom for these technologies, Elemento added.

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5 Medical Innovations You Probably Didn't Notice Happened in 2020 - Healthline


Global CAR-T Pipeline Insight Report 2020: Overview, Landscape, Therapeutic Assessment, Current Treatment Scenario and Emerging Therapies -…

Wednesday, December 30th, 2020

Dublin, Dec. 30, 2020 (GLOBE NEWSWIRE) -- The "CAR-T - Pipeline Insight, 2020" drug pipelines has been added to's offering.

The "CAR-T - Pipeline Insight, 2020," report provides comprehensive insights about 250+ companies and 250+ pipeline drugs in CAR-T pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

CAR-T: Overview

CAR-T is a type of treatment in which a patient's T cells (a type of immune system cell) are changed in the laboratory so they will attack cancer cells. T cells are taken from a patient's blood. Then the gene for a special receptor that binds to a certain protein on the patient's cancer cells is added to the T cells in the laboratory. The special receptor is called a chimeric antigen receptor (CAR). Large numbers of the CAR T cells are grown in the laboratory and given to the patient by infusion. CAR T-cell therapy is used to treat certain blood cancers, and it is being studied in the treatment of other types of cancer. Also called chimeric antigen receptor T-cell therapy.

Potential Mechanisms of CAR-T Cell-Mediated Toxicity

Significant progress has been made in the field of cancer immunotherapy, and CAR-T cells have shown outstanding efficacy in clinical trials. As with all technologies, CAR-T technologies also need to go through a long process of development, and CAR-T cell therapy has related acute and chronic toxicities that have become a roadblock on the developmental path. If these setbacks are not overcome, it will be difficult to make a more significant breakthrough.

Cytokine Release Syndrome

Cytokine release syndrome (CRS) is the most common toxic side effect in CAR-T cell therapy. CRS is a systemic inflammatory response caused by the significant increase in cytokines accompanied by the rapid in vivo activation and proliferation of CAR-T cells, usually occurring within a few days after the first infusion. CRS is a clinical condition with mild symptoms of fever, fatigue, headache, rash, joint pain, and myalgia. Severe CRS cases are characterized by tachycardia, hypotension, and high fever. Mild to moderate CRS is usually self-limiting and can be managed through close observation and supportive care. Severe CRS must be treated with tocilizumab or steroids alone for intensive treatment.

Advances in Research of CAR-T Cell Therapy for Solid Tumors

Although early CAR-T cell trials of solid tumors did not show the same success as observed in leukemia trials, a better understanding of the multiple barriers seen in solid tumors could promote the design of clinical trials for CAR-T cells. In this early stage of clinical development, CAR-T cells offer much hope. The ability of genetic manipulation techniques to modify CAR-T cells provides almost unlimited opportunities for other changes and improvements, thus providing a strong desire for future success.

Global Landscape of CAR-T Cell Therapy

At present, CAR-T cells are widely used in cellular immunotherapy for various tumors. According to statistics, more than 300 clinical trials of CAR-T cell therapies have been approved by many national drug regulatory agencies, including the FDA of the United States. Statistical data from these clinical trials show that although the effects of various clinical trials vary due to the use of different sources and the preparation techniques of CARs and T cells, as well as differences in pretreatment and combinations of drugs, overall, CAR-T cells are effective in treating tumors with an effective rate of 30% to 70% or even more than 90%. For example, the complete remission rate for r/r ALL treated with the Novartis drug CTL0l9, which the FDA has approved, is 93%. Perhaps CAR-T cell therapy will ultimately remedy the fate of human cancer.

CAR-T Emerging Drugs Chapters

This segment of the CAR-T report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

CAR-T: Therapeutic Assessment

This segment of the report provides insights about the different CAR-T drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in CAR-T

There are approx. 250+ key companies which are developing the therapies for CAR-T. The companies which have their CAR-T drug candidates in the most advanced stage, i.e. phase III include, Janssen Research & Development, ViiV Healthcare, Sorrento Therapeutics, Celgene, Novartis, Abbott etc.

Report Highlights

Current Treatment Scenario and Emerging Therapies:

Key Players

Key Products

For more information about this drug pipelines report visit

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Global CAR-T Pipeline Insight Report 2020: Overview, Landscape, Therapeutic Assessment, Current Treatment Scenario and Emerging Therapies -...


VBL Therapeutics Announces First Patient in Europe in the OVAL Trial – BioSpace

Wednesday, December 30th, 2020

TEL AVIV, Israel, Dec. 29, 2020 (GLOBE NEWSWIRE) -- VBL Therapeutics(Nasdaq: VBLT) today announced the expansion of its ongoing OVAL Phase 3 study investigating ofranergene obadenovec (VB-111), for the treatment of platinum-resistant ovarian cancer into Europe, where the first patient has now been enrolled. The study continues to actively recruit patients in the U.S. and Israel, with over 200 patients enrolled to date.

VB-111 is our proprietary anti-cancer gene therapy product candidate that has shown overall survival benefit across multiple tumor types, said Dror Harats, M.D., Chief Executive Officer of VBL Therapeutics. We are pleased to expand the OVAL potential registration study of VB-111 in patients with late stage ovarian cancer to Europe, which is expected to accelerate our recruitment pace, diversify the patient population in the study and support our dialogue with European regulatory authorities as we get closer to potential commercialization. If successful and approved, VB-111 has the potential to establish a new standard of care in a challenging disease setting where patients currently have limited options.

Interim analysis from OVAL demonstrated VB-111s significant response rate of 58% or higher in the first 60 patients. According to the Company update on November 16, 2020, the high response rate of >50% in the total evaluable patient population was still maintained with approximately 200 patients enrolled.

The EU expansion follows two completed analyses by the independent Data Safety Monitoring Committee (DSMC) which recommended to continue the study as planned. The next DSMC review is expected in the first quarter of 2021.

VB-111 has received an Orphan Designation for the treatment of ovarian cancer from theEuropean Commission.

About the OVAL study (NCT03398655)OVAL is an international Phase 3 randomized pivotal potential registration clinical trial that compares a combination of VB-111 and paclitaxel to placebo plus paclitaxel, in patients with platinum-resistant ovarian cancer. The study is planned to enroll approximately 400 patients. OVAL is conducted in collaboration with theGOG Foundation, Inc., an independent international non-profit organization with the purpose of promoting excellence in the quality and integrity of clinical and basic scientific research in the field of gynecologic malignancies.

About VB-111 (ofranergene obadenovec)VB-111 is an investigational, first-in-class, targeted anti-cancer gene therapy agent that is being developed to treat a wide range of solid tumors. VB-111 is a unique biologic agent that uses a dual mechanism to target solid tumors. Its mechanism combines blockade of tumor vasculature with an anti-tumor immune response. VB-111 is administered as an IV infusion once every 6-8 weeks. It has been observed to be well-tolerated in >300 cancer patients and demonstrated activity signals in an all comers Phase 1 trial as well as in three tumor-specific Phase 2 studies. VB-111 has received an Orphan Designation for the treatment of ovarian cancer from theEuropean Commission. VB-111 has also received orphan drug designation in both the US andEurope, and fast track designation in the US for prolongation of survival in patients with rGBM. VB-111 successfully demonstrated proof-of-concept and survival benefit in Phase 2 clinical trials in radioiodine-refractory thyroid cancer and recurrent platinum-resistant ovarian cancer (NCT01711970).

About VBLVascular Biogenics Ltd., operating asVBL Therapeutics, is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class treatments for areas of unmet need in cancer and immune/inflammatory indications.

Forward Looking StatementsThis press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, look forward to, may, plan, potential, predict, project, should, will, would and similar expressions. These forward-looking statements may include, but are not limited to, statements regarding our programs, including VB-111, including their clinical development, therapeutic potential and clinical results. These forward-looking statements are not promises or guarantees and involve substantial risks and uncertainties. Among the factors that could cause actual results to differ materially from those described or projected herein include uncertainties associated generally with research and development, clinical trials and related regulatory reviews and approvals, the risk that historical clinical trial results may not be predictive of future trial results, that our financial resources do not last for as long as anticipated, and that we may not realize the expected benefits of our intellectual property protection. In particular, the DSMC recommendation that the OVAL trial proceed is not assurance that the trial will meet its primary endpoint of overall survival once completed, or that we will obtain positive results to support further development of this candidate. A further list and description of these risks, uncertainties and other risks can be found in our regulatory filings with theU.S. Securities and Exchange Commission, including in our annual report on Form 20-F for the year endedDecember 31, 2019, and subsequent filings with theSEC. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.VBL Therapeuticsundertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise.

INVESTOR CONTACT:Irina KofflerLifeSci 970-4681

VBL Therapeutics Announces First Patient in Europe in the OVAL Trial - BioSpace


After years of potential, cell and gene therapy is ready for the pharmaceutical mainstream – PMLiVE

Tuesday, December 22nd, 2020

The argument for continued investment

C> is a high potential and maturing sector, and is an already crowded environment, playing host to numerous start-ups and now, through M&A, recognised big pharma firms. Much like the rush to find a COVID-19 vaccine that dominates headlines worldwide, not every company involved will be able to succeed.

But finnCaps finnLife watch list of 50 leading AIM-listed biotech companies demonstrates that there is room for numerous companies to contribute to, and profit from, C>. Examining three entirely different approaches to CAR-T therapy, it is possible to see just how much space there is for this exciting sector, therefore displaying the case for continued investment.

Innovative CAR-T therapy demonstrates the depth of C> potential

CAR-T therapy in its existing form is a relatively new and specialised approach at treating cancer. It takes T cells from a patients bloodstream and genetically modifies them in a laboratory. These T cells are then injected back into the bloodstream with the aim of targeting and killing cancer cells.

While it has been shown to be an effective treatment, there are risks and side effects. One is the two-step autologous process (the slow time it takes for cell expansion sometimes as long as two weeks) while another is cytokine release syndrome (CRS), which occurs when cytokine molecules are inadvertently released, but too quickly to target just the tumours and instead target healthy cells.

The next generation of CAR-T treatments shows that there is space for a multitude of start-ups to be active in the C> space as they all help find varied solutions to these problems without negating the effectiveness of CAR-T.

One example is Horizon Delivery, a company that is developing its CYAD-02 project, which will help transport T cells more effectively to the tumour via the use of SMARTvector products.

The product underwent its first phase 1 trial test in January 2020 with a patient who was suffering from acute myeloid leukaemia. Horizon Delivery is also an industry leader in CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) screenings, meaning they can identify key genes or genetic sequences that draw out specific functions of a cell type from thousands of potential variants.

In a cancer context, this means they can route out and exclusively eliminate problematic cells that may have shown signs theyd resist a future cancer treatment.

Another example is Maxcyte, a global cell- based therapies and life sciences company that is developing its CARMA process, where a patients peripheral blood mononuclear cells (PBMCs) are removed and modified. The modified cells can then be used to target an array of different cancers.

Currently the company is conducting a phase 1 trial for advanced ovarian cancer in a dose escalation trial that will treat four separate cohorts the fourth of which was administered in March 2020.

Another example which shows the versatility of new CAR-T innovation is provided by Oxford Biomedica, a gene and cell therapy company specialising in the development of gene-based medicines.

Rather than a contained project or platform, its contribution to CAR-T is through a contract manufacturing development organisation. Collaborating with pharma companies, Oxford Biomedica uses its infrastructure to produce other companies licensed products, including Novartis Kymriah treatment (alongside other undisclosed CAR-T-related products).

With fast-moving innovation finally allowing multiple C> treatments to gain regulatory approval, along with a huge pipeline of upcoming therapies and an influx of funding and M&A activity, investing in C> no longer entails taking a bet on potential the future is finally here.

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After years of potential, cell and gene therapy is ready for the pharmaceutical mainstream - PMLiVE


Neurogene adds another $115M to the pot for trials for its gene therapies – FierceBiotech

Tuesday, December 22nd, 2020

Neurogene has raised $115 million in a second-round financing that will accelerate its plans to start clinical trials of a range of gene therapies for inherited neurological diseases, initially focusing on a form of Batten disease.

Batten disease is a group of disorders caused by a deficiency in proteins that allow fatty substances build up in nervous tissue, causing seizures, visual impairment, mobility loss and early death.

The New York biotechfounded by former Wall Street analyst Rachel McMinn, Ph.D., two years agosaid some of the cash will be used to advance its lead programs targeting Batten disease caused by CLN5 and CLN7 mutationstwo rare and rapidly progressive subtypes that occur in later childhood.

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It will also fund development of candidates for lysosomal storage disorders Charcot-Marie-Tooth disease (CMT) type 4J, caused by changes in the FIG4 gene, and aspartylglucosaminuria (AGU), which arises when the AGA gene is mutated.

The funding will also go toward developing its gene therapy platform and scaling up its manufacturing capacity. It adds to the $68.5 million the biotech raised in its series A round last year.

The AGU and CMT4J programs had been scheduled to reach the clinic this year, but Neurogene has been making quicker progress with the Batten disease drive.

Furthest along right now is its CLN5 candidate, which should start clinical trials next year. Batten disease is an autosomal recessive disorder, which means it only develops if a person inherits two copies of a faulty gene for their parents.

Neurogene intends to treat it by using an adeno-associated virus vector to deliver a replacement CLN5 gene, restoring the activity of the protein it codes for. While the function of that protein isnt well understood, the hope is that restoring its activity will slow down or halt the progression of the disease.

Neurogene isnt the only group eyeing the potential for gene therapies to make a difference to the lives of patients with Batten disease.

Abeona Therapeutics has been working in this area for a few years, and earlier this year licensed its ABO-202 candidate for CLN1 diseasealso known as infantile Batten diseaseto Taysha Gene Therapies for $7 million upfront and up to $56 million in milestones. It is also developing ABO-201 for CLN3 disease.

Last year, Amicus Therapeutics presented the first results with its CLN6 gene therapyacquired as part of its $100 million takeover of Celenex in 2018that seem to indicate a slowing of neurological decline.

Meanwhile, researchers at Cornell University have carried out a phase 1 gene therapy trial in late- infantile (CLN2) disease with similar positive results, and have also started a phase 1/2 study in this group.

Gene therapy has generated tremendous hope for the many families and patients with severe genetic disorders, McMinn said.

We believe our focus on improved product design, innovative technology, cutting-edge vector manufacturing and premier analytics will help fulfill the potential of genetic treatments.

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Neurogene adds another $115M to the pot for trials for its gene therapies - FierceBiotech


Thermo Fisher Invests in Gene Therapy Future with Huge Expansions – BioSpace

Tuesday, December 22nd, 2020

Tada Images/Shutterstock

Thermo Fisher Scientific has been as busy as Santas elves this December. The company is significantly expanding its footprint with new facilities in the United States and in Europe.

This week, the company announced it started construction of a new cGMP plasmid DNA manufacturing facility inCarlsbad, Calif.This expansion builds on the company's continued investment in cell and gene therapy services. The site will expand the company's clinical and commercial capabilities for cGMP plasmid DNA used as a critical raw material to develop and manufacture cell and gene-based therapies including life-saving cancer treatments as well as mRNA vaccines. In addition, the site will have the capability to produce large-scale plasmid DNA as a primary drug substance for DNA therapies.

The California site will span 67,000 square feet and is expected to be completed in the first half of 2021. The facility will feature advanced technologies, including single use equipment with up to 1,000L scale, digital connectivity and data visibility to enable operational efficiencies and operator training, the company said. The new commercial facility will add approximately 150 jobs over the next 12 months.

The race to develop new transformative cell and gene therapies and vaccines is outpacing supply of commercial-quality plasmid DNA that can be produced at scale, Mike Shafer, senior vice president and president of pharma services at ThermoFisher said in a statement. Our new state-of-the art site will not only tackle the supply bottleneck for our customers, but also uniquely positions us to deliver robust, end-to-end cell and gene therapy capabilities.

Shafer said Thermo Fisher is making strategic investments in capacity, technology and expertise across its global network so the company can accelerate innovation and enhance productivity for its customers.

The California site isnt the only expansion the company is undertaking. To meet accelerating demand for robust clinical supply chain services throughout Europe, Thermo Fisher Scientific has expanded its pharma services footprint with two new facilities inRheinfeldenand Weil am Rhein, Germany. The new sites will boost clinical supply chain continuity and specialized cold chain and cryogenic expertise across Europeand globally.

TheRheinfeldensite opens in late December 2020 and the Weil am Rhein site will open in January 2021.InRheinfelden, the new 86,000-square-foot/8,000-square-meter facility significantly increases the company's footprint for secondary packaging, storage, logistics and distribution of clinical supplies to investigator sites across Europe. In Weil am Rhein, the new 9,600-square-foot/890-square-metercryocenterprovides specialized ultra-low-temperature, cryogenic storage and cold chain expertise for clinical supply chain needs for cell and gene-based therapies, including COVID-19 vaccine candidates.

These facilities combined with our established regulatory expertise will give customers the continuity and in-region capabilities to support clinical trials across multiple therapy areas, Shafer said.

In addition to the construction of new facilities, earlier this month, Thermo Fisher announced the expansion of several of its existing sites that will increase the companys capabilities for sterile drug product development and commercial manufacturing of critical medicines, therapies and vaccines.

Thermo Fisher is expanding its facilities in Greenville, N.C., Swindon, England and two sites in Italy, Ferentino and Monza. Not only will the expansion of these facilities boost commercial production lines and support capabilities for aseptic liquid and lyophilized vial filling, but the projects will also add approximately 1,000 new jobs. The expansions are expected to be completed over the next two years, Thermo Fisher said in its announcement.

In addition to expansions inNorth AmericaandEurope, the company recently announced significant projects inAsia-Pacific, including a new sterile manufacturing facility inSingaporeand a new integrated biologics and sterile drug development and manufacturing site inHangzhou, China.

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Thermo Fisher Invests in Gene Therapy Future with Huge Expansions - BioSpace


Taysha Gene Therapies Announces Queen’s University’s Receipt of Clinical Trial Application Approval from Health Canada for Phase 1/2 Clinical Trial of…

Tuesday, December 22nd, 2020

DetailsCategory: DNA RNA and CellsPublished on Tuesday, 22 December 2020 18:50Hits: 116

TSHA-101 to be first bicistronic vector evaluated in human clinical trials; TSHA-101 designed to deliver both HEXA and HEXB transgenes within a single AAV9 vector construct

TSHA-101 CTA is the second clinical trial clearance received, in addition to TSHA-118s open investigational new drug application for CLN1

Interim data from Phase 1/2 trial anticipated in 2021

DALLAS, TX, USA I December 21, 2020 I Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that Queens University in Ontario, Canada, received Clinical Trial Application (CTA) approval from Health Canada for its investigator-sponsored Phase 1/2 trial exploring TSHA-101, Tayshas investigational AAV9-based gene therapy, for the treatment of infantile GM2 gangliosidosis.

TSHA-101 will be the first bicistronic vector to enter a first-in-human clinical study, which is a significant milestone for Taysha and for the field of gene therapy, said Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. GM2 is a devastating lysosomal storage disease with no approved treatments and todays CTA approval marks a formative moment for children suffering from this rapidly progressive and fatal disease.

The trial will be a single arm, open-label Phase 1/2 trial evaluating the use of TSHA-101 for the treatment of infants with GM2. The study will be sponsored by Queens University and led by Jagdeep S. Walia, MBBS, FRCPC, FCCMG, Clinical Geneticist and Associate Professor Head, Division of Medical Genetics (Department of Pediatrics) at Queens, and Director of Research (Department of Pediatrics), at the Kingston Health Sciences Centre.

Preclinical evidence to date supports our belief that TSHA-101, when given intrathecally as a bicistronic transgene packaged into a single AAV9 vector, has the potential to address the lysosomal enzyme deficiency, to change the disease trajectory and to improve patient survival, said Dr. Jagdeep S. Walia. We are pleased to have the support of Health Canada as we continue to advance TSHA-101.

Todays CTA approval is a culmination of our teams and Dr. Walias tireless efforts and a momentous occasion for children affected by GM2 along with their parents and caregivers, said RA Session II, Founder, President and CEO of Taysha. We are grateful to our partners at Queens University for their work to advance this gene therapy into the clinic.

About GM2 Gangliosidosis

GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder and a family of neurodegenerative genetic diseases that includes Tay-Sachs and Sandhoff diseases. The disease is caused by defects in the HEXA or HEXB genes that encode the two subunits of the -hexosaminidase A enzyme. These genetic defects result in progressive dysfunction of the central nervous system. There are no approved therapies for the treatment of the disease, and current treatment is limited to supportive care.

About TSHA-101

TSHA-101 is an investigational gene therapy administered intrathecally for the treatment of infantile GM2 gangliosidosis. The gene therapy is designed to deliver two genes HEXA and HEXB driven by a single promoter within the same AAV9 construct, also known as a bicistronic vector. This approach allows the simultaneous expression of a 1:1 ratio of the two subunits of protein required to generate a functional enzyme. It is the first and only bicistronic vector currently in clinical development and has been granted Orphan Drug and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA).

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at

SOURCE: Taysha Gene Therapy

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Taysha Gene Therapies Announces Queen's University's Receipt of Clinical Trial Application Approval from Health Canada for Phase 1/2 Clinical Trial of...


Lilly picks up gene therapy programme in $1bn Prevail Therapeutics acquisition deal – PMLiVE

Tuesday, December 22nd, 2020

Eli Lilly has stepped into the gene therapy space after announcing a deal to acquire Prevail Therapeutics, a company focused on developing adeno-associated virus (AAV)-based gene therapies for neurodegenerative diseases.

Lilly will acquire Prevail for $22.50 per share in cash, plus one $4 contingent value right dependent on the first regulatory approval of a product from Prevails pipeline.

This reflects a potential consideration of up to $26.50 per share in cash for a total consideration of approximately $1.04bn.

For Lilly, the acquisition will extend its focus into developing gene therapies, establishing an in-house gene therapy programme anchored by Prevails current portfolio and AAV-based technology.

Prevails pipeline spans clinical-stage and preclinical neuroscience assets, including lead gene therapies PR001 for patients with Parkinsons disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD) and PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN).

The companys preclinical pipeline also includes PR004, a potential gene therapy for patients with specific synucleinopathies, as well as candidates for Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders.

"The acquisition of Prevail will bring critical technology and highly skilled teams to complement our existing expertise at Lilly, as we build a new gene therapy programme anchored by well-researched assets, said Mark Mintun, vice president of pain and neurodegeneration research at Lilly.

We look forward to completing the proposed acquisition and working with Prevail to advance their ground-breaking work through clinical development, he added.

For Prevail to achieve the full value of the contingent CVR payment, the first regulatory approval arising from its current gene therapy pipeline must happen by 31 December 2024.

Failing regulatory approval by this date, Lilly said in a statement that the value of the CVR will decrease by approximately 8.3 cents per month until the expiration date 1 December 2028.

Within Prevails clinical pipeline, PR001 has already scored a US Food and Drug Administration (FDA) fast-track designation for the treatment of PD-GBA patients and nGD.

It has also been granted an FDA orphan drug designation for the treatment of Gaucher disease, and rare paediatric disease designation for the treatment of nGD.

Prevails PR006 gene therapy also has an FDA and European Commission orphan designation for the treatment of FTD, with the FDA also handing it a fast-track designation for FTD-GRN.

In November, Lilly signed a deal with Precision BioSciences focused on genome editing research, with an initial focus on developing in vivo therapies for Duchenne muscular dystrophy and two other undisclosed gene targets.

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Lilly picks up gene therapy programme in $1bn Prevail Therapeutics acquisition deal - PMLiVE


After leaving Wall Street to launch a gene therapy upstart, Rachel McMinn nabs $115M to drive her first candidate to the clinic – Endpoints News

Tuesday, December 22nd, 2020

When former analyst Rachel McMinn started Neurogene from her apartment around three years ago, she would joke that theyd get office space as soon as her living room table was no longer big enough to hold company meetings.

We lasted about a year before my living room couldnt take it anymore, she said.

With several gene therapies for Batten disease and other lysosomal storage disorders in the preclinical and discovery stage, Neurogene is now bound for the clinic. And on Wednesday, they announced a $115 million Series B to get them there.

Gene therapy has generated so much enthusiasm for patients and families with these devastating disorders, but theres still a lot of science and innovation left on the table, McMinn said.

The CEO said Neurogene will split the Series B funds into four buckets, the first of which is advancing multiple gene therapy programs into the clinic. She anticipates filing the first IND in 2021 for CLN5, a rapidly progressive subtype of Batten disease caused by a variant in the CLN5 gene.

The second so-called bucket for the Series B funds will be expanding the companys portfolio, followed by another bucket for augmenting our resources for our novel technology platform, the CEO said. Then comes manufacturing.

Weve got the ability to make virus in-house, and the money from the financing will allow us to take that vector to the next stage and make GMP quality vector for use in dosing and clinical trials, McMinn said.

Because Neurogene manufactures products in-house, the biotech has gotten around the massive gene therapy manufacturing bottleneck, which has Big Pharma and big biotech spending billions on retrofitted plants and gene therapy factories.

The concept of gene therapy is simple: A viral particle is used to deliver a healthy copy of a gene to a patient with a dysfunctional gene. In the case of Neurogenes CLN5 candidate, viral vectors shuttle a payload into the body designed to make the CLN5 gene.

Over the next year, key milestones will be filing our first IND, completing the refurbishment of our GMP manufacturing facility, (and) advancing our programs towards the clinic, McMinn said. After CLN5, the goal is to file one to two INDs a year, she added.

The CEO previously served as an analyst at Piper Jaffray, Cowen and Bank of America Merrill Lynch, and as chief business and strategy officer at Intercept. During her time as an analyst, McMinn said most people would stay away from investing in neurology companies because drugs inevitably fail.

Theres really been nothing, very little innovation in devastating neurological disorders, for quite a long time, she said, adding that she was inspired to jump into R&D by an older brother who is neurologically impaired.

Neurogene attracted a slate of new and old investors, including EcoR1 Capital which led the round, and Redmile Group, Samsara BioCapital, Cormorant Asset Management, BlackRock, funds managed by Janus Henderson Investors, Casdin Capital, Avidity Partners, Ascendant BioCapital, Arrowmark Partners, Alexandria Venture Investments, and an undisclosed leading healthcare investment fund.

For me, I really want to make a difference, McMinn said, adding later, Im personally driven by developing something that is life-altering for people that really have no other option.

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After leaving Wall Street to launch a gene therapy upstart, Rachel McMinn nabs $115M to drive her first candidate to the clinic - Endpoints News


Global Gene Therapy Market Report 2020-2030 Featuring Novartis, Bluebird Bio, Spark Therapeutics, Audentes Therapeutics, Voyager Therapeutics,…

Tuesday, December 22nd, 2020

DUBLIN, Dec. 17, 2020 /PRNewswire/ -- The "Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change" report has been added to's offering.

Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change provides the strategists, marketers and senior management with the critical information they need to assess the global gene therapy market market.

Major players in the gene therapy market are Novartis AG, Bluebird Bio, Inc., Spark Therapeutics, Inc., Audentes Therapeutics, Voyager Therapeutics, Applied Genetic Technologies Corporation, UniQure N.V., Celgene Corporation, Cellectis S.A. and Sangamo Therapeutics.

The global gene therapy market is expected to decline from $3.22 billion in 2019 to $3.18 billion in 2020 at a compound annual growth rate (CAGR) of -1.30%. The decline is mainly due to the COVID-19 outbreak that has led to restrictive containment measures involving social distancing, remote working, and the closure of industries and other commercial activities resulting in operational challenges. The market is then expected to recover and reach $6.84 billion in 2023 at a CAGR of 29.09%.

The gene therapy market consists of sales of gene therapy related services by entities (organizations, sole traders and partnerships) that manufacture gene therapy drugs. Gene therapy is used to replace faulty genes or add new genes to cure disease or improve the body's ability to fight disease. Only goods and services traded between entities or sold to end consumers are included.

North America was the largest region in the gene therapy market in 2019.

The gene therapy market covered in this report is segmented by gene type into antigen; cytokine; suicide gene; others. It is also segmented by vector into viral vector; non-viral vector; others, by application into oncological disorders; rare diseases; cardiovascular diseases; neurological disorders; infectious diseases; others, and by end users into hospitals; homecare; specialty clinics; others.

In December 2019, Roche, a Switzerland-based company, completed its acquisition of Spark Therapeutics for $4.3 billion. With this deal, Roche is expected to strengthen its presence in the gene therapy segment, support transformational therapies and increase its product portfolio. Spark Therapeutics is a US-based company involved in gene therapy.

The high prices of gene therapy medicines are expected to limit the growth of the gene therapy market. The pressure to contain costs and demonstrate value is widespread. Political uncertainty and persistent economic stress in numerous countries are calling into question the sustainability of public health care funding. In less wealthy countries, the lack of cost-effective therapies for cancer and other diseases has influenced the health conditions of the population and has led to a low average life expectancy.

Luxturna, a one-time treatment for acquired retinal eye disease, costs $850,000 in the US and 613,410 in the UK, despite a markdown that is applied through Britain's National Health Service. Zolgensma, for spinal muscular atrophy, is valued at $2.1 million in the US and Zynteglo, which focuses on a rare genetic blood disorder, costs $1.78 million, thus restraining the growth of the market.

The use of machine learning and artificial intelligence is gradually gaining popularity in the gene therapy market. Artificial intelligence (AI) is the simulation of human intelligence in machines, which are programmed to display their natural intelligence. Machine learning is a part of AI.

Machine learning and AI help companies in the gene therapy market to conduct a detailed analysis of all relevant data, provide insights between tumor and immune cell interactions, and offer a more accurate evaluation of tissue samples often conflicted between different evaluators. For instance, since January 2020, GlaxoSmithKline, a pharmaceutical company, has been investing in AI to optimize gene therapy and develop off-the-shelf solutions for patients. It is also expected to reduce turnaround time and also the cost of gene therapies.

Key Topics Covered:

1. Executive Summary

2. Gene Therapy Market Characteristics

3. Gene Therapy Market Size And Growth 3.1. Global Gene Therapy Historic Market, 2015 - 2019, $ Billion 3.1.1. Drivers Of The Market 3.1.2. Restraints On The Market 3.2. Global Gene Therapy Forecast Market, 2019 - 2023F, 2025F, 2030F, $ Billion 3.2.1. Drivers Of The Market 3.2.2. Restraints On the Market

4. Gene Therapy Market Segmentation 4.1. Global Gene Therapy Market, Segmentation By Gene Type, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.2. Global Gene Therapy Market, Segmentation By Vector, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.3. Global Gene Therapy Market, Segmentation By Application, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

4.4. Global Gene Therapy Market, Segmentation By End Users, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

5. Gene Therapy Market Regional And Country Analysis 5.1. Global Gene Therapy Market, Split By Region, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion 5.2. Global Gene Therapy Market, Split By Country, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion

Companies Mentioned

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Taysha Gene Therapies looks to build on rapid IPO with $85M dev-manufacturing plant in North Carolina – Endpoints News

Tuesday, December 22nd, 2020

Taysha Gene Therapies went public with a bang earlier this year with a $157 million IPO just five months after its Series A financing. Now, with the help of the city of Durham, North Carolina, Taysha is plotting an expansive development and manufacturing facility to ramp up supplies for the clinic and potentially the commercial market.

The new 187,000-square-foot cGMP plant will create roughly 200 jobs for Taysha Gene Therapies, which formed in late 2019 with the sole focus of developing therapeutics for monogenic diseases using an adeno-associate virus methodology. The announcement continues a rapid ascent for Taysha, which launched Series A financing just in April, and a mere five months later had its own Nasdaq ticker, offering a $157 million IPO and pricing shares at $20 apiece.

The facility is the result of a public-private partnership between Taysha, the city of Durham and the state of North Carolina. Taysha will invest $75 million into the facility, with additional state and local incentives totaling another $9.4 million. All told, the 200 jobs will come to fruition over a two-and-a-half-year period across all functions, including gene therapy development, analytics, manufacturing and quality control testing.

Tayshas facility is expected to be fully operational by 2023 and will add 2,000 liters of capacity supporting all aspects of scalable gene therapy manufacturing. The company said in a news release it will now be able to meet the clinical and commercial demands within the field when combined with its existing collaborations with the University of Texas Southwestern Medical Center and CDMO Catalent.

With our outstanding team of experts leading the charge, we expect this facility will serve as a center of excellence for gene therapy development, from preclinical studies through commercialization, and will further our leadership position in gene therapy as well as support our next phase of growth, RA Session II, Tayshas president, founder and CEO, said in a press release.

The new facility will serve as an integral part of rapid expansion for the Dallas-based biotech, which said it expects to have four open Investigational New Drug applications in 2021. Tayshas most advanced of those is a drug being developed for the treatment of GM2 gangliosidosis, a family of neurodegenerative disorders that includes Tay-Sachs disease and Sandhoff disease.

The other three such programs are looking to treat Rett Syndrome, epilepsy and SURF1 deficiencies.

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Taysha Gene Therapies looks to build on rapid IPO with $85M dev-manufacturing plant in North Carolina - Endpoints News


Abeona Therapeutics Announces Acceptance of Late-Breaker Abstracts Highlighting New Clinical Data for Novel AAV-based Gene Therapies in MPS IIIA and…

Tuesday, December 22nd, 2020

NEW YORK and CLEVELAND, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that abstracts detailing new interim results from its ABO-102 Phase 1/2 Transpher A study for MPS IIIA and ABO-101 Phase 1/2 Transpher B study for MPS IIIB have been accepted for platform oral presentations during the late-breaking abstract session at the 17th Annual WORLDSymposium being held February 8-12, 2021.

Children born with MPS IIIA and MPS IIIB experience progressive neurodevelopmental decline and loss of motor function that is life-threatening, said Michael Amoroso, Chief Operating Officer of Abeona. We are excited to share new analyses from the Transpher A study that will add to the understanding of the potential for ABO-102 to help preserve neurocognitive development in patients with MPS IIIA when they are treated at a young age, and new results from the Transpher B study that will provide insights into ABO-101s biologic effect in patients with MPS IIIB.

Presentation Details

Title: Updated Results of Transpher A, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial of ABO-102 Gene Therapy for Sanfilippo Syndrome Type A (Mucopolysaccharidosis IIIA)Abstract Number: 390Presenter: Kevin Flanigan, M.D., Center for Gene Therapy at Nationwide Childrens HospitalDate/Time: Friday, February 12, 2021, time to be determined

Title: Updated Results of Transpher B, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial of ABO-101 Gene Therapy for Sanfilippo Syndrome Type B (Mucopolysaccharidosis IIIB)Abstract Number: 407Presenter: Maria Jose de Castro, M.D., Hospital Clnico Universitario Santiago de CompostelaDate/Time: Friday, February 12, 2021, time to be determined

About the Annual WORLDSymposium The WORLDSymposium is designed for basic, translational and clinical researchers, patient advocacy groups, clinicians, and all others who are interested in learning more about the latest discoveries related to lysosomal diseases and the clinical investigation of these advances. For additional information on the 17th Annual WORLDSymposium, please visit

About the Transpher A Study The Transpher A Study (NCT02716246) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). The study, also known as ABT-001, is intended for patients from birth to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-102 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment changes and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

About the Transpher B Study The Transpher B Study (NCT03315182) is an ongoing, two-year, open-label, dose-escalation, Phase 1/2 global clinical trial assessing ABO-101 for the treatment of patients with Sanfilippo syndrome type B (MPS IIIB). The study, also known as ABT-002, is intended for patients from birth to 2 years of age, or patients older than 2 years with a cognitive developmental quotient of 60% or above. ABO-101 gene therapy is delivered using AAV9 technology via a single-dose intravenous infusion. The study primary endpoints are neurodevelopment changes and safety, with secondary endpoints including behavior evaluations, quality of life, enzyme activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate levels in CSF, plasma and urine, and brain and liver volume.

About ABO-102 ABO-102 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type A (MPS IIIA), a rare lysosomal storage disease with no approved treatment that primarily affects the central nervous system (CNS). ABO-102 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the SGSH gene to cells of the CNS and peripheral organs. The therapy is designed to address the underlying SGSH enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations for the ABO-102 clinical program. In the EU, the Company holds PRIME and Orphan medicinal product designations.

About ABO-101 ABO-101 is a novel gene therapy in Phase 1/2 development for Sanfilippo syndrome type B (MPS IIIB), a rare lysosomal storage disease with no approved therapy that primarily affects the central nervous system (CNS). ABO-101 is dosed in a one-time intravenous infusion using a self-complementary AAV9 vector to deliver a functional copy of the NAGLU gene to cells of the CNS and peripheral tissues. The therapy is designed to address the underlying NAGLU enzyme deficiency responsible for abnormal accumulation of glycosaminoglycans in the brain and throughout the body that results in progressive cell damage and neurodevelopmental and physical decline. In the U.S., Abeona holds Fast Track and Rare Pediatric Disease designations for ABO-101 and Orphan Drug designation in both the U.S. and EU.

About Sanfilippo Syndrome Type A (MPS IIIA) Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the CNS and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIA present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive facial features with thick eyebrows or a unibrow, full lips and excessive body hair for ones age, and liver/spleen enlargement are also present in early childhood. MPS IIIA is caused by genetic mutations that lead to a deficiency in the SGSH enzyme responsible for breaking down glycosaminoglycans, which accumulate in cells throughout the body resulting in rapid health decline associated with the disorder.

About Sanfilippo syndrome type B (MPS IIIB) Sanfilippo syndrome type B (MPS IIIB) is a rare and fatal lysosomal storage disease with no approved therapy that primarily affects the central nervous system and is characterized by rapid neurodevelopmental and physical decline. Children with MPS IIIB present with progressive language and cognitive decline and behavioral abnormalities. Other symptoms include sleep problems and frequent ear infections. Additionally, distinctive signs such as facial features with thick eyebrows or a unibrow, full lips and excessive body hair for ones age and liver/spleen enlargement are also present. The underlying cause of MPS IIIB is a deficiency in the NAGLU enzyme responsible for breaking down glycosaminoglycans, which accumulate throughout the body resulting in rapid decline associated with the disorder.

About Abeona Therapeutics Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. Abeonas clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa in Phase 3 development, as well as ABO-102 and ABO-101, novel AAV-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB), respectively, in Phase 1/2 development. The Companys portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical needs. Abeonas novel, next-generation AIM capsids have shown potential to improve tropism profiles for a variety of devastating diseases. Abeonas fully functional, gene and cell therapy GMP manufacturing facility produces EB-101 for the pivotal Phase 3 VIITAL study and is capable of clinical and commercial production of AAV-based gene therapies. For more information, visit

Forward-Looking StatementsThis press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. These statements include statements about the Company exploring all strategic options, including the sale of some or all of its assets or sale of the Company. We have attempted to identify forward-looking statements by such terminology as may, will, believe, estimate, expect, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to the potential impacts of the COVID-19 pandemic on our business, operations, and financial condition, the outcome of the strategic review, continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the outcome of any future meetings with the U.S. Food and Drug Administration or other regulatory agencies, the impact of competition, the ability to secure licenses for any technology that may be necessary to commercialize our products, the ability to achieve or obtain necessary regulatory approvals, the impact of changes in the financial markets and global economic conditions, risks associated with data analysis and reporting, and other risks disclosed in the Companys most recent Annual Report on Form 10-K and subsequent quarterly reports on Form 10-Q and other periodic reports filed with the Securities and Exchange Commission. The Company undertakes no obligation to revise the forward-looking statements or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise, except as required by the federal securities laws.

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Abeona Therapeutics Announces Acceptance of Late-Breaker Abstracts Highlighting New Clinical Data for Novel AAV-based Gene Therapies in MPS IIIA and...


Orchard Therapeutics Receives EC Approval for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) – GlobeNewswire

Tuesday, December 22nd, 2020

First gene therapy to receivefull EU marketing authorization for eligible MLD patients

One-time treatment with Libmeldy has been shown to preserve motor and cognitive function

Achievement shared with research alliance partners Fondazione Telethon and Ospedale San Raffaele

BOSTON and LONDON and MILAN, Italy, Dec. 21, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, and its research alliance partners Fondazione Telethon and Ospedale San Raffaele, today announced that the European Commission (EC) granted full (standard) market authorization for Libmeldy (autologous CD34+ cells encoding the ARSA gene), a lentiviral vector-based gene therapy approved for the treatment of metachromatic leukodystrophy (MLD), characterized by biallelic mutations in theARSAgene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy is the first therapy approved for eligible patients with early-onset MLD.

MLD is a very rare, fatal genetic disorder caused by mutations in the ARSA gene which lead to neurological damage and developmental regression. In its most severe and common forms, young children rapidly lose the ability to walk, talk and interact with the world around them, and most pass away before adolescence. Libmeldy is designed as a one-time therapy that aims to correct the underlying genetic cause of MLD, offering eligible young patients the potential for long-term positive effects on cognitive development and maintenance of motor function at ages at which untreated patients show severe motor and cognitive impairments.

Todays EC approval of Libmeldy opens up tremendous new possibilities for eligible MLD children faced with this devastating disease where previously no approved treatment options existed, said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. Libmeldy is Orchards first product approval as a company, and I am extremely proud of the entire team who helped achieve this milestone. We are grateful for and humbled by the opportunity to bring this remarkable innovation to young eligible patients in the EU.

With Libmeldy, a patients own hematopoietic stem cells (HSCs) are selected, and functional copies of the ARSA gene are inserted into the genome of the HSCs using a self-inactivating (SIN) lentiviral vector before these genetically modified cells are infused back into the patient. The ability of the gene-corrected HSCs to migrate across the blood-brain barrier into the brain, engraft, and express the functional enzyme has the potential to persistently correct the underlying disease with a single treatment.

The EC approval of Libmeldy comes more than a decade after the first patient was treated in clinical trials performed at our Institute, and ushers in a remarkable and long-awaited shift in the treatment landscape for eligible MLD patients, said Luigi Naldini, M.D, Ph.D., director of the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. Our team at SR-Tiget has been instrumental in advancing the discovery and early-stage research of this potentially transformative therapy to clinical trials in support of its registration through more than 15 years of studies supported by Fondazione Telethon and Ospedale San Raffaele, and we are extremely proud of this achievement and what it means for patients and the field of HSC gene therapy.

MLD is a heart-breaking disease that causes immeasurable suffering and robs children of the chance of life, said Georgina Morton, chairperson of ArchAngel MLD Trust. As a community, we have been desperate for a treatment for young MLD patients, and we are incredibly excited to now have such a ground-breaking option approved in the EU.

The marketing authorization for Libmeldy is valid in all 27 member states of the EU as well as the UK, Iceland, Liechtenstein and Norway. Orchard is currently undertaking EU launch preparations related to commercial drug manufacturing, treatment site qualification and market access.

Data Supporting the Clinical and Safety Profile of Libmeldy

The marketing authorization for Libmeldy is supported by clinical studies in both pre- and early- symptomatic, early-onset MLD patients performed at the SR-Tiget. Early-onset MLD encompasses the disease variants often referred to as late infantile (LI) and early juvenile (EJ). Clinical efficacy was based on the integrated data analysis from 29 patients with early-onset MLD who were treated with Libmeldy prepared as a fresh (non-cryopreserved) formulation. Results of this analysis indicate that a single-dose intravenous administration of Libmeldy is effective in modifying the disease course of early-onset MLD in most patients.

Clinical safety was evaluated in 35 patients with MLD (the 29 patients from the integrated efficacy analysis as well as six additional patients treated with the cryopreserved formulation of Libmeldy). Safety data indicate that Libmeldy was generally well-tolerated. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies (AAA) reported in five out of 35 patients. Antibody titers in all five patients were generally low and no negative effects were observed in post-treatment ARSA activity in the peripheral blood or bone marrow cellular subpopulations, nor in the ARSA activity within the cerebrospinal fluid. In addition to the risks associated with the gene therapy, treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

For further details, please see the Summary of Product Characteristics (SmPC).

About MLD and Libmeldy

MLD is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see. In its late infantile form, mortality at five years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1

Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), also known as OTL-200, is approved in the European Union for the treatment of MLD in eligible early-onset patients. In the U.S., OTL-200 is an investigational therapy which has not been approved by the U.S. Food and Drug Administration (FDA) for any use. Libmeldy was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters inLondonandU.S.headquarters inBoston. For more information, please, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (, the investor relations website (, and on social media (Twitter andLinkedIn), including but not limited to investor presentations and investor fact sheets,U.S. Securities and Exchange Commissionfilings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

About Fondazione Telethon, Ospedale San Raffaele and the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele, a clinical-research-university hospital established in 1971 to provide international-level specialized care for the most complex and difficult health conditions, and Fondazione Telethon, an Italian biomedical charity born in 1990 and focused on rare genetic diseases. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases. Over the years, the Institute hasgiven a pioneering contribution to the field with relevant discoveries in vector design, gene transfer strategies, stem cell biology, identity and mechanism of action of innate immune cells. SR-Tiget has also established the resources and framework for translating these advances into novel experimental therapies and has implemented several successful gene therapy clinical trials for inherited immunodeficiencies, blood and storage disorders, which have already treated >115 patients and have led through collaboration with industrial partners to the filing and approval of novel advanced gene therapy medicines.

For more information:

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including its plans and expectations for the commercialization of Libmeldy, and the therapeutic potential of Libmeldy, including the potential implications of clinical data for eligible patients. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation:: the risk that prior results, such as signals of safety, activity or durability of effect, observed from clinical trials of Libmeldy will not continue or be repeated in our ongoing or planned clinical trials of Libmeldy, will be insufficient to support regulatory submissions or marketing approval in the US or to maintain marketing approval in the EU, or that long-term adverse safety findings may be discovered; the inability or risk of delays in Orchards ability to commercialize Libmeldy, including the risk that we may not secure adequate pricing or reimbursement to support continued development or commercialization of Libmeldy; the risk that the market opportunity for Libmeldy, or any of Orchards product candidates, may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards quarterly report on Form 10-Q for the quarter endedSeptember 30, 2020, as filed with theU.S. Securities and Exchange Commission(SEC), as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.


InvestorsRenee LeckDirector, Investor Relations+1

MediaChristine HarrisonVice President, Corporate Affairs+1

1 Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature.Journal of Child Neurology2010, DOI:

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Orchard Therapeutics Receives EC Approval for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) - GlobeNewswire


Health Canada approves Zolgensma, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA) – Canada NewsWire

Tuesday, December 22nd, 2020

Zolgensma is a gene therapy designed to address the genetic root cause of SMA by replacing the missing or defectiveSMN1gene1.It is administered during an intravenous (IV) infusion, delivering a new working copy of the SMN1 gene into a patient's cells, halting disease progression and restoring production of SMN protein1.

"SMA can be a devastating diagnosis for families to receive. Without treatment, many children would not be able to meet important developmental milestones like lifting their head, sitting or walking.Even breathing and swallowing can become difficult in the severe, infant-onset form of this disease," said Dr. Hugh McMillan, Pediatric Neurologist at the Children's Hospital of Eastern Ontario in Ottawa."The approval of Zolgensma in Canada offers children an opportunity to maximize their developmental potential from this one-time therapy.The decision to treat based upon weight may allow children diagnosed slightly later to also benefit from this therapy."

"When I first started diagnosing SMA, I couldn't have imagined that we would see such scientific advancements," said Dr. Nicolas Chrestian, Chief of Paediatric Neurology, specialized in neuromuscular disorders at Centre Hospitalier Mre Enfant Soleil, Universit Laval in Qubec City. "Zolgensma offers, in a single dose, the possibility of halting the progression of this degenerative condition that can rob children of regular developmental milestones."

In Canada each year, approximately one in 10,000 babies are born with SMA,a rare, genetic neuromuscular disease caused by a defective or missingSMN1gene3. Without a functionalSMN1gene, infants with SMA lose the motor neurons responsible for muscle functions such as breathing, swallowing, speaking and walking2. Left untreated, muscles become progressively weaker2,3. In the most severe form, this eventually leads to paralysis and ultimately permanent ventilation or death by age 2 in more than 90%of cases4.

"The SMA community is thrilled to have another treatment option to offer hope to families grappling with an SMA diagnosis. The approval of Zolgensma couldn't come soon enough. We will continue to advocate until everyone who needs access to treatment can benefit from innovations like this," said Susi Vander Wyk, Executive Director, CureSMA Canada.

"Today's announcement about the Canadian approval of Zolgensma is a significant milestone in our journey to reimagine medicine by changing the treatment paradigm for children with SMA." said Andrea Marazzi, Country Head, Novartis Pharmaceuticals Canada. "Our commitment to the SMA community truly comes to life when those that could benefit most from Zolgensma can access it. This is why we continue to work collaboratively with the pan-Canadian Pharmaceutical Alliance, provinces and territories to make this happen as quickly as possible."

The efficacy and safety data supporting the approval of Zolgensma in treating pediatric patients with SMA are derived from completed and ongoing open-label, single-arm, clinical trials in patients with infantile-onset SMA and 2 copies of SMN2 gene; and presymptomatic genetically diagnosed SMA and 2 or 3 copies of SMN2 gene1.

Zolgensma is the only gene therapy approved by Health Canada for the treatment of SMA1. Thirteen treatment sites have been identified in leading healthcare institutions with SMA expertise. The sites are located in: Vancouver, BC; Edmonton, AB; Calgary, AB; Saskatoon, SK; Winnipeg, MB; London, ON; Hamilton, ON; Toronto, ON; Ottawa, ON; Montreal, QC; Quebec City, QC; Halifax, NS.

About Spinal Muscular AtrophySMA is the leading cause of genetic infant death2. Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities5.This is why it is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression6.Early diagnosis is especially critical in the most severe form, where motor neuron degeneration starts before birth and escalates quickly5. Newborn screening for SMA is currently being implemented in Ontario and piloted in Alberta7,8.

About Novartis in Gene Therapy and Rare DiseaseNovartis is at the forefront of cell and gene therapies designed to halt diseases in their tracks or reverse their progress rather than simply manage symptoms. The company is collaborating on the cell and gene therapy frontier to bring this major leap in personalized medicine to patients with a variety of diseases, including genetic disorders and certain deadly cancers. Cell and gene therapies are grounded in careful research that builds on decades of scientific progress. Following key approvals of cell and gene therapies by health authorities, new treatments are being tested in clinical trials around the world.

About Novartis in CanadaNovartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. In 2019, the company invested $51.8 million in research and development in Canada. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 1,500 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. For further information, please consult

About Novartis globallyNovartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 110,000 people of more than 140 nationalities work at Novartis around the world. Find out more at

Zolgensma is a registered trademark of Novartis Gene Therapies.

Novartis Gene Therapies has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for thein vivogene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Gnthon forin vivodelivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self-complementary DNA technology for the treatment of SMA.


SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations, Julie Schneiderman, +1 514 633 7873, E-mail: [emailprotected]

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Health Canada approves Zolgensma, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA) - Canada NewsWire


What is gene therapy? – The Star Online

Tuesday, December 22nd, 2020

I have heard about people using genes to treat diseases nowadays, but I am not sure what this gene therapy means.

Gene therapy involves trying to alter or modify the genes inside your bodys cells in order to treat or stop a disease.

Since 2017, the US Food and Drug Administration (FDA) has approved three different types of gene therapy.

Maybe we can start at the beginning: what are genes?

Genes are the basic physical and functional unit of heredity.

Our genes are made out of DNA (deoxyribonucleic acid).

Each person has two copies of each gene one inherited from your mother and the other inherited from your father.

Each human being has around 20,000 to 25,000 genes.

These genes code for the way your body and mind are structured.

Some genes act as instructions (a blueprint) for your body to make various proteins, which in turn form your cells and organs, and the enzymes and hormones that regulate your body.

Other genes do not code for proteins.

Most genes are the same for all human beings, which is why we all look like human beings (and not a kangaroo, fish, bird or an alien)!

However, just under 1% of our genes vary slightly between each person.

That is why we have different races, heights, propensity for different diseases, curly or straight hair, etc.

These small differences also contribute to why we all look different from one another.

Genes that dont work as they should also cause diseases in the human body.

What types of diseases are caused by faulty genes?

These are what we call genetic disorders.

A genetic disease is any type of disease caused by an abnormality in our genetic blueprint.

This abnormality can range from very minor to significantly major.

What we consider minor is, for example, a small mutation in the DNA of a single gene resulting in the change of a single base protein.

What we consider major is a gross chromosomal abnormality, such as the addition of a whole chromosome or the subtraction of one.

Some genetic disorders are inherited from our parents.

Others are caused by mutations due to our environment.

Examples of single gene disorders, which are caused by the alteration of just one gene in our bodies, are:

Examples of multifactorial inheritance, which are caused by a combination of environmental factors and mutations in many of our genes, are:

If we inherited these genes from our parents, then how can we possibly modify or alter them? This sounds terribly like science fiction.

We are rapidly approaching that era where what used to be science fiction could become part of our everyday life.

In gene therapy, scientists can:

How do they do this? Do they have to harvest my cells? Im scared just thinking about it!

Many of the vectors are viruses, especially adenoviruses (not coronaviruses!).

Viruses have a natural ability to deliver genetic material into our cells.

After all, their main purpose is to attach themselves to cells and reproduce themselves.

Sometimes, the vector or virus is injected straight into our bodies, where they will deliver the gene that will modify our cells.

They are injected straight into the part of our body that has those defective cells.

Other times, we have to harvest healthy tissue from our body that needs to be modified.

These are usually tissues containing immune cells or stem cells, e.g. blood or bone marrow.

These tissue samples are then taken to the lab and specific cells are separated out.

The viral vector containing the corrective gene is then introduced to the harvested cells in the lab.

The modified cells are left to multiply, and then injected back into us.

Once inside our bodies, they will continue to multiply and eventually treat the disease or correct the defect within us.

Learn more about gene therapy in the next Tell Me About column on Dec 31 (2020).

Dr YLM graduated as a medical doctor, and has been writing for many years on various subjects such as medicine, health, computers and entertainment. For further information, email The information contained in this column is for general educational purposes only. Neither The Star nor the author gives any warranty on accuracy, completeness, functionality, usefulness or other assurances as to such information. The Star and the author disclaim all responsibility for any losses, damage to property or personal injury suffered directly or indirectly from reliance on such information.

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What is gene therapy? - The Star Online


Researchers discover new way to deliver DNA-based therapies for diseases – UMN News

Tuesday, December 22nd, 2020

University of Minnesota Twin Cities researchers in the Department of Chemistry have created a new polymer to deliver DNA and RNA-based therapies for diseases. For the first time in the industry, the researchers were able to see exactly how polymers interact with human cells when delivering medicines into the body. This discovery opens the door for more widespread use of polymers in applications like gene therapy and vaccine development.

The research is published in the Proceedings of the National Academy of Sciences (PNAS), a peer-reviewed multidisciplinary scientific journal.

Gene therapy involves altering the genes inside the bodys cells to treat or cure diseases. It requires a carrier that packages the DNA to deliver it into the celloftentimes, a virus is used as a carrier. Packaging of nucleic acids is also used in vaccines, such as the recently developed messenger RNA (mRNA) COVID-19 vaccine, which is enclosed in a lipid.

The research team is led by chemistry professor Theresa Reineke and associate professor Renee Frontiera. Reinekes lab synthesizes polymers, which are long-chain molecules that make up plastics, to use for packaging the nucleic acids instead.

Its kind of like ordering something from Amazon, and its shipped in a box, Reineke explained. Things get broken if theyre not delivered in a package. Thats basically what were doing here but on a nano-level. Were taking these really sensitive RNA and DNA cargo that are susceptible to enzymatic degradation, that wont get to their target unless you have something to protect them.

The researchers designed the copolymer using quinine, a naturally occurring substance used in tonic water, and 2-hydroxyethyl acrylate (HEA), which makes the material soluble and is used in a variety of personal care and medical materials. Because quinine is fluorescent, the research team was able to track the DNA package throughout the body and into the cells using Raman spectroscopy, a chemical imaging technique.

Weve discovered a new packaging tool with this natural product thats important for all of these high-flying, important fields like gene therapy and vaccines, said Reineke, who is also a Distinguished McKnight University Professor. And, it works in a variety of cell-types. On top of that, its got all of these cool featuresits fluorescent, we can track it, its Raman active, and that allowed us to understand a lot of fundamentals about these packaging systems that were impossible to probe before we incorporated this natural product.

Polymer-based drug delivery is significantly cheaper than using viruses, especially for gene therapy, which can cost up to $2 million for a single injection. However, the main barrier preventing widespread polymer use was that scientists didnt know a lot about how the polymer package actually interacts with cells in the body.

This research helps clear up that uncertainty. Frontieras lab specializes in chemical imaging. Using Raman spectroscopy, they discovered that a cells own proteins play a key role in unpacking the nucleic acid cargo once the polymer carrier enters the cell.

Its very satisfying to know how this is actually happening, what the process of delivery is, and to actually see that in real-time, Frontiera said. A key point is that these polymers also work very well. For all the beneficial attributes, theyre also incredibly effective at getting the payload into cells, and we were able to tell why, which doesnt always happen in this field.

Reineke and Frontiera have been collaborating since 2013. Reinekes lab has patented the quinine polymers, and the researchers hope that a company might license this technology in the future. The College of Science and Engineering team also collaborated with University of Minnesota Medical School Distinguished McKnight University Professor Jakub Tolar to test the effectiveness of the polymer carriers in relevant cell types.

Other members of the research team include chemistry researchers Craig Van Bruggen (Ph.D. student) and David Punihaole (postdoctoral associate), chemical engineering and materials science student Andrew Schmitz, and genetics Ph.D. student Allison Keith. This research was funded by the National Science Foundation and the National Institutes of Health.

Read the full research paper entitled Quinine copolymer reporters promote efficient intracellular DNA delivery and illuminate a protein-induced unpackaging mechanism on the PNAS website.

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Researchers discover new way to deliver DNA-based therapies for diseases - UMN News


ASH: UniQure/CSL hem B gene therapy curbs bleeding in phase 3even in patients with anti-AAV antibodies – FierceBiotech

Friday, December 11th, 2020

People with hemophilia B lack a protein that helps their blood clot, so they rely on lifelong infusions of that protein to manage their disease. UniQure and CSL Behrings hemophilia B gene therapy could transform chronic care into a one-time treatmentand its latest data suggest it could work for patients considered unsuitable for gene therapy.

The treatment, etranacogene dezaparvovec, curbed bleeding episodes and nearly eliminated the need for infusions of clotting Factor IX (FIX) in a phase 3 study testing it in severe or moderately severe hemophilia B. The study, HOPE-B, found that 26 weeks after treatment, the gene therapy had reduced bleeds that needed treatment by 91%, with 87% of 54 patients reporting such bleeds. Eighty-three percent of the patients reported no bleeds at all, including suspected bleeds that did not require treatment.

RELATED: BioMarin's hemophilia gene therapy 'Roc solid' after 4 years

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The treatment also boosted FIX activity to an average of 37.2%, up from less than 2% at the start of the trial, meeting its primary endpoint.

The study, to be presented virtually Tuesday at the annual meeting of the American Society of Hematology, found that nearly all the patients52 out of 54, or 98%no longer needed infusions of FIX to prevent bleeding episodes. Of the remaining two patients, one suffered an infusion reaction during treatment and did not get the full gene therapy dose, while the other had very high levels of antibodies that neutralize the adeno-associated virus (AAV) used to deliver the treatment.

Many people have a natural immunity to AAVs because theyre exposed to them in the environment. Though useful for fighting off infection, this immunity can render gene therapies ineffective. That appears not to be the case with uniQure and CSLs treatment. The patient for whom the gene therapy did not work was an outlier, with antibody levels nearly five times as high as the level expected in more than 95% of the general population. In patients with more typical antibody levels, investigators saw no correlation between the presence of those antibodies and FIX activity, the company said in a statement.

Importantly, these data also show that those patients in the trial who may not have been eligible for other gene therapies because they had pre-existing neutralizing antibodies (NAbs) have achieved results with etranacogene dezaparvovec that are comparable to the results of patients who did not have pre-existing NAbs, Steven Pipe, M.D., a professor of pediatrics and pathology at the University of Michigan and the studys lead investigator, said in a statement.

RELATED: CSL to pay $450M to buy uniQure's hemophilia B gene therapy

This is an important distinction as this is the only known clinical trial that has maximized patient eligibility in this way. The initial data also show that etranacogene dezaparvovec has been generally well tolerated to date, he said.

Most of the side effects were mild (82%). The most common treatment-related side effect was elevated liver enzymes, which affected 17% of patients but was treated effectively with steroids. Infusion reactions and flu-like symptoms each affected 13% of patients.

The data come five months after CSLpicked up the global rights to etranacogene dezaparvovec for $450 million upfront. These latest data set up the companies in a battle to bring a new hemophilia B gene therapy to market, pitting them against the likes of Pfizer and Spark/Roche.

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ASH: UniQure/CSL hem B gene therapy curbs bleeding in phase 3even in patients with anti-AAV antibodies - FierceBiotech


Bayer strengthens cell and gene therapy focus with new platform – PMLiVE

Friday, December 11th, 2020

Bayer has established a new strategic platform focused on cell and gene therapy, following its AskBio acquisition in October.

The new cell and gene therapy platform will house both AskBio and another of Bayers recently acquired firms, BlueRock Therapeutics, under one roof.

Bayer will seek to strengthen its own internal cell and gene therapy capabilities, with the newly established platform also enabling it to pursue external opportunities to bolster its presence in this area.

This is a defining moment for Bayer. Cell and gene therapies are leading innovation in healthcare, and it is our goal to be at the forefront of this revolution in science, said Stefan Oelrich, a member of the board of management, Bayer AG and president Pharmaceuticals Division.

Bayer paid an initial $2bn to gain full rights to AskBios gene therapy platform, which included an intellectual property portfolio and an established contract development and manufacturing organisation (CDMO).

AskBios adeno-associated gene therapy platform has already shown promise in a number of diseases, with the companys lead research programmes focused on Pompe disease, Parkinsons and congestive heart failure.

The German pharma company also bought out its private equity partner Versant Venture and founders in its cell therapy joint venture BlueRock Therapeutics for $240m.

BlueRock was established in late 2016 with $225m in start-up funding from Bayer and investment firm Versant, shortly after Bayer backed gene-editing specialist Casebia via its Leaps by Bayer investment arm.

The goal [of the platform] is to build robust platforms with broad application across different therapeutic areas, Bayer added in a statement.

The company also said that it has a vibrant cell and gene therapy pipeline with five advanced assets, as well as over 15 preclinical candidates.

The emerging bio revolution represents a once-in-a-lifetime opportunity and a new era for Bayer, said Wolfram Carius, head of Bayers new cell and gene therapy platform.

A dedicated C> Platform is vital to accelerate innovation at its source, and to ensure its translation into tangible therapies for patients who have no time to wait, he added.

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Bayer strengthens cell and gene therapy focus with new platform - PMLiVE


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