StemCell Therapy

How prepared do you think we were for this moment of social distancing and for this global moment of hunkering down amidst uncertainty?In terms of social distancing, weve been social distancing; weve been virtualizing our lives since at least the advent of the telegraph in the 19th century. We have this idea of distance even now, where were communicating from away and communicating to others. But we also, as humans, have this deep need for physical connectivity. We are not virtual beings. And so, emotionally, were not ready for it. All of these structures for physical connectivity are gone, at least temporarily. Were almost in this Battlestar Galactica remake moment where were having to reconceptualize space and community. Its not that we will become un-physical beings, but were gonna have to figure out different ways of virtually sharing emotion and connectivity, at least until this danger passes.

Thats why organizations like PEN that are so focused on values are so critical, because these are the conversations that we have to have. Were going to have this incredible technology, but its up for grabs whether these technologies will be used to help or harm us.

You wrote at CNN.com about the human need for intimate physical connectivity. Can technology be a substitute for that? It seems like probably not.It cant be a substitute, but it can be a complement. And again, in our best possible world, I, for one, would love to live in some kind of hippie commune with real people there, and I also live, like many people, this global life where my friends and contacts are distributed around the world. I think we need to find that balance. But at times like this, our lives are becoming and feeling more virtual. And yes, theres a loss, and I think many of us are mourning that loss. But this is the world that we have now, and we have to make the most of it. Theres a lot of simple things that people can do. Make a list of all the people who you love and care about in your life, all the people who you think may be feeling isolated or alone, and just create a schedule of reaching out to them. My girlfriend and I are doing a virtual tea party with friends on Sunday where were gonna make tea, theyre gonna make tea, were gonna connect on FaceTime. We have to think of how we might do things differently. But its also not the case that when this crisis ends, society is just going to snap back to where it was, and were going to say, Wow, that was a crazy experience. Theres something happening now that is going to last beyond this.

What are some things that could be irrevocably different about our culture and the way we work and live, as a result of this moment?Were for sure not going fully back on virtualization. Were going to do things differently. Our sense of space is going to be different. A lot of people who are now working from home arent going to go back to physical offices because once companies figure out how they can work in this way, itll just be cheaper to have people stay at home. Were certainly going to change the way we think about global public health. If you asked a regular person, Wouldnt it make sense to have a super empowered World Health Organization with a global surveillance system that whenever any trip wire was hit, youd have an emergency response team that would fly to wherever that was and they would set up a command center and do what needed to be done? They would say, Yeah, dont we have that? And the answer is we dont. Because we have starved organizations like the WHO, because we have states that are demanding a level of control that doesnt make sense in our world of global challenges. One of the things that Im working on very, very actively now is imagining a third leg of the global political stool in addition to states and international institutions, and that is the democratic expression of the needs of our common humanity. It seems like its this big, crazy idea. But in these negotiations, no one is saying, Hey, climate change affects all of us; destroying our oceans affects all of us; global pandemics affect all of us. Who is standing up to help humanity? And thats what I think we need now.

I feel like Im at war from the battlestation of my office here on 81st Street in New York, so Im pretty focused on reading what I need to read now.

In Hacking Darwin, you wrote about genetics, you wrote about changing our genetic identity, perhaps to yield cures for diseases. Are you more or less optimistic about the potential for genetic science and cures than you were before?Im extremely optimistic. We are facing an enormous challenge today, but we now have almost godlike capacities to read, write, and hack the code of life. And those tools, Im firmly convinced, are going to save us, and were going to figure out treatments and were gonna have a vaccine not just for this, but for all kinds of challenges in the future. But these technologies dont come with a built-in value system. All technologies are value-neutral. Its up to us to determine what are the values that will guide the application of our most powerful technologies, and thats the issue. Thats why organizations like PEN that are so focused on values are so critical, because these are the conversations that we have to have. Were going to have this incredible technology, but its up for grabs whether these technologies will be used to help or harm us.

Finally, what are you reading, watching, or listening to right now?I would advise people at times of crisis like this to read poetry and literature. Im trying to do a little bit of that, but Im just all in and obsessed. Just last night I finished this incredible book, Spillover, by the amazing journalist David Quammen. And thats about zoonotic viruses like this, and our experiences in the past. Im now reading Betrayal of Trust by Laurie Garrett, which is about the destruction of our public health infrastructure. So when this is done, Im just going to beand I myself am a novelistback to reading the novels that I love so much. Maybe Ill read Proust and start thinking about Maman and her madeleine. But for now, I feel like Im at war from the battlestation of my office here on 81st Street in New York, so Im pretty focused on reading what I need to read now.

Wed like to know what books youre reading and how youre staying connected in the literary community. Click here to leave a voicemail for us. Your message could end up on a future episode of this podcast!

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The PEN Pod: Reimagining the Future with Jamie Metzl - PEN America

The quarter was brutal to say the least for most asset classes amid the coronavirus (COVID-19) pandemic. The S&P 500 Index was down about 20% for the quarter.

Amid the market mayhem, some health care stocks defied the downturn, thanks to announcements concerning development of drug/vaccine/diagnostic tests for new coronavirus.

Focusing on biotechs (leaving out diagnostics stocks), here the top five winners and losers for the quarter.

Benzinga is covering every angle of how the coronavirus affects the financial world.For daily updates,sign up for our coronavirus newsletter.

Genprex Inc (NASDAQ: GNPX): (+656.25%)

After ending 2019 in penny stock territory, shares of this gene therapy company began to gain ground after the company announced Fast Track Designation for its immunogene therapy in combination with AstraZeneca's(NYSE: AZN) Tagrisso for treating lung cancer. The momentum accelerated after it signed an exclusive agreement to license a diabetes gene therapy from the University of Pittsburgh.

After topping $7 in late February, the stock came off the highs amid the COVID-19 sell-off and managed to end the quarter with huge gains.

Vaxart Inc (NASDAQ: VXRT): (+405.71%)

Vaxart is a COVID-19 play and much of the quarter's gains were achieved on the back of the experimental oral vaccine candidate it's developing in partnership with Emergent Biosolutions Inc (NYSE: EBS).

Ibio Inc (NYSE: IBIO): (+324%)

Ibio, which develops human therapeutic proteins using advanced genetic engineering, joined the fray for a COVID-19 vaccine, which explains the surge in the stock.

Novavax, Inc. (NASDAQ: NVAX) (+241.2%)

Novavax was the beneficiary of dual catalysts: a COVID-19 vaccine in development and positive late-stage readout for its flu vaccine.

Trillium Therapeutics Inc (NASDAQ: TRIL): (+192.23%)

Thisimmuno-oncology company did not have much developments to justify its gain for the quarter.

Following a jump of about 63% in a single session in late February, the company issued a statement thatsaid "it is not aware of any material, undisclosed information related to the company that would account for the recent increase in the market price and level of trading volume of its common shares."

Related Link: Attention Biotech Investors: Mark Your Calendar For These April PDUFA Dates

Milestone Pharmaceuticals Inc (NASDAQ: MIST): (-88.51%)

This cardiovascular-diseases-focused biopharma was cruising along fine until COVID-19 sell-off started in March. The real punch came from an adverse clinical readout.

Novan Inc (NASDAQ: NOVN): (-84.97%)

Novan, which leverages on nitric oxide's naturally occurring anti-microbial and immunomodulatory mechanisms of action to treat various diseases, fell steeply at the start of the year. The trigger was a late-stage readout of its SB206 in molluscum contagiosum, which showed that the pipeline asset did not achieve statistically significant results for the primary endpoint.

The stock did not recover from this onslaught.

Acasti Pharma Inc (NASDAQ: ACST): (-84.49%)

Acasti also succumbed to a negative clinical readout for its lead prescription drug candidate CaPre, which did not achieve statistical significance for the primary endpoint of a late-stage study that evaluated it for treating elevated levels of triglycerides.

The company is now seeking FDA guidance for unblinding data from another Phase 3 study, and therefore expects a delay in reporting of topline results until the third quarter.

Salarius Pharmaceuticals Inc (NASDAQ: SLRX): (-81.98%)

This oncology-focused biotech gradually declined through the quarter, with some steep sell-off materializing amid its presentation at the BIO CEO & Investor conference in mid-February.

Amarin Corporation plc (NASDAQ: AMRN): (-81.34%)

Amarin shares, which ran up ahead and after the late-December FDA verdict on its application seeking label expansion for its fish oil pill, pulled back in January. The weakness intensified through the market meltdown. A negative court ruling sent the stock reeling this week.

2020 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

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10 Biotech Winners And Losers In Q1 - Benzinga

In the field of prevention, the work of Mexican molecular medicine researcher Laura Palomares stands out. And today, her team is developing a vaccine against SARS-CoV-2, based on the work they have been doing in recent years against dengue and zika.

I am convinced that the only way that we are going to be able to respond to this type of pandemic in a timely manner is going to be using platforms. I am referring to a vaccine, for which we already have the entire production, development, stability train, etc. , said the chemical engineer from the Instituto Tecnolgico y de Estudios Superiores de Monterrey (ITESM), that holds a masters in Biotechnology, and a doctorate in science from UNAM.

Many times we think that the laboratory is going to discover a vaccine to cure the patient, and it is not like that. This type of vaccine requires a lot of time and a lot of effort in developing the processes for production and characterization, before reaching the final patient, Laura Palomares added.

With this idea in mind, the also researcher at the Institute of Biotechnology (IBt) of UNAM has promoted the development of one of these technological and methodological platforms focused on the aforementioned Zika and Dengue viruses, conditions particularly significant for Mexico due to their high numbers of contagion, every year in different parts of the country.

The result has been a vaccine created with recombinant DNA technology, which Palomares calls a chimera.

Lets put it in simple words, for people to understand: If we take away from the platform the zika and dengue viruses, and we put the coronavirus there, that way we can get a vaccine against SARS-Cov-2, says the member of the University Commission for Attention of the Coronavirus Emergency.

What took us two years in genetic engineering, adding on and taking off proteins, understanding how these capsids were going to be assembled, characterizing them, etc., all that we had already done. So now, we are replacing that with SARS-CoV-2, and that is precisely why we have advanced so much right now , Palomares continued.

The approach to the development of vaccines through platforms has also been the route taken by two vaccines against Covid-19 in the world that are currently under clinical evaluation: that of the North American company Moderna and that of the Chinese company CanSino Biologics, stated the expert.

The coronavirus vaccine is in the testing phase in animal models, a process in which the Zika and dengue vaccine has already been evaluated. If everything progresses positively, Palomares estimates that the first human tests could be carried out in three years.

In the case of the SARS-CoV-2 vaccine that she and her team are currently developing, they plan to collaborate wth the Mexican company Liomont, which has a manufacturing plant that would allow the production of this vaccine, this way Mexico does not have to depend on transnational companies.

So this pandemic is obviously terrible for us, because it is affecting the health of a large part of the population, but also a great opportunity to raise awareness, the researcher concluded.

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Mexico is already testing its own Covid-19 vaccine - The Yucatan Times

Pharma and biopharma companies dont want to work on their relationships. These companies want their relationships to work for them. Fair enough. Still, when it comes to relationships with contract manufacturing and drug development organizations (CDMOs), pharma and biopharma companies cant avoid intimacy. These relationships are rewarding to the extent pharma and biopharma companies are clear about their needs and the kinds of associations consistent with those needs.

Typically, the most pressing need is keeping costs down. But other needs are important, too. These include expertise with specialized technologies, challenging drugs, and complex formulations. Identifying needs will help pharma and biopharma companies decide whether their CDMOs should help with discrete activities or provide complete, end-to-end services. Pharma and biopharma companies may also want their CDMOs to help with business strategy and regulatory compliance.

Besides identifying potentially compatible CDMO partners, pharma and biopharma companies need to build CDMO relationships characterized by mutual trust. Here again, clear communication is essential. It can help both parties in a CDMO relationship resolve misunderstandings and overcome unanticipated challenges together. For a fuller discussion of the ways CDMO relationships can benefit from clear communication, see the rest of this article, which presents various pointers and perspectives from CDMO experts.

Thierry Cournez is head of end-to-end solutions at MilliporeSigma, which offers a comprehensive portfolio of high-quality products and services, including testing services, for biopharmaceutical development. According to Cournez, most emerging biotech companies that have very early data and need to take their molecule to commercialization dont have all the expertise in-house that they need to navigate the entire process. One major trend is complete services, as opposed to la carte offerings.

MilliporeSigmas Plug & Play Upstream Development Service eliminates the need to work with multiple vendors for upstream development, relieving bottlenecks and reducing time to clinic. The service covers cell line development from DNA transfection to cell banking. Process development activities, which run in parallel, start when the company receives material from the first clones.

Cournez says that two services that will continue to be important in pharmaceutical development are process development and analytical services. A robust process is critical for manufacturing success, he explains, and analytical services are the foundation that supports the entire life cycle of biologics.

A trend toward all-in-one CDMO services has also been observed by Richard Shook, director of drug product technical services and business integration at Cambrex. Any time a client has to go to multiple vendors, it creates a lot of seams and communication problems, he points out. A lot of dots are not connected. Critical items can be lost, especially internal knowledge. When a client works with a single vendor, he stresses, the partners create a knowledge base that can be carried forward with the project. Cambrex provides drug substance, drug product, and analytical services across the entire drug lifecycle.

Fujifilm Diosynth Biotechnologies (FDB) is a division of Fujifilm that focuses on biopharmaceutical contract manufacturing, especially drug substances for biologics. That includes cell culture and fermentation, development and manufacturing, and advanced therapies like gene therapy. Fujifilms director of strategic business development, Daniel DeVido, PhD, says there is growing interest in gene therapy products and gene modified cell therapies.

In the area of viral vectors, new products on the market such as Luxturna (from Spark Therapeutics) and Zolgensma (from Novartis) have moved that sector of the industry forward. Newly approved chimeric antigen receptor (CAR) T-cell therapies, such as Kymriah (from Novartis) and Yescarta (from Kite Pharma) are also injecting energy into the field. And monoclonal antibodies have been going strong for the last 10 to 15 years, with approximately 80 therapies approved and on the market.

The industry is well funded right now, DeVido points out. A lot of companies are pushing candidates forward.

That increased demand for cell culture services brings new technical challenges. Everybodys looking for increased titers, DeVido emphasizes. For gene therapy, yields and titers are much less than they are for cell culture, so everybodys looking for the next thing that will get gene therapy to produce on the scale that monoclonal antibodies are on now.

Lonza offers a range of CDMO services. Karen Fallen, the companys head of mammalian and microbial development and manufacturing, says that Lonza works with companies from several different segments, including small and virtual companies that have limited in-house resources and capabilities. A lot of them are really focusing on the science, she notes. Theyre looking for preclinical and clinical services.

In Lonzas view, some of the trends among the smaller companies are due to larger Series A financings. In past years, Series A deals would have been $10 or $15 million, but now they are running higher, up to $70 or $80 million. They have different ambitions now, Fallen points out. They want and are able to take the molecule further along the supply chain, even to launch. They want to stay with Lonza longer before they partner up with large pharma and/or out-license these molecules. She adds that Lonzas customers also have more complex molecules in their pipelines.

Lonzas other big segment consists of large pharma companies. They have assets, and they have experience, she says. What theyre looking for now is newer technologies, with newer modalitiesbioconjugation, highly potent small molecules, or cell and gene therapies, for example.

Almac Group provides an extensive range of contract development and manufacturing services across the drug development life cycle. The increased interest in pediatric formulations is driving a demand for mini-tablets, especially those in stick-pack dosage form. The rapidly expanding oncology space, by its nature, creates a need for CDMOs that have extensive capabilities in processing highly potent active pharmaceutical ingredients at the small-to-medium scale.

Were seeing an industry trend toward higher value, lower volume products, says Jonas Mortensen, vice president of business development at Almac. Our clients are asking us to take on commercial supply of their product, often at, or close to, the same scale we had previously provided for their clinical studies.

To meet these new needs, Almac has installed multiple stick-pack machines across its sites in the United Kingdom and the United States. Almac is also finalizing the qualification of a dedicated suite of eight processing rooms and equipment solely designed for, and dedicated to, processing of highly potent active pharmaceutical ingredients.

Mortensen anticipates that some near-term trends in CDMO services will include supply chain risk mitigation, end-to-end services, and GMP floor space. CDMOs, he points out, are increasingly being asked to demonstrate their ability to support multisite supply strategies through global facilities or act as a secondary site of manufacture.

Communication is a common theme when it comes to recommendations for working with a CDMO. MilliporeSigmas Cournez says that biotech companies should choose a CDMO that has the most experienced people in-house. Doing so can help biotech companies avoid having to deal with multiple vendors. He also recommends having a dedicated project manager who can provide transparent communication with the vendor and connect with subject matter experts in case of unexpected changes.

Good communication also contributes to transparency in a project. Project transparency is really important, insists Cambrex Shook. That can be limited due to the competitive landscape of the project.

If problems arise during project execution, ownership and communication is really important. If its not there, losses occur and there are timeline setbacks. This could impact the scope, and once you get off scope, [it takes] money and resources to get back on track.

An illustration of the importance of communication comes from Catalent, a company that offers a range of CDMO services, including its recently introduced GPEx Boost technology for cell line development. Michael Riley, vice president and general manager of biologics at Catalent, says that in a program the company is currently working on, a customer was on a highly accelerated path to a product filing for a fast-track product. Catalent was working with regulatory authorities to characterize the companys manufacturing process and move toward validation of that process. To do that, Riley explains, we had to have very robust conversations between multiple functions within our organization and their organization from a quality and development standpoint.

Trust can be a delicate issue in relations between a CDMO and a customer. To illustrate this point FDBs DeVido describes a face-to-face discussion that the company had with one of its customers. This discussion, which took place in FDBs office in Cambridge, MA, resolved some contractual disagreements. We were able to sit around the table and go through the legal issues, DeVido recalls. We cleared up a lot very quickly.

When you sit down face to face and have good discussions, he says, everyones a little more comfortable. Even though people may feel theyre not completely safeguarded from a one-in-a-million occurrence, they may feel comfortable the two parties are going to work together through whatever the issue is.

DeVido said that once youve selected a CDMO, its important to be transparent and trust the company. Youve done your due diligence, he proposes. Now trust your selection and the system theyre operating in.

Benefits of working with an experienced vendor can go beyond development and manufacturing. Cournez says that in one instance, an emerging biotech customer had the opportunity to engage in licensing discussions with a large pharmaceutical company. Because the emerging biotech was small, we hosted the large pharma company at one of our sites and ran the due diligence, which was a great success, he relates. This former emerging biotech now funds many different programs because of the success of their first molecule.

Some vendors warn that business strategies can backfire. Focusing too much on price and speed to market can be risky when researching or working with CDMO/CMO partners, according to Cournez. The service provider must simplify the process and reduce touchpoints throughout the process.

Mortensen says that due to the significant investment required in resource and training, many smaller biopharma companies often do not have a regulatory affairs department of their own. Therefore, its critical for sponsors to recognize the consultative benefit CDMOs bring to the table as an extension of their company to help fill in any regulatory knowledge gaps. This timely advice, integrated with early- and late-stage development, can enable a sponsor to adequately prepare, ensuring little or no delay when bringing its products to market.

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Making the Most of a CDMO Relationship - Genetic Engineering & Biotechnology News

Science writer, New Yorker contributor and author of the book Denialism Michael Specter joins Felix Salmon on the Slate Money podcast to break down the ongoing coronavirus crisis.

Specter explains how the virus spreads, potential food and medicine shortages it could cause and the possibility of developing immunity to infection. While the pandemic has shocked most of the world, Specter argues the only thing that should surprise anyone is the inept response of policy makers to the outbreak, particularly in the United States.

Specter also challenges some common misconceptions surrounding biotechnology, including the idea that GMOs are unnatural. Concerns about monoculture, the practice of growing a single crop like corn, on the other hand, are valid, Specter says. But that issue has nothing to do with genetic engineering. Its a problem that could be solved by a change in government policies: ending subsidies to corn and soybean growers. However, there are trade offs involved, and eliminating monoculture farms isnt the simple decision it seems.

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Podcast: Science writer Michael Specter on what you should know about the coronavirus, food security and GMOs - Genetic Literacy Project

NASHVILLE, Tenn., April 1, 2020 /PRNewswire/ --Concert Genetics, a technology company dedicated to advancing precision medicine, today announced the publication ofreal-world data on utilization and coding variability in medical claims for Next-Generation Sequencing (NGS)-based diagnostic tests. The study was done in collaboration with Merck, known as MSD outside the United States and Canada, and focuses on diagnostic testing among cancer patients in the U.S. It was accepted for presentation in the General Poster Session at the National Comprehensive Cancer Network's NCCN 2020 Annual Conference and is available online from JNCCNJournal of the National Comprehensive Cancer Network.

"The breathtaking speed of innovation in precision medicine is outpacing the healthcare system's ability to adapt," said Rob Metcalf, CEO of Concert Genetics. "As a result, the real-world data for observational research is often unavailable, too sparse, or insufficiently granular for evidence development. Concert's focus is delivering transparency and connectivity in genetic testing to enable precision medicine, and we were delighted to utilize our proprietary data and patented analytics to make this research possible."

The joint abstract is titled "Real-World Utilization and Coding Variability in Medical Claims for Next-Generation Sequencing (NGS)-Based Diagnostic Tests Among Cancer Patients in the U.S." It was scheduled to be presented at NCCN 2020, which was postponed due to COVID-19. The abstract is available at the following URL: https://jnccn.org/view/journals/jnccn/18/3.5/article-pHSR20-083.xml.

Concert's proprietary method for collecting and analyzing data in this space is described in U.S. Patent No. 10,223,501: "Tracking, Monitoring, and Standardizing Molecular and Diagnostic Testing Products and Services."

About Concert GeneticsConcert Genetics is a software and managed services company that advances precision medicine by providing thedigital infrastructure for reliable and efficient management of genetic testing. Concert's market-leading genetic test order management platform leverages a proprietary database of the U.S. clinical genetic testing market (today more than 140,000 testing products) and integrates with leading electronic health record and laboratory information management systems. Concert also provides genetic testing management solutions to leading health plans across the U.S. Learn more at http://www.ConcertGenetics.com.

CONTACT

Nick Tazik Concert Genetics (615) 861-2634 ntazik@concertgenetics.com

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Concert Genetics Presents Real-World Data on Utilization of NGS-Based Diagnostic Tests in NCCN 2020 Abstract - Yahoo Finance

A new study comparing genetics and the neural wiring of the brain suggests a diagnosis of attention-deficit/hyperactivity disorder (ADHD) results from a combination of factors. Investigators discovered that it takes many common genetic variations combining together in one individual to increase risk substantially.

At the same time, neuroimaging (MRI) experts have found differences in how the brains of people diagnosed with ADHD are functionally connected. However, its unclear how genetic risk might be directly related to altered brain circuitry in individuals diagnosed with ADHD.

In the new study, researchers focused their imaging analyses on selected brain regions, looking specifically at the communication between those regions and the rest of the brain in children with the diagnosis.

They discovered that one brain regions connectivity was linked to a higher risk of ADHD. However, a second, different part of the brain seemed to compensate for genetic effects and reduce the chances of an ADHD diagnosis.

The authors believe this research will lead to a better understanding of how genetic risk factors alter different parts of the brain to change behaviors and why some people at higher genetic risk do not exhibit ADHD symptoms.

The study appears in Biological Psychiatry: Cognitive Neuroscience and Neuroimaging.

We are now in a phase with enough data to answer some questions about the underlying genetics of a disorder that in the past have been difficult to elucidate, said senior author Damien Fair, Ph.D.

Previous imaging studies had shown different functional connectivity, and we assume those have a genetic basis.

ADHD is a neurodevelopmental psychiatric disorder that affects about 5 percent of children and adolescents and 2.5 percent of adults worldwide. The disorder is characterized by inattentive or hyperactive symptoms with many variations.

The paper focuses on 315 children between the ages of 8 and 12 who participated in a longitudinal ADHD study that began in 2008 at the Oregon Health & Science University in Portland. Fair, a neuroscientist and imaging researcher, and co-author Joel Nigg, Ph.D., a pediatric psychologist participated in the study. Robert Hermosillo, Ph.D., a postdoctoral researcher in Fairs lab, led the study.

The research team selected three areas of the brain based on a brain tissue database that showed where ADHD risk genes were likely to alter brain activity. To measure the brain communication to-and-from these regions on each side of the brain, the researchers used resting-state non-invasive magnetic resonance imaging (MRI) scans.

To begin to bridge genetic and neuroimaging studies of ADHD, researchers used MRI to scan the brains of children. Two regions previously associated with ADHD stood out. In one, a higher ADHD genetic risk correlated with a more active brain circuit anchored by the nucleus accumbens (orange arrow). Interestingly a weaker connection anchored by the caudate nucleus (blue arrow) seemed to protect children at high genetic risk from ADHD behaviors.

Next, they calculated a cumulative ADHD genetic risk score in the children, based on recent genome-wide studies, including a dozen higher-risk genetic regions reported two years ago by a large international collaboration called the Psychiatric Genetics Consortium.

In one brain region anchored by the nucleus accumbens, they found a direct correlation with genetics. Increased genetic risk means stronger communication between the visual areas and the reward centers, explained Hermosillo.

Another brain region anchored by the caudate yielded more puzzling results until the researchers tested its role as a mediator between genetics and behavior.

The less these two regions talk to each other, the higher the genetic risk for ADHD, said Hermosillo. It seems to provide a certain resiliency against the genetic effects of ADHD. Even among those with high risk for ADHD, if these two brain regions are communicating very little, a child is unlikely to end up with that diagnosis.

A third region, the amygdala, showed no correlation between connectivity to the other brain regions and the genetics.

According to the authors, the findings suggest that a genetic score alone will not be enough to predict ADHD risk in individuals because the results show both a genetic and neural contribution toward an ADHD diagnosis.

A future diagnostic tool will likely need to combine genetics and brain functional measures. The brain is not at the mercy of genes, added Hermosillo. Its a dynamic system not preprogrammed for disorders. It has the capacity to change.

Source: Elsevier

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Study Probes Interaction of Genetics and Neural Wiring in ADHD - PsychCentral.com

Yann Joly, Research Director of the Centre of Genomics and Policy

Research Director of the Centre of Genomics and Policy and Associate Professor at the Department of Human Genetics, Yann Joly is a Lawyer Emeritus from the Quebec Bar and a Fellow of the Canadian Academy of Health Sciences. He is currently a member of the Quebec task force on theCOVID-19 Biobank.

Last week, Joly and his collaborators from 16 countries announced the establishment of the international Genetic Discrimination Observatory (GDO). A world first, the GDO is an online platform committed to preventing the misuse of a patients genetic information. This is particularly important within the current context of the COVID-19 pandemic when researchers are collecting samples and data from patients in order to better understand this new disease and develop effective vaccines or therapeutics.

In this Q&A, Joly gives readers more information on genetic discrimination and what is being done to combat it.

Genetic discrimination (GD) means treating people differently from the rest of the population or unfairly profiling them based on actual or presumed genomic and other predictive medical data. The genetic information contained in an individuals DNA can uniquely identify or provide some information about a person, including future probabilities that this individual will develop diseases. Other predictive health information, such as biomarkers, can also be used to discriminate and should also be considered under the GD heading.

This information can be of interest to third parties like insurers, employers, or government officials. Like sexual, ethnic or disability-based discrimination, genetic discrimination is a source of exclusion and can limit the social and professional opportunities of a person thus becoming a source of psychological distress.

There are documented cases of GD reported in studies carried out in a limited number of countries based on predictive test results and family history for a handful of severe single-gene conditions in the context of life insurance or employment. The available evidence is fragmentary, and the methodology used in many studies is inconsistent.

The Genetic Non-Discrimination Act (hereinafter S-201) was passed in April 2017 and is currently applicable in Canada. While it does not solve all the challenges posed by genetic discrimination, it is an important first step. The Act generally makes it a criminal offense to require a person to undergo a genetic test or to report the results as a condition precedent to the provision of goods and services. However, the Quebec Court of Appeal recently declared that the core elements of S-201 were not constitutionally valid.

This decision was appealed to the Supreme Court of Canada and we are currently waiting for their decision on the matter. In the meantime, S-201 continue to be applied. If the Supreme Court is of a similar opinion to that of the Court of Appeal, it could be invalidated.

In addition to the protection provided by S-201, Canadian privacy laws would fully apply to genetic data, which is considered personal information.

Genetic information is increasingly shared across national borders or transcending them, thus limiting the effectiveness of protections built solely around national approaches. Strictly legal solutions, because they tend to be static, are also challenged to keep pace with rapidly evolving science such as genetics.

At its core discrimination is a social phenomenon that needs to be addressed collaboratively and internationally by all stakeholders. The GDO will provide the platform to undertake this important work, which will include documenting instances of genetic discrimination, identifying most effective preventing measures and conveying information, tools and good practices to all stakeholders including the public.

COVID-19 presents Quebecers with an unprecedented health threat that requires us to stand together as a society and take action to protect one another and help find medical solutions to the disease. The COVID-19 Biobank provides us a unique opportunity to learn more about the biological foundations of the disease, individuals at risk and preventive solutions.

The risk of discrimination associated with providing a biological sample and medical information to the Biobank is very small. The data provided is research information that is not clinically validated and should be of no interest to most third parties. Moreover, the collected information is coded, and protected by confidentiality laws and robust security measures. Furthermore, data access will be subject to ethics approval and in some cases controlled access measures.

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Yann Joly on the fight against genetic discrimination - McGill Reporter

MELBOURNE, Australia, April 01, 2020 (GLOBE NEWSWIRE) -- Molecular diagnostics company Genetic Technologies Limited(ASX: GTG; NASDAQ: GENE, Company), provides the following update to the market.

Colorectal test sales commence

As announced to the market on 31 January 2020, the Company has fully commissioned its Australian Laboratory for the provisioning of its generation 3 breast cancer test (GeneType for Breast Cancer), with sales having commenced during the quarter. We are pleased to report that this is now accompanied by the first-to-market GeneType for Colorectal Cancer test with sales also having now commenced.

Medical practitioner support

Both tests have been well received by medical practitioners, with over 15 clinics in Australia having agreed to offer the GeneType portfolio of tests. Overall, in excess of 100 test kits having been requested and approximately half of those samples have been received into the Melbourne laboratory during the first 4 weeks on market.

High risk breast cancer patients detected

An interim analysis of those patients who received the GeneType for Breast Cancer test in Australia has revealed that 4 patients have already been identified as potentially being at high risk of developing breast cancer. It is highly significant that these patients were not eligible for hereditary mutation testing and would not have been identified as high risk during routine health checks.

Clinical utility and reimbursement initiatives Genetic Technologies continues to progress its in-house R&D program in support of the tests. Data derived from large scale cohort studies, have confirmed that both the discrimination and calibration of both GeneType tests are excellent and set the stage for progressing our plans to develop clinical utility evidence in support of our reimbursement strategy.

Clinical utility studies have been designed to support both the breast and colorectal cancer tests and the Company is currently engaging with potential clinical collaborators to complete these studies at the earliest opportunity. As alluded to above, the evidence is building that the GeneType tests have the potential to personalize individual screening recommendations.

Tele-health and Consumer Initiated testing to relieve practitioner stress and opens access to Mass consumer markets

Notwithstanding the above, the Company is taking an aggressive and pro-active response to the current COVID-19 pandemic. In light of world-wide recommendations on social distancing, which is impacting on our ability to fully engage with physicians, we have brought forward our plans to introduce a Consumer Initiated Testing (CIT) Platform. The Company is in the final stages of choosing an independent provider network who will oversee patient ordering of the consumer-initiated testing (CIT) pipeline. This sales pipeline deviates from a traditional sales approach that targets clinicians and instead allows patients to request a test directly, with clinician oversight of the testing process through an independent provider network and telemedicine. When a consumer is interested in purchasing a GeneType product, a clinician from this independent network will review the patient health history before confirming whether or not the patient may proceed with testing. The independent clinician will then order the test for the patient and will also review the final test results prior to delivering them back to the patient

In an age where primary care clinicians are overburdened by large patient volume and thus restricted to short clinical visits, certain disease risk assessments are often overlooked, particularly if the patient is asymptomatic, or visually healthy.

The current push to adopt telemedicine as recently announced by Australias minister for Health Minister Greg Hunt is exactly on point with GTGs CIT platform strategy and has been in development by GTG over the past 3 months.

Personalized proactive approach to health

Understanding a patients risk of developing disease can lead to earlier screening, more frequent and alternative surveillance methods, options for risk-reducing medications, and at the very least, some suggested lifestyle changes. However, in order to be proactive about screening and prevention measures, patients and physicians alike, must understand the patients risk of developing disease. By allowing our GeneType risk assessment product lines to be requested via CIT, we are enabling a patient to take a proactive approach to his/her healthcare.

Dr George Muchnicki Acting CEO and Justyn Stedwell Company SecretaryOn behalf of the Board of DirectorsGenetic Technologies Limited+61 3 9412 7000

About Genetic Technologies Limited

Genetic Technologies Limited (ASX: GTG; Nasdaq: GENE) is a diversified molecular diagnostics company. GTG offers cancer predictive testing and assessment tools to help physicians proactively manage patient health. The Companys lead products GeneType for Breast Cancer for non-hereditary breast cancer and GeneType for Colorectal Cancer are clinically validated risk assessment tests and are first in class.

Genetic Technologies is developing a pipeline of risk assessment products.

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

Investor Relations and Media (US)Dave Gentry, CEORedChip CompaniesOffice: 1 800 RED CHIP (733 2447)Cell: US 407 491 4498dave@redchip.com

Caution Regarding Forward-Looking Statements

This press release contains forward-looking statements Words such as may, should, could, would, predicts, potential, continue, expects, anticipates, future, intends, plans, believes, estimates, and similar expressions, as well as statements in future tense, often signify forward-looking statements. Forward-looking statements should not be read as a guarantee of future performance or results and may not be accurate indications of when such performance or results will be achieved. Forward-looking statements are based on information that the Company has when those statements are made or managements good faith belief as of that time with respect to future events, and are subject to risks and uncertainties that could cause actual performance or results to differ materially from those expressed in or suggested by the forward-looking statements. The Company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise.

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Genetic Technologies Limited Announces Market Update on Sales, Early Test Results and Launch of Consumer Initiated Testing - BioSpace

Aaron Grech April 1st, 2020 - 8:11 PM

The godfather of heavy metal Ozzy Osbourne is making mind blowing health progress according to his daughter Kelly Osbourne, who recently spoke to Entertainment Tonight. The performer was receiving stem cell treatment recently, to help support his battle against complications caused by Parkinsons disease.

Seeing after one treatment of stem cell what has happened and the progress that hes made is mind-blowing, Kelly Osbourne stated. He wants to get up. He wants to do things. He wants to be part of the world again. Hes walking better. Hes talking better. His symptoms are lessening. He is building the muscle strength back that he needs after his spine surgery.

Osbourne revealed that he had Parkinsons disease at the beginning of this year during an interview withGood Morning AmericasRobin Roberts. Kelly Osbourne also explained that the her father has been feeling better as a result of his treatment, although he is still forced to stay inside due to the fears associated with the spread of COVID-19.

Everything is starting to fall into place now and it has given us so much hope, she explained. We are very grateful to the doctors that are helping him. Hes ready to get out of the house and now he cant get out of the house. He keeps saying to me, Ive been on quarantine for almost two years, and now Im feeling better and the world is on quarantine.

Osbourne has faced a slew of health complications in recent years, froma bout ofpneumonia, to a stage injury which requiredintense surgery.He has had to cancel multiple tours during the past couple of years due to his health concerns although he was in the studio last year to record his latest studio album release Ordinary Man.

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Ozzy Osbournes Daughter Kelly Reports the Singer Is Making Mind Blowing Progress After Stem Cell Treatment - mxdwn.com

Significant Clinical Progress Across Target Indications during 2019 and 2020 Year-to-Date

NEW YORK and LONDON, March 31, 2020 - Akari Therapeutics, Plc (Nasdaq: AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where complement (C5) and/or leukotriene (LTB4) systems are implicated, today announced financial results for the fourth quarter and full year ended December 31, 2019, as well as recent business highlights.

2019 was a very important year for the company as we generated positive clinical data across all four of our programs. For BP, AKC and HSCT-TMA, the rapid patient response we generally saw in our clinical studies validates these disease targets for nomcaopan where the specific dual action of the drug provides a potential significant differentiation with its inhibition of both the complement (C5) and leukotriene (LTB4) pathways, said Clive Richardson, Chief Executive Officer of Akari Therapeutics. In 2020, we look forward to expanding these programs further and plan on focusing on preparatory work for potential pivotal studies in anticipation of lessening the impact of the COVID-19 pandemic. At the same time we are working with our employees, partners and patients to help ensure their safety and maintain continuity where possible.

Full Year 2019 and Recent Business Highlights

Akaris strategy is to focus on orphan inflammatory diseases with significant unmet medical need, where the role of the complement and leukotriene systems are implicated. Akaris lead programs are in BP, AKC, and HSCT-TMA where clinical data with nomacopan has shown rapid and sustained clinical improvement in patients. These diseases have no approved treatments.

The Company is working with clinical sites and is following regulatory and health agency guidance related to the COVID-19 pandemic to help ensure the safety of its employees and patients. Our BP study has completed recruitment while our AKC study has halted recruitment with around two thirds of patients recruited. We expect delays in opening sites for our HSCT-TMA program. We expect our long-term safety program will shift to being managed on a country by country basis and some disruption is expected.

Phase II clinical trial in patients with BP

In the fourth quarter of 2019, interim Phase II trial results with nomacopan were presented at the 28th European Academy of Dermatology and Venereology (EADV) Congress. The data showed that four of the six patients were classified as at the upper limit of moderate BP. The four patients saw a rapid and significant improvement in symptoms, with a mean 63% decline in BPDAI score and mean 68% decline in blister score by Day 42, with either no or minimal early steroid treatment with one moderate patient having a flare up post Day-28. The data showed nomacopans potential as a possible treatment for BP with the additional and important benefit of reducing steroid use which has multiple adverse effects including a threefold increased risk of mortality. The Phase II trial has completed recruitment, with full data expected in the second quarter of 2020.

During the third quarter of 2019, the FDA granted orphan drug designation for nomacopan for the treatment of BP. The Company is now evaluating pivotal trial designs.

Phase III clinical trial in pediatric patients with HSCT-TMA

Initiated a pivotal Phase III trial for HSCT-TMA with nomacopan following the opening of an IND by the FDA. As a result of the COVID-19 pandemic, although we are looking to continue the process of site openings, we anticipate this will be delayed and hence any enrollment. This two-part Phase III study in pediatric patients with HSCT-TMA is based on guidance from the Companys end-of-Phase II meeting with the FDA. Part A of the trial is a dose confirmation study with the dosing agreed with FDA via their Model Informed Drug Development Program (MIDD). Part B of the trial is a single arm responder-based efficacy study that will follow an interim analysis of Part A and a meeting with the FDA. This devastating condition has an estimated 80% mortality rate in children, at elevated risk of dying who will be recruited to the trial and has no approved treatments. Akari has both FDA fast track pediatric patients and orphan drug designation status for this program.

Phase I/II clinical trial in patients with AKC

In 2019, the Company successfully completed Part A of the Phase I/II clinical trial in severe AKC patients who showed a rapid overall improvement of a mean 55% in the composite clinical score. The nomacopan eye drops were found to be comfortable and well tolerated with no reported drug related serious adverse events. Enrollment in the Part B placebo-controlled efficacy arm of the study has now stopped due to the COVID outbreak, but recruited patients continue to be treated. We anticipate that when the trial closes, we will have data on around two thirds of the target 19 patient study.

During the first quarter of 2020, the Company announced new preclinical data indicating that PAS-nomacopan, the long acting form of the drug, significantly reduced both retinal inflammation and intraocular VEGF. PAS-nomacopan was found to reduce intraocular VEGF levels by as much as the anti-VEGF antibody with 74% (p=0.04) and 68% (p=0.05) reductions respectively, compared to saline control. Furthermore, while clinically assessed inflammation increased in both the control and anti-VEGF groups by 49% and 33%, respectively, PAS-nomacopan treatment resulted in a 9% reduction in inflammation which represents a 58% difference compared to control assessed by retinal fundoscopy (p=0.02). This therapeutic activity across multiple pathogenic pathways (VEGF, inflammation and complement) supports the potential for nomacopan as a new mode of action for the treatment of back of the eye diseases.

PNH program

The Company continues to accumulate positive long-term treatment data, which includes more than 30 cumulative patient-years of data with 14 PNH patients across four clinical trialswith no reported drug related serious adverse events. Interim data from the Phase III CAPSTONE study on the first eight PNH patients who were all transfusion dependent at entry to the CAPSTONE trial show that all four patients randomized to nomacopan were transfusion independent for the first six months of treatment while all four patients on standard of care (SOC) remained transfusion dependent. Recruitment into the Phase III CAPSTONE study has been discontinued, although a PNH program may be re-initiated to potentially take advantage of the new high concentration formulation.

Akari has been granted orphan status from theFDA andthe European Medicines Agency(EMA) for nomacopan for treatment of PNH.

Fourth Quarter and Full Year 2019 Financial Results

A copy of the Companys Annual Report on Form 20-F for the year ended December 31, 2019 has been filed with the Securities and Exchange Commission and posted on the Companys website at http://investor.akaritx.com/financial-information/sec-filings. You may request a copy of the Companys Form 20-F, at no cost to you, by writing to the Financial Controller of the Company at 75/76 Wimpole Street, London W1G 9RT, United Kingdom or by calling the Company at +44 20 8004 0261.

Important Message Regarding COVID-19

Public health epidemics or outbreaks could adversely impact our business. In late 2019, a novel strain of COVID-19, also known as coronavirus, was reported in Wuhan, China. While initially the outbreak was largely concentrated in China, it has now spread to several other countries, including in the United Kingdom and the United States, and infections have been reported globally. In particular, our clinical trial sites are based in areas currently affected by coronavirus. Epidemics such as this can adversely impact our business as a result of disruptions, such as travel bans, quarantines, and interruptions to access the trial sites and supply chain, which could result in material delays and complications with respect to our research and development programs and clinical trials. Moreover, as a result of coronavirus, there is a general unease of conducting unnecessary activities in medical centers. As a consequence, our ongoing trials have been halted or disrupted. It is too early to assess the full impact of the coronavirus outbreak on trials for nomacopan, but coronavirus is expected to affect our ability to complete recruitment in our original timeframe. The extent to which the coronavirus impacts our operations will depend on future developments, which are highly uncertain and cannot be predicted with confidence, including the duration and severity of the outbreak, and the actions that may be required to contain the coronavirus or treat its impact. In particular, the continued spread of the coronavirus globally, could adversely impact our operations and workforce, including our research and clinical trials and our ability to raise capital, could affect the operations of key governmental agencies, such as the FDA, which may delay the development of our product candidates and could result in the inability of our suppliers to deliver components or raw materials on a timely basis or at all, each of which in turn could have an adverse impact on our business, financial condition and results of operation.

About Akari Therapeutics

Akari is a biopharmaceutical company focused on developing inhibitors of acute and chronic inflammation, specifically for the treatment of rare and orphan diseases, in particular those where the complement (C5) or leukotriene (LTB4) systems, or both complement and leukotrienes together, play a primary role in disease progression. Akari's lead drug candidate, nomacopan (formerly known as Coversin), is a C5 complement inhibitor that also independently and specifically inhibits leukotriene B4 (LTB4) activity. Nomacopan is currently being clinically evaluated in four indications: bullous pemphigoid (BP), atopic keratoconjunctivitis (AKC), thrombotic microangiopathy (TMA), and paroxysmal nocturnal hemoglobinuria (PNH). Akari believes that the dual action of nomacopan on both C5 and LTB4 may be beneficial in AKC and BP. Akari is also developing other tick derived proteins, including longer acting versions.

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Akari Therapeutics Reports Fourth Quarter and Full Year 2019 Financial Results and Business Highlights - PharmiWeb.com

VANCOUVER, Washington, March 31, 2020 (GLOBE NEWSWIRE) CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today that the U.S. Food and Drug Administration (FDA) just provided clearance for initiation of a Phase 2 trial with CytoDyns leronlimab to treat COVID-19 patients with mild to moderate indications. The Companys investigational new drug, leronlimab, has been administered to 10 severely ill patients with COVID-19 at a leading medical center in the New York City area under an emergency IND recently granted by the FDA. The treatment with leronlimab is intended to serve as a therapy for patients who experience mild-to-moderate respiratory complications as a result of contracting SARS-CoV-2 causing the Coronavirus Disease 2019 (COVID-19).

The Phase 2 clinical trial is a randomized, double blind, placebo-controlled study to evaluate the efficacy and safety of leronlimab in patients with mild to moderate documented COVID-19 illness and calls for 75 planned patients in up to 10 centers in the United States. Patients enrolled in the trial are expected to have a treatment window of approximately 6 weeks.

Bruce Patterson, M.D., Chief Executive Officer and founder of IncellDx, a diagnostic partner and advisor to CytoDyn, commented, We are very pleased with the FDAs responsiveness to facilitate the commencement of this important Phase 2 trial. In light of the test results of the various immunologic markers from the critically ill patients treated under the emergency IND, we remain hopeful that leronlimab may be therapeutically beneficial to those COVID-19 patients with mild to moderate indications.

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn said, The FDA has been very collaborative with our team to accelerate the opportunity to introduce a potentially beneficial treatment to so many patients affected by this horrific pandemic. We hope to complete enrollment of this trial very quickly.

About Coronavirus Disease 2019SARS-CoV-2 was identified as the cause of an outbreak of respiratory illness first detected in Wuhan, China. The origin of SARS-CoV-2 causing the COVID-19 disease is uncertain, and the virus is highly contagious. COVID-19 typically transmits person to person through respiratory droplets, commonly resulting from coughing, sneezing, and close personal contact. Coronaviruses are a large family of viruses, some causing illness in people and others that circulate among animals. For confirmed COVID-19 infections, symptoms have included fever, cough, and shortness of breath. The symptoms of COVID-19 may appear in as few as two days or as long as 14 days after exposure. Clinical manifestations in patients have ranged from non-existent to severe and fatal. At this time, there are minimal treatment options for COVID-19.

About Leronlimab (PRO 140)The FDA has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer.Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH.Leronlimab has completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting aPhase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD.

About CytoDynCytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells.The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in April of 2020 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than five years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is atwww.cytodyn.com.

Forward-Looking StatementsThis press releasecontains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes, hopes, intends, estimates, expects, projects, plans, anticipates and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. The Companys forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i)the sufficiency of the Companys cash position, (ii)the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv)the Companys ability to enter into partnership or licensing arrangements with third parties, (v)the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi)the Companys ability to achieve approval of a marketable product, (vii)the design, implementation and conduct of the Companys clinical trials, (viii)the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix)the market for, and marketability of, any product that is approved, (x)the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi)regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii)general economic and business conditions, (xiii)changes in foreign, political, and social conditions, and (xiv)various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form10-K, and any risk factors or cautionary statements included in any subsequent Form10-Q or Form8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CYTODYN CONTACTSInvestors:Dave Gentry, CEORedChip CompaniesOffice: 1.800.RED.CHIP (733.2447)Cell: 407.491.4498dave@redchip.com

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FDA Clears CytoDyn's Phase 2 Randomized Trial to Treat Mild-to-Moderately Ill Coronavirus Patients with Leronlimab; Enrollment to Begin Immediately -...

WALTHAM, MACoronavirus that causes COVID-19 disease is the worst pandemic the world has ever seen. If you are confused with the overwhelming information about the virus, you may want to watch this video interview with Dr. Sailaja Reddy, MD, IFMCP, ABoIM, ABOM, CCMS.

In an exclusive video interview with INDIA New England News, Dr. Reddy explains coronavirus, how it is different from other viruses, how we can prevent it from infection, how it infects and how to take care after recovery from COVID-19.

To watch the full interview, please click here or on the image below.

Dr. Reddy educates, supports, treats and transforms peoples lives by getting to the root cause of illness using the best of science and nature. Dr. Reddy is functional and integrative medicine specialist, amd is founder of RootHealthMD in Framingham, Mass, focusing on reversal of chronic diseases and supports the development of vibrant longevity.

Dr. Reddy did her residency at St. Elizabeths Hospital and received advanced training in obesity medicine, Integrative medicine and culinary medicine. She is a certified physician through Institute of Functional Medicine.

Immunity, says Dr. Reddy, is a defense system that has biological structure and process within humans that protects against diseases.

Our body builds immunity through healthy lifestyle. Diet, genetics and environment play a major role, says Dr. Reddy, adding that one should change lifestyle in order to build immunity.

A diet that is nutrient rich, with low sugar, good quality protein and less processed will strengthen your immune system, says Dr. Reddy. The latest science suggests that being fit boosts our immune systems, and that even a single workout can amplify and improve our ability to fight off germs.

PS: This information is for information purposes only. If you have any discomfort, please consult your physician immediately.

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Everything You Need to Know About Coronavirus: Before, During, After and How to Build Immunity Against Future Viruses - India New England

As the coronavirus spreads around the globe, it has mutated in tiny, subtle ways. Those mutations arent cause for concern, and so far, dont appear to be making the virus any more or less dangerous. But scientists can use those slight changes to track the virus from person to person, and location to location.

If we identify a new outbreak cluster in one state, and theres a question of whether its related to a previous cluster or not, the small mutational changes can help you figure out if theyre connected, says Patrick Boyle, a synthetic biologist at Ginkgo Bioworks.

The coronavirus is made up of around 29,000 building blocks of genetic material called nucleotides. Like other biotechnology companies and labs, Ginkgo has the technology to take a sample of the virus and read out the full sequence of those nucleotides. For the most part, the sequence will be the same in each sample. But the virus makes copies of itself within a human host, and sometimes, it can make mistakes switching one or two nucleotides out for another. The version of the virus with those changes can then be passed on when that person infects someone else.

Ginkgo is repurposing its systems, which normally dont sequence viruses, to analyze as many samples of the coronavirus as possible. The goal is to help build out the maps that show how the virus jumped from one person to the next. Theyre hoping to scale up to be able to publish the full genetic sequence of 10,000 virus samples a day.

Despite the skyrocketing numbers of COVID-19 cases in the US, only a limited number of virus samples collected in the country have been sequenced in full. Scientists have more sequences from Washington state than other places. Consequently, they know more about the trajectory of the outbreak in Washington than they know about outbreaks in other states.

Some of that genetic data is how Trevor Bedford, a virologist at the Fred Hutchinson Cancer Research Center, was able to link a case of COVID-19 diagnosed on February 27 in Washington to a case that was diagnosed in late January in the state indicating that the virus had been circulating locally, and undetected, for that entire time. It also showed that the January case sparked a cluster of illnesses that spread through the community.

Other states are starting to do the same kind of detective work, using genetic sequences to help clarify their outbreaks. An analysis of nine virus samples collected in Connecticut showed that the some were related to viruses found in Washington state, which suggests that the coronavirus was spreading domestically, not being repeatedly brought in from other countries. The analysis has not yet been peer-reviewed or published. Other preliminary research examined virus samples from northern California, and found that the coronavirus was introduced to the area at multiple points.

One challenge in expanding the number of virus sequences available, Boyle says, is obtaining patient samples to analyze. Labs in the US and other countries that are running tests for the virus receive hundreds or thousands of patient samples each day. But the focus of those labs is checking a sample to see if the coronavirus is there and the patient has COVID-19 or if it isnt. The emphasis on testing and diagnosing patients is critical to track the pandemic, Boyle says.

The problem is, it only gives you a positive or negative answer, he says. Tests dont provide any extra information about the particular virus in each patient. Ginkgo plans to partner with testing labs, so that they can take a closer look at the virus in a patient sample after the testing is done. Other labs and groups worldwide are embarking on similar projects: a research consortium in the United Kingdom, for example, has over $20 million in funding to sequence samples. Boyle says that Gingko is coordinating with some other labs interested in this work.

Theyre also making sure that they can access the chemicals and other supplies they need to run the genetic analysis, Boyle says. We want to make sure that our supply stream is not competing with the supply stream that keeps the testing running.

Expanding the number of coronavirus sequences available will give scientists a picture of the outbreak, in the US and around the world. Along with testing, its one way scientists can keep track of the viruss movements and help to rein it in.

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Genetic analysis of the coronavirus gives scientists clues about how its spreading - The Verge

Humanity really has only two options to confront the coronavirus pandemic currently sweeping the planet. The first is to mount a rolling program of lockdowns and other drastic social distancing strategies to restrain the pace of the virus epidemic, with a view to gradually building up natural herd immunity among the human population.

That strategy, especially if combined with successful anti-viral drug treatments and a massively upscaled testing effort, should give some relief. But it would come at the likely cost of many millions of deaths and incalculable worldwide economic damage, hitting especially hard in countries with little resilience and limited healthcare infrastructure.

The second approach is to develop a vaccine, and to do so as rapidly as possible. A fully effective vaccine would not just tame COVID-19 but possibly eradicate it altogether as the world successfully did with smallpox and is on the verge of doing with polio (both also viral diseases).

These two approaches will most likely be concurrent: the first will buy us time, while the second provides an exit strategy from a constant pattern of repeating lockdowns and travel restrictions that could otherwise go on for years.

With the current total of confirmed cases rapidly closing in on one million worldwide, the true picture is most likely that many tens of millions of people have already caught COVID-19. Humanitys most desperate challenge, therefore, is to find an effective vaccine.

Fortunately, science is already stepping up. History was made on March 16, when the first clinical trial volunteer was injected with an investigational vaccine for coronavirus at the Kaiser Permanente Washington Health Research Institute in Seattle.

The volunteer was mother-of-two Jennifer Haller, a 43-year-old Seattle resident who told National Public Radio that she wanted to do something because theres so many Americans that dont have the same privileges that Ive been given.

The vaccination was produced by Moderna, with the first batch being delivered to the US National Institutes of Health a remarkable 42 days after the viral genome was first sequenced in China.

This Phase 1 trial does not yet test the efficacy of the vaccine against COVID-19. Carried out over six weeks among a group of 45 healthy adult volunteers aged between 18 and 55, it will test the basic safety of the proposed vaccine and its ability to stimulate an immune response in the human body.

Although the Phase 1 trial will continue with the Seattle-area recruits being monitored for a whole year, the urgency of the global situation means that the collaborators will likely rush to Phase 2 at the same time, testing the ability of the vaccine to prevent infection by the novel coronavirus SARS-CoV-2 that causes COVID-19.

The Moderna vaccine trial is a world first not just for the particular disease target but because it is one of a whole new potential class of vaccines that employ messenger RNA (mRNA) to program human cells to produce the viral proteins that trigger an immune response, rather than injecting proteins or viral particles directly, as have most previous vaccines.

This natural role of mRNA is why Modernas approach is so quick. Normal vaccines have to be produced from actual viruses, which are grown within chicken eggs and then refined into sufficient quantities to be directly injected once weakened or killed into the human body. This takes months, at a minimum, and is difficult to scale quickly.

For the mRNA approach, all that was needed was the correct viral genetic sequence, which in the case of SARS-CoV-2 encodes for the spike proteins that enable the virus to gain entry into human respiratory cells. This genetic sequence for the viral protein can then be encoded into mRNA synthetically generated in a lab a rapid process that is easy to scale.

Thats the good news. The bad news is that the mRNA approach, while undoubtedly quick and versatile, is so new that it has yet to be fully proven in any vaccine in either humans or animals. Some tests have shown efficacy against rabies, for example, but others have shown little lasting immune response.

The mRNA approach is therefore a moon-shot rather than a marathon. Even so, Moderna is optimistic enough to already be making plans to produce millions of doses intended for health workers initially as early as this fall.

Other companies and partnerships are also racing to develop a vaccine using the same mRNA approach. One of these, the German firm CureVac, generated so much interest that President Trump reportedly tried to acquire it in order to ensure any potential vaccine would be available to Americans first.

Like Moderna, CureVacs efforts are supported financially by CEPI the international Coalition for Epidemic Preparedness Innovations, which has raised over $700 million from governments around the world and philanthropic foundations like the Bill & Melinda Gates Foundation (which also supports the Cornell Alliance for Science) and Wellcome.

While Moderna has been able to restart vaccine projects originally intended for MERS and SARS, CureVac has already achieved some success with an mRNA vaccine against rabies virus in humans. In a Phase 1 trial doses as low as a millionth of a gram of mRNA vaccine were sufficient to fully protect humans against rabies, it reported in January.

Such small doses offer major promise for immunizing huge numbers of people if CureVac is able to achieve the same success with SARS-CoV-2 as it has with rabies and move rapidly into Phase 2 trials to further demonstrate real efficacy.

Also in Germany, BioNTech and Pfizer are racing to shift their mRNA vaccine work from influenza to SARS-CoV-2, and are aiming to start clinical trials as soon as April. As part of a broader collaboration, BioNTech has already demonstrated that an mRNA vaccine protected mice and non-human primates against Zika virus, raising hopes for similar effectiveness against COVID-19.

RNAs double-stranded cousin, DNA, is also being deployed in a novel but equally promising vaccine system against the coronavirus. The approach is related, but rather than injecting mRNA directly into cells so that it can produce viral proteins, DNA is inserted, which in turn produces mRNA inside cells to do the same job.

This DNA is not intended to integrate into the genome of the target cell in humans indeed if this happens, damaging mutations might occur. Instead, DNA is formed into circular plasmids which operate separately to the integral genetic material inside a cells nucleus. Like genomic DNA however, these plasmids are read and transcribed via mRNA into viral proteins which can then prime the bodys immune system against a later invasion by the real virus.

The US-based Inovio Pharmaceuticals announced on 12 March that it had received a grant of $5 million from the Bill & Melinda Gates Foundation to accelerate the testing of a DNA vaccine for COVID-19, with a view to starting Phase 1 clinical trials in April.

Inovio has another advantage: its DNA vaccine INO-4700 was the only vaccine candidate against MERS to progress to Phase 2 trials demonstrating, at least initially, the potential feasibility of the DNA approach. The US Department of Defense with an eye to protecting its military personnel all over the world against COVID-19 has pumped another $11.9 million into INO-4800. The company has also demonstrated protection in early trials using its DNA vaccine against Chikungunya, Zika and influenza viruses.

CEPI is not putting all its eggs in one basket, however. As well as DNA and RNA systems, another promising approach for a COVID-19 vaccine is to use a genetically engineered measles vaccine a strategy supported by a $5 million CEPI grant split between collaborating institutions Themis in Vienna, Institut Pasteur in France and the University of Pittsburghs Center for Vaccine Research.

This takes the live attenuated measles virus vaccine a vaccine with a long history of safe use, having been used to immunize billions of children over the last 40 years and uses reverse genetics technology to insert new genes coding for proteins expressed by other viruses. These then induce an immune response against the new virus whose genetic material has been introduced.

The research team aims to have a COVID-19 candidate vaccine ready for animal testing as soon as April, with wider tests in human volunteers by the end of the year.

Measles virus is not the only candidate for the vector approach. Chinese scientists have reported that they are about to proceed to Phase I human trials with a vaccine candidate starting at the pandemics epicenter in Wuhan. The scientists have genetically engineered a replication-defective adenovirus type 5 (Ad5) as a vector to express the SARS-CoV-2 spike protein, with the resulting vaccine candidate named Ad5-nCoV.

This is perhaps the easiest approach, as all that has to happen is for the engineered harmless adenovirus to infect patients in order to trigger the production of antibodies which should be effective against invading novel coronavirus too. The Chinese company CanSion Biologics has successfully demonstrated this approach with another fully completed vaccine against Ebola, Ad5-EBOV, which is already on the market in China.

A more tried-and-tested approach already widely used to produce flu vaccines is to grow viral proteins directly: these are then injected as a vaccine into human patients so that the immune system is already primed against the real pathogen when it attempts to infect the body. Usually chicken eggs are used, but to speed things up insect cell lines are becoming the preferred option for the coronavirus pandemic.

Here genetics is again an important component: the company Novavax uses a baculovirus vector to genetically engineer an insect cell line originally isolated decades ago from the ovaries of the fall armyworm. The baculovirus transports genes into the insect cells, which program them to manufacture viral proteins that are correctly folded and biologically active, more reliably enabling the human immune system to produce antibodies against them.

According to Novavax, its resulting recombinant protein nanoparticles then self-assemble into a structure that approximates the actual virus, helping enhance the immune response. It claims to have already tested this system in RSV virus, a recalcitrant pathogen that has so far resisted attempts at a vaccine. This approach looks promising enough that CEPI has pumped $4 million in so far with a view to launching Phase I trials by late spring 2020.

In a similar way, the company Sanofi is taking a snippet of genetic code from SARS-CoV-2 and splicing it also via baculovirus into insect cell lines. Its advantage, made in a pitch to the US government that resulted in a big cash injection, is that it already has an FDA-approved facility that could make 600 million doses a year of any resulting vaccine.

Plants can also be engineered to produce viral proteins. The company Medicago is working with genetically modified tobacco plants with this aim in mind. To speed things up, instead of adding new genes to the nucleus of cells and regenerating entire plants from these single cells (as happens with conventional plant genetic engineering), it uses the Agrobacterium vector in a vacuum to transfer recombinant DNA directly into the nucleus of fully-grown leaf cells. This DNA enables the production of the desired viral proteins without ever being integrated into the genome, enabling proteins to be harvested from transformed leaves within a matter of days.

Using this system, Medicago claims to have produced a virus-like particle of the coronavirus within just 20 daysof the SARS-CoV-2 genetic sequence becoming available. The government of Canada quickly put millions of dollars behind the effort as a result.

Astonishingly, given that the coronavirus pandemic is now threatening to devastate societies and economies around the planet on a scale second only to a world war, this effort is still short of cash. CEPI has issued an urgent call for funding, seeking to raise $2 billion: it says just $375 billion by the end of March would enable four-to-six vaccine candidates to move rapidly towards phase 2/3 trials.

Scientists are also hoping desperately that SARS-CoV-2 does not rapidly mutate as influenza viruses tend to do, which would likely reduce the effectiveness of any single vaccine. So far, according to researchers studying 1,000 samples of the virus from around the world, this seems not to be the case.

This means that the race to find a vaccine, and to do so in sufficient time to salvage the situation before the world tips into an economic depression and millions of people die, has a decent chance of success and that any successful vaccine would likely confer lasting immunity.

Meanwhile, all of humanity is waiting. And if the scientists do succeed in this urgent challenge, it will very likely be due to modern genetics. Though genetic engineering was once a dirty word, it now could literally help save the world.

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Science to the rescue? How modern genetics could help save the world from coronavirus - Alliance for Science - Alliance for Science

Demonstrates broad biodistribution, including across the blood-brain barrier into the central nervous system, and into skeletal muscle, in non-human primates (NHPs) after systemic administration

Durable and therapeutically relevant drug concentrations achieved in NHPs after single intravenous dose

Potent cell-based activity and allele-specific enrichment in patient-derived cell lines

Platform validation data supports expansion of the therapeutic pipeline into new organ systems previously unreachable with first-generation antisense oligonucleotide technology

Management to hold a conference call today at 8 a.m. ET

PITTSBURGH, March 31, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq:NBSE) (NeuBase or the Company), a biotechnology company developing next-generation antisense oligonucleotide (ASO) therapies to address genetic diseases, today announced positive preclinical data from its pharmacokinetics studies in non-human primates (NHPs) and in vitro pharmacodynamics data in patient-derived cell lines. NeuBase believes these data validate the key advantages of the proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL) platform and support the Companys decision to advance the development of its Huntingtons disease (HD) and myotonic dystrophy type 1 (DM1) programs, as well as the potential expansion of its therapeutic pipeline into other indications.

Dr. George Church, professor of genetics at Harvard Medical School and member of the National Academy of Sciences, stated, Given the activity and broad biodistribution observed in these studies and the potential for easier target definition, I believe the PATrOL technology may have a potent impact on the future of drug development and treatment of genetic diseases.

Non-Human Primate Pharmacokinetic Study

Quantitative whole-body autoradiography was performed on NHPs.A PATrOL-enabled compound was radio-labeled, and theresulting material was injected into NHPs at 5 mg/kg via a bolus tail vein injection. At four hours, twelve hours, and seven days post-dosing, NHPs were sacrificed andsectioned into 40 m slices.Slices were exposed to autoradiography imaging plates alongside a dilution series of radioactive PNA in whole blood.Upon imaging, the dilution series enabled an analysis of the amount of compound in each of the tissues. In addition, prior to sacrifice, whole blood, urine, and feces were collected from the NHPs at specified timepoints.The major conclusions from this study include:

Rapid uptake of compound out of the bodys circulation after systemic intravenous administration, with a half-life in circulation of approximately 1.5 hours;

Compound penetrates every organ system studied, including the central nervous system and skeletal muscle;

Compound crosses the blood-brain barrier and into the key deep brain structures, including the caudate, supporting a key capability for the development of the Companys lead program in HD; Delivery of the compound to skeletal muscle, the primary organ system that is affected in DM1;Because both HD and DM1 have manifestations outside of the primary affected organ, the broad biodistribution of the compounds may enable a potential whole-body therapeutic solution in both indications.

96% of administered compound remained in vivo after a one-week period (latest timepoint tested);Redistribution over one week after administration between organ systems enriches concentrations in key brain regions up to two-fold, including in those deep brain structures most relevant for HD;Retention of ~90% of compound concentrations achieved in skeletal muscle over the course of one-week post-single-dose administration; and

Patient-Derived Huntingtons Cell Line Pharmacodynamic Studies

Multiple Huntingtons disease candidate compounds were incubated with HD-derived cells and assayed for their toxicity and their ability to selectively knock down mutant huntingtin protein (mHTT) expression by engaging with the CAG repeat expansion in the huntingtin (HTT) gene transcript. Multi-well plates were seeded with cells and candidates were added to the culture at various concentrations.Cells were grown for three days and thereafter assayed for cell death.Cell pellets were also collected, lysed, and run on gradient SDS-PAGE gels.Following the transfer of the proteins to a membrane, the membrane was probed with anti-huntingtin and anti-beta-actin antibodies.Secondary antibodies were used to image the immunoblots.The beta-actin bands were used to normalize the amount of protein across the wells.The amounts of mutant and wild type huntingtin protein in treated cells were compared to untreated cells to determine the level of knockdown.The major conclusions from this study include:

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Activity in engaging target disease-causing transcripts and knocking-down resultant malfunctioning mHTT protein levels preferentially over normal HTT protein knock-down; and

Dose limiting toxicities were not observed relative to a control either at or above the doses demonstrating activity in human cells in vitro.

In addition, PATrOL enabled compounds were generally well-tolerated in vivo after systemic administration, both after single dose administration in NHPs and multi dose administration in mice for over a month.

We believe the PATrOL platform has the potential to create drugs that are easy for patients to take at infrequent intervals after they have tested positive for a genetic disease but before symptoms emerge, said Dietrich Stephan, Ph.D., chief executive officer of NeuBase. We believe the best way to effectively manage degenerative genetic diseases is to get ahead of the disease process, and we believe that can only be achieved with early diagnosis coupled with well-tolerated, effective, and easily administered therapies.

Dr. Robert Friedlander, chief medical officer of NeuBase and member of the National Academy of Medicine, stated, An allele specific approach that can be systemically administered and cross the blood brain barrier would be an ideal drug profile for many untreatablegenetic diseases.I believe that NeuBase is moving towards realizing this goal.

The intersection of the NHP pharmacokinetic data and the in vitro patient-derived pharmacodynamic data provides a roadmap to create a pipeline of therapeutic candidates which can reach target tissues of interest after systemic administration and achieve the desired activity at that dose. NeuBase believes the data from these studies support the advancement of the Companys HD and DM1 programs into lead optimization and subsequent IND-enabling studies, as well as provide a roadmap for the future expansion of the Companys therapeutic pipeline into other indications, including oncology.

Dr. Sam Broder, former Director of the National Cancer Institute of the National Institutes of Health and member of the National Academy of Sciences, stated, I believe that the NeuBase strategy of targeting transcripts before they become dangerous mutant proteins has the potential to deliver a dramatic improvement in our collective capabilities to effectively treat a wide range of genetic diseases, including some of the most deadly cancers, by targeting driver mutations and accelerating immunotherapy capabilities.

Conference Call

NeuBase Therapeutics, Inc. will discuss these data and next steps for development during a webcasted conference call with slides today, March 31, 2020, at 8:00 a.m. ET. The live and archived webcast of this presentation can be accessed through the IR Calendar page on the Investors section of the Companys website, http://www.neubasetherapeutics.com. The dial-in details for the call are 877-451-6152 (domestic) or +1-201-389-0879 (international), and conference ID: 13701118. The archived webcasts will be available for approximately 30 days following the presentation date.

About NeuBase Therapeutics

NeuBase Therapeutics, Inc. is developing the next generation of gene silencing therapies with its flexible, highly specific synthetic antisense oligonucleotides. The proprietary NeuBase peptide-nucleic acid (PNA) antisense oligonucleotide (PATrOL) platform is designed to permit the rapid development of targeted drugs, thereby potentially increasing the treatment opportunities for the hundreds of millions of people affected by rare genetic diseases, including those that can only be treated through accessing of secondary RNA structures. Using PATrOL technology, NeuBase aims to first tackle rare, genetic neurological disorders.

Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements include, among other things, statements regarding the Companys goals and plans and the Companys pharmacokinetics and pharmacodynamics studies. These forward-looking statements are distinguished by use of words such as will, would, anticipate, expect, believe, designed, plan, or intend, the negative of these terms, and similar references to future periods. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those described in the risk factors contained in our filings with the U.S. Securities and Exchange Commission, may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Companys current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Companys plans to develop and commercialize its product candidates; the Companys plans to commence clinical trials in Huntingtons disease and myotonic dystrophy type 1 and to potentially expand the pipeline into other indications; the utility of the preclinical data generated in existing studies performed by the Company in determining the results of potential future clinical trials and of the potential benefits of the PATrOL platform technology; the timing of initiation of the Companys planned clinical trials; the timing of the availability of data from the Companys clinical trials; the timing of any planned investigational new drug application or new drug application; the Companys plans to research, develop and commercialize its current and potential future product candidates; the clinical utility, potential benefits and market acceptance of the Companys current and potential future product candidates; the Companys commercialization, marketing and manufacturing capabilities and strategy; the Companys ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors in our filings with the U.S. Securities and Exchange Commission. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

NeuBase Investor Contact:Dan FerryManaging DirectorLifeSci Advisors, LLCDaniel@lifesciadvisors.comOP: (617) 535-7746

NeuBase Media Contact:Travis Kruse, Ph.D.Russo Partners, LLCtravis.kruse@russopartnersllc.comOP: (212) 845-4272

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NeuBase Therapeutics Announces Positive, Preclinical Data Validating its Novel Genetic Therapy PATrOL Platform - Yahoo Finance

Sarah Cutshall | Visual Editor

Not leaving home leaves plenty of time to spend with your pets.

Were sure youre up to date with all the bad news thats been circulating. Stocks are down. Schools are closed. People are under orders to stay home, etc. With all the depressing news of the last month or two, were almost certain youve been missing out on some of the good things that have happened around the world. That being said, the editorial board presents you with 10 positive things to briefly distract you from all the negative headlines.

You heard us right, ladies and gents. Jack Black is on TikTok. Whether youre a fan of his or not, this is probably going to be a wild and entertaining ride.

Across the world, people are recovering from COVID-19. Wuhan, China the site of the first outbreak is largely on the mend. Meanwhile, in Italy, a 101-year-old man recovered from the virus and was released from the hospital he was being treated in. Elsewhere, people are making similar recoveries.

Thats right college students who previously barely had time to eat, let alone cook, are now standing in front of the stove with a recipe pulled up on their phone screens. What exactly are we cooking? Well, it seems like the top choice is bread. Our take on this: nothing is more comforting than bread thats fresh out of the oven.

Yeah. That Toxic. On March 20, the one and only Andrew Hozier Byrne covered Toxic by Britney Spears on an Instagram live concert. We dont need to explain why this is positive.

Turns out even a global pandemic cant stop love. A couple in New York City got married in the street while their friend officiated from a fourth-floor window. Similarly, in Pittsburgh, a couple got married on their front porch with their neighbors as witnesses standing six feet away, of course.

Things are looking up, especially when it comes to the weather! As spring comes in, the outside world promises to get warmer and unless youre living in Pittsburgh sunnier.

With many places under orders to stay home, pets are bound to be less lonely. Their owners are at home with them, which is basically what dogs have been dreaming about forever. Cats could probably care less about this news, though they probably dont mind having their owners around to sit on.

Animal shelters have seen a large increase in people interested in fostering pets. This is great for both the pets and the people fostering them as the pets get to live with a family rather than in a shelter, and the family gets to experience the joys of having a pet.

Two years after he received a stem cell treatment to cure HIV, Adam Castillejo is still free from the virus. While doctors say that the treatment he received was high-risk and not a widespread solution, this is still by and large good news hopefully, we are moving towards finding a less invasive and more widely accessible cure.

Since the Montreal Protocol was enacted in 1987, the ozone layer has been on the mend. The protocol phased out numerous ozone depleting substances such as chlorofluorocarbons, and its clear now that this made a positive change for the ozone layer. The atmospheric presence of these substances is declining, allowing the ozone layer to heal itself.

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Editorial: Top 10 positive things going on right now - University of Pittsburgh The Pitt News

NEW YORK, March 31, 2020 (GLOBE NEWSWIRE) -- Incysus Therapeutics, Inc. (Incysus), a clinical-stage biopharmaceutical company focused on delivering an innovative gamma-delta () T cell immunotherapy for the treatment of cancers, today announced that the Company will present at the 2020 NYC Spring Oncology Investor Conference being held on March 31st and April 1st. William Ho, President and Chief Executive Officer of Incysus Therapeutics, will present a company update. With the on-going COVID-19 concerns, the Companys presentation will be live broadcast over a premier, private, and secure Zoom conference account. Please see below for more information.

About The NYC Oncology Investor ConferenceThe NYC Oncology Investor Conference brings together leading life science and oncology venture capitalists, family offices, lawyers, pharma executives, startup public and private cancer companies and cancer foundations for a discussion on trends, opportunities and risks in oncology investing (www.oncologyinvestorconference.com).

About Incysus Therapeutics, Inc.Incysus is focused on delivering a novel off-the-shelf cell therapy for the treatment of cancer. By using genetically modified gamma-delta () T cells, the Companys technology addresses the challenges that immunotherapies face targeting cold, low mutation cancers. Incysus immuno-oncology programs include activated and gene-modified adoptive cellular therapies that protect cells from chemotherapy and allow novel combinations to disrupt the tumor microenvironment and more selectively target cancer cells. The Companys first clinical program delivering a genetically modified T cell for the treatment of newly diagnosed glioblastoma (GBM) can be found here: (NCT04165941) and its second clinical program with an allogeneic T cell therapy for leukemia and lymphoma patients undergoing allogeneic stem cell transplantation can be found here: (NCT03533816). For more information about the Company and its programs, visitwww.incysus.com.

Forward Looking StatementsCertain statements herein concerning the Companys future expectations, plans and prospects, including without limitation, the Companys current expectations regarding its business strategy, product candidates, and clinical development process and timing, constitute forward-looking statements. The use of words such as may, might, will, should, expect, plan, anticipate, believe, estimate, project, intend, future, potential, or continue, the negative of these and other similar expressions are intended to identify such forward looking statements. Such statements, based as they are on the current expectations of management, inherently involve numerous risks and uncertainties, known and unknown, many of which are beyond the Companys control. Consequently, actual future results may differ materially from the anticipated results expressed in such statements. In the case of forward-looking statements regarding investigational product candidates and continuing further development efforts, specific risks which could cause actual results to differ materially from the Companys current expectations include: scientific, regulatory and technical developments; failure to demonstrate safety, tolerability and efficacy; final and quality controlled verification of data and the related analyses; expense and uncertainty of obtaining regulatory approval, including from the U.S. Food and Drug Administration; and the Companys reliance on third parties, including licensors and clinical research organizations. Do not place undue reliance on any forward-looking statements included herein, which speak only as of the date hereof and which the Company is under no obligation to update or revise as a result of any event, circumstances or otherwise, unless required by applicable law.

Contact:William HoIncysus Therapeutics, Inc.+1 646.600.6GDTinfo@incysus.comwww.incysus.com

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INCYSUS THERAPEUTICS TO PRESENT AT THE NYC SPRING ONCOLOGY INVESTOR CONFERENCE - Yahoo Finance

Michelle Kuppersmith's doctor recommended a bone marrow biopsy after suspecting she had a rare blood disorder. Though the biopsy was done by an in-network provider at an in-network hospital, Kuppersmith learned she was on the hook for $2,400 for out-of-network genetic profiling. Shelby Knowles for KHN hide caption

Michelle Kuppersmith's doctor recommended a bone marrow biopsy after suspecting she had a rare blood disorder. Though the biopsy was done by an in-network provider at an in-network hospital, Kuppersmith learned she was on the hook for $2,400 for out-of-network genetic profiling.

Michelle Kuppersmith feels great, works full time and exercises three to four times a week. So she was surprised when a routine blood test found that her body was making too many platelets, which help control bleeding.

Kuppersmith's doctor suspected the 32-year-old Manhattanite had a rare blood disorder called essential thrombocythemia, which can lead to blood clots, strokes and, in rare cases, leukemia.

Her doctor suggested a bone marrow biopsy, in which a large needle is used to suck out a sample of the spongy tissue at the center of the patient's hip bone.

Doctors examine the bone marrow under a microscope and analyze the DNA. The procedure allows doctors to judge a patient's prognosis and select treatment, if needed. Kuppersmith had heard the procedure can be intensely painful, so she put it off for months.

The biopsy performed by a provider in her insurance network, at a hospital in her network lasted only a few minutes, and Kuppersmith received relatively good news.

While a genetic analysis of her bone marrow confirmed her doctor's suspicions, it showed that the only treatment she needs, for now, is a daily, low-dose aspirin. She will check in with her doctor every three to four months to make sure the disease isn't getting worse.

All in all, Kuppersmith felt relieved.

Then she got a notice saying her insurer refused to pay for the genetic analysis, leaving her responsible for a $2,400 payment.

The patient: New York resident Michelle Kuppersmith, 32, who is insured by Maryland-based CareFirst Blue Cross Blue Shield. She works as director of special projects at a Washington-based watchdog group. Because she was treated in New York, Empire Blue Cross Blue Shield which covers that region handled part of her claim.

Total amount owed: $2,400 for out-of-network genetic profiling

The providers: Kuppersmith had her bone marrow removed at the Mount Sinai Ruttenberg Treatment Center in New York City, which sent her biopsy sample to a California lab, Genoptix, for testing.

Medical services: Bone marrow biopsy and molecular profiling, which involves looking for genetic mutations

What gives: The field of molecular diagnostics, which includes a variety of gene-based testing, is undergoing explosive growth, said Gillian Hooker, president of the National Society of Genetic Counselors and vice president of clinical development for Concert Genetics, a health IT company in Nashville, Tennessee.

A report from Concert Genetics, a company that helps clients manage genetic testing, found there are more than 140,000 molecular diagnostic products on the market, with 10 to 15 added each day.

The field is growing so quickly that even doctors are struggling to develop a common vocabulary, Hooker said.

Kuppersmith underwent a type of testing known as molecular profiling, which looks for DNA biomarkers to predict whether patients will benefit from new, targeted therapies. These mutations aren't inherited; they develop over the course of a patient's life, Hooker said.

Medicare spending on molecular diagnostics more than doubled from 2016 to 2018, increasing from $493 million to $1.1 billion, according to Laboratory Economics, a lab industry newsletter.

Charges range from hundreds to thousands of dollars, depending on how many genes are involved and which billing codes insurers use, Hooker said.

Based on Medicare data, at least 1,500 independent labs perform molecular testing, along with more than 500 hospital-based labs, said Jondavid Klipp, the newsletter's publisher.

In a fast-evolving field with lots of money at stake, tests that a doctor or lab may regard as state-of-the-art an insurer might view as experimental.

Worse still, many of the commercial labs that perform the novel tests are out-of-network, as was Genoptix.

Stephanie Bywater, chief compliance officer at NeoGenomics Laboratories, which owns Genoptix, said that insurance policies governing approval have not kept up with the rapid pace of scientific advances. Kuppersmith's doctor ordered a test that has been available since 2014 and was updated in 2017, Bywater said.

Although experts agree that molecular diagnostics is an essential part of care for patients like Kuppersmith, doctors and insurance companies may not agree on which specific test is best, said Dr. Gwen Nichols, chief medical officer of the Leukemia & Lymphoma Society.

Tests "can be performed a number of different ways by a number of different laboratories who charge different amounts," Nichols said.

Insurance plans are much more likely to refuse to pay for molecular diagnostics than other lab tests. Laboratory Economics found Medicare contractors denied almost half of all molecular diagnostics claims over the past five years, compared with 5-10% of routine lab tests.

With so many insurance plans, so many new tests and so many new companies, it is difficult for a doctor to know which labs are in a patient's network and which specific tests are covered, Nichols said.

"Different providers have contracts with different diagnostic companies," which can affect a patient's out-of-pocket costs, Nichols said. "It is incredibly complex and really difficult to determine the best, least expensive path."

Kuppersmith said she has always been careful to check that her doctors accept her insurance. She made sure Mount Sinai was in her insurance network, too. But it never occurred to her that the biopsy would be sent to an outside lab or that it would undergo genetic analysis.

She added: "The looming threat of a $2,400 bill has caused me, in many ways, more anxiety than the illness ever has."

The resolution: Despite making dozens of phone calls, Kuppersmith got nothing but confusing and contradictory answers when she tried to sort out the unexpected charge.

An agent for her insurer told her that her doctor hadn't gotten preauthorization for the testing. But in an email to Kuppersmith, a Genoptix employee told her the insurance company had denied the claim because molecular profiling was viewed as experimental.

A spokesperson for New York-based Empire Blue Cross Blue Shield, which handled part of Kuppersmith's claim, said her health plan "covers medically necessary genetic testing."

New York, one of 28 states with laws against surprise billing, requires hospitals to inform patients in writing if their care may include out-of-network providers, said attorney Elisabeth Benjamin, vice president of health initiatives at the Community Service Society, which provides free help with insurance problems.

A spokesperson for Mount Sinai said the hospital complies with that law, noting that Kuppersmith was given such a document in 2018 nearly one year before her bone marrow biopsy and signed it.

Benjamin said that's not OK, explaining: "I think a one-year-old, vague form like the one she signed would not comply with the state law and certainly not the spirit of it."

Instead of sending Kuppersmith a bill, Genoptix offered to help her appeal the denied coverage to CareFirst. At first, Genoptix asked Kuppersmith to designate the company as her personal health care representative. She was uncomfortable signing over what sounded like sweeping legal rights to strangers. Instead, she wrote an email granting the company permission to negotiate on her behalf. It was sufficient.

A few days after being contacted by KHN, Kuppersmith's insurer said it would pay Genoptix at the in-network rate, covering $1,200 of the $2,400 charge. Genoptix said it has no plans to bill Kuppersmith for the other half of the charge.

The takeaway: Kuppersmith is relieved her insurer changed its mind about her bill. But, she said: "I'm a relatively young, savvy person with a college degree. There are a lot of people who don't have the time or wherewithal to do this kind of fighting."

Patients should ask their health care providers if any outside contractors will be involved in their care, including pathologists, anesthesiologists, clinical labs or radiologists, experts said. And check if those involved are in-network.

"Try your best to ask in advance," said Jack Hoadley, a research professor emeritus at Georgetown University. "Ask, 'Do I have a choice about where [a blood or tissue sample] is sent?'"

Ask, too, if the sample will undergo molecular diagnostics. Since the testing is still relatively new and expensive most insurers require patients to obtain "prior authorization," or special permission, said Dr. Debra Regier, a medical geneticist at Children's National Hospital in Washington and an associate with NORD, the National Organization of Rare Diseases. Getting this permission in advance can prevent many headaches.

Finally, be wary of signing blanket consent forms telling you that some components of your care may be out-of-network. Tell your provider that you want to be informed on a case-by-case basis when an out-of-network provider is involved and to consent to their participation.

Bill of the Month is a crowdsourced investigation by Kaiser Health News and NPR that dissects and explains medical bills. Do you have a perplexing medical bill you want to share with us? Tell us about it here.

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Bill Of The Month: Pricey Genetic Test For Essential Thrombocythemia : Shots - Health News - NPR

Universityof Toronto researcherSachdev Sidhuand his collaborators are engineering antibody molecules that can neutralize the novel coronavirus in the body before it invades cells.

Sidhu (left) already leads a differentteam that received supportin the first round of federal funding. The goal of that project is to design antiviral medicines that block viral replication.

With our two funded projects, we are working to develop molecules that can target the virus both inside human cells and on the outside to prevent it from getting in, says Sidhu, who is a professor of molecular genetics in the Faculty of Medicine.

Rini has previously helped to determine how antibodies bind to and inactivate the SARS virus, the coronavirus that caused the outbreak in Asia more than 15 years ago. Also on the team isAlan Cochrane, a professor in the department of molecular genetics and an HIV virologist with expertise in viral RNA processing.

The antibodies will be engineered to block the so-called S-protein that forms spikes on the virus's surface. The spikes lock on to a protein called ACE2 on the surface of human cells to gain entry. Coating viral particles with synthetic antibodies should prevent the spikes from binding to ACE2.

Sidhu and Rini will also engineer antibodies that bind ACE2 to make it inaccessible to the virus. This type of engineered immunity surpasses the capacity of the bodys natural immune system since antibodies that react against self-proteins have been filtered out. If successful, the approach may obviate worries about viral mutations that can render drugs ineffective to new emerging viral strains becausethe host protein ACE2 does not change over time.

Sidhus team has advanced a technology called phage display to rapidly create and select human antibodies with desired biological properties, including blocking the virussspike protein. Over the last decade, his team has created hundreds of antibodies with therapeutic potential some of which are in clinical development through spin-off companiesand large pharmaceutical firms.

The group has demonstrated success with both approaches for inhibiting viral entry, having developed neutralizing antibodies that target the Ebola virus as well as antibodies that target the human host receptor of hantavirus or hepatitis C. Moreover, other research has shown that antibodies targeting SARS, a related virus whose genetic material is over 80 per cent identical to the one causing COVID-19, can clear infection in cells and mice.

Using phage display, in which tiny bacterial viruses called phages are instructed to create vast libraries of diverse antibodies, the team will select the antibodies that can kill the virus in human cells before testing them on mice and, eventually, patients. Experiments on mice could start within three to six months, Sidhu says.

In addition to creating antibodies tailored to the new virus from scratch, the researchers will also modify existing SARS-blocking antibodies so that they attack COVID-19 and provide an additional route to the development of a therapeutic.

Given the global spread of the virus, its possible that it will become endemic and circulate in the population like seasonal flu. And, like the flu, it could mutate into new strains that will evade acquired immunity and the vaccines that are being developed. By generating a panel of different antibodies, the researchers aim to stay one step ahead of the virus.

Our advances in antibody engineering technologiesand access to the complete genomes of the COVID-19 virus and its relatives provides us with an opportunity to create tailored therapeutic antibodies at a scale and speed that was not possible even a few years ago, says Sidhu.

Ultimately, we aim to optimize methods to the point where the evolution of new drugs will keep pace with the evolution of the virus itself, providing new and effective drugs in response to new outbreaks.

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Researchers at U of T developing antibodies to 'neutralize' novel coronavirus before it invades cells - News@UofT