Monthly Archives: May 2020

Viewpoint: COVID-19 food shortagesWhy the pandemic is a warning to embrace agricultural technology – Genetic Literacy Project

Posted: May 5, 2020 at 7:52 am

We will always remember a world before and after the COVID-19 pandemic. Before the novel coronavirus began making its way across the globeinfecting over a million people, killing thousands and straining food supply chainsfew individuals in developed nations worried about going hungry, let alone the threat posed by infectious disease. How things have changed in just a few short months.

Terrible as our current predicament is, it has afforded us an opportunity to rethink our relationship with technology and the importance of scientific innovation in helping us achieve higher standards of living. We now wonder what kind of world we can and should collectively shape. As the pandemic progresses and catapults us into to uncertain territory, the question worth asking is this: will we learn from our trying experience and embrace agricultural technology to build a more thoughtful, just and sustainable world, or return to our pre-COVID ways of thinking until the next pandemic rears its ugly head?

Our food during the COVID-19 pandemic and beyond

On April 5, 2020, The UN Food and Agriculture Ogranization (FAO) alerted the world about a looming food crisis as a result of the COVID-19 pandemic. Closed borders to migrant workers, disruption in the food supply chain and economic recession are likely to cause havoc in food production and delivery. Like healthcare workers, farmers and other food supply chain workers are our bloodline. Without agricultural workers, most of us would starve, yet modern society values them so little as to virtually ignore the issues they face.

People can stop doing many things in this time of global pandemic, but they cannot stop eating. Could malnutrition and starvation raise their ugly heads again in Europe, where food (which is mostly imported) has been abundant since the end of World War II? It is unthinkable, but to avert this outcome, we need efficiency and innovative technology to continue fueling large-scale, sustainable agriculture and drive a gentler, less aggressive bioeconomy to support our environment. But the developed world is pursuing these policies at a dangerously slow pace.

In Europe, compared to more impoverished regions of the world, only a very small percentage of the population is engaged in agricultural production. If cheap prices and food wastage are good indicators, the sense of where food comes from and the many perils farmers go through to produce and commercialize it seems to have been lost in sophisticated urban societies with a preference for organic, chemical-free, GMO-free, natural food and other such products.

We seem to understand little about where wheat, rice, maize, sugar, oil, coffee, cacao for chocolate, coconut oil, bananas or avocados come from. While we pick our inexpensive tropical fruit from our local supermarket shelves and check to see if it has a Rainforest Certified sticker, I wonder how many of us know about the lengths banana farmers in Ecuador or Honduras go to produce Sigatoka-free bananas, or rust-free coffee (both are fungal pathogens deadly to these crops) without using fungicides and fertilizers the crops require to stay healthy and produce reasonable yields. Like human epidemics, crop and animal epidemics exist too, and most people never hear or worry about them.

Our anti-chemical stance when it comes to food production is crippled by an embarrassing double standard. Many of us believe farmers are irresponsible for using pesticides and fertilizers to grow healthy crops, yet we wouldnt try to overcome a severe case of COVID-19 with good intentions, prayers and cups of hot tea with lemon and honey! Wed happily accept a state-of-the-art treatment prescribed by our doctors.

But perhaps its time we consistently live up to our convictions. If we think farmers dont need genetically engineered pest-resistant or drought-resistant crops, maybe we should manage COVID-19 in the future without a genetically engineered vaccine as well, opting for the slow, conventional route of vaccine development, or worse, no vaccine at all, like some fringe activists advocate.

I dont call out this double standard to be clever. We all acknowledge, most scientists included, that agricultural companies and farmers may abuse the use of agrochemicals to safeguard yields, and that we need to find alternatives to potentially harmful pesticides when possible. The broader point is that biotech crops are a crucial part of that effort.

There is also agreement that we need to use less land, energy, water and fewer agricultural inputs to promote sustainable agriculture, slow down climate change and maintain healthy biodiversity, which ultimately will help our species survive into the future. Genetically engineered crops contribute to all these goals.

I applaud the slow food and urban farming movements, because these are important and creative initiativesyet they are mainly solutions for affluent societies. They dont work nearly as well for poor societies that need efficient industrial agriculture, especially to feed densely populated cities.

Agriculture vs. the environment

Should we go back to our imagined idyllic farming past? I dont think we can with a population of seven billion people that continues to grow. The industrial revolution started in Europe in the 18th century and brought significant social changes that resulted in an increase in population and urbanization. In 18th century Europe, small-scale farming gave way to intensive, efficient and large-scale agriculture that was required to feed an ever-growing urban population of new professionals and factory workers that no longer worked in the fields, often producing poor yields.

The explosive population growth in Europe and North America began much earlier than in less developed regions of Asia, Africa and Latin America, which are still growing today. Towards the late 20th century and after recovering from two world wars, Europes main problem was not feeding more people, but maintaining a healthy population and boosting economic growth (food could be imported) to maintain a high standard of living.

The poorer countries in the East and South watched the Wests industrialization and increase in wealth with wonder and, naturally, wanted to emulate this glorious path out of poverty and into progress. These nations began to copy rich countries shortly thereafter. Their populations grew exponentially, which small-scale subsistence agriculture could no longer support. People left the fields and moved to cities in search of jobs. Spectacular advances in the 1960s and 1970s in agricultural science collectively known as the Green Revolution greatly increased agricultural output and averted famine, especially in Asia and Latin America.

Like in Europe and North America, agriculture also became industrialized and mechanized: irrigation systems were installed, plant breeding produced more and better varieties of crops and chemical fertilizers and pesticides were developed. The paradigm was what the plant needed (water, food, no competition from weeds and no pests), the plant got, so farmers changed the plants environment to suit its needs. For once, Malthus and his dire predictions of population growth exceeding food production were proved wrong.

With a misguided sense that we had enough food for everyone, many commentators adopted the mantra that the worlds remaining food-shortage problem was one of distribution, not of insufficient production. Agricultural innovation after doing so much good, paradoxically, became the main enemy of the environment and biotechnologists the bad guys.

Public funding for agricultural research and extension dwindled in most countries and the slack was picked up mainly by the private sector. The Monsantos of the world were born and were universally hated. Nonetheless, the agricultural biotechnology revolution had begun, with an important paradigm shift from the Green Revolution. Instead of changing the environment to suit the plant, the plant could be adapted to a changing environment by genetic engineering and the use of microbes.

Europe slows the biotech revolution

By the late 20th century, the rules to industrialization had changed dramatically, mainly in the European Union, which also dominated the UN agencies for food and the environment.

New and stringent agricultural policies came from this affluent food-importing region, with repercussions for everybody else during the first years of the 21st century, after genetically modified crops were first commercialized.

EU and UN programs provided aid funding for biosafety projects related to environmental conservation in many developing countries in Southeast Asia, Africa and Latin America. Most programs came with strings attached to the new European policies on agricultural biotechnology. Generous funds were disbursed to ministries of the environment (not agriculture), although the programs were concerned with new agricultural technologies.

Bizarrely and very unfortunately, agriculture and biotechnology became the environments worst enemies under these UN programs. The term biosafety was borrowed from microbiology, yet the programs were not designed to promote biosafety against crop, animal or human pathogens to avert pandemics like SARS, avian flu or COVID-19, but to safeguard the environment, including human and animal health, against the perceived risks of biotechnology.

Specifically, the biosafety guidelines for genetically modified crops (or Living Modified Organisms) developed through conventional genetic engineering were designed to manage the (exaggerated) biological risks set by the Convention of Biological Diversity, through its famous Cartagena Protocol on Biosafety of Biotechnology.

Most developing countries, trained in biosafety of biotechnology by generous European and UN programs blindly adopted Eurocentric guidelines to the detriment of local agriculture and the environment. Without appropriate policies and lacking incentives for innovation from demanding markets and low prices, they are stuck with outdated and low-yielding technologies that cause great damage to the environment.

Today, many countries in Latin-America like Ecuador, Peru, Bolivia and Guatemala, are trying to change their ill-conceived and hopelessly outdated legislation as demands increase to solve urgent food production problems. The last decade has also witnessed fast development of other biotechnologies that can aid these efforts, such as CRISPR genome editing and gene drives that span medical, veterinary, environmental and industrial applications. Shaping biotechnology policies from ministries of the environment makes no sense in these circumstances.

Will coronavirus be a game changer?

Maybe a couple of decades ahead of the biotech revolution, the information technology or infotech revolution (Al, IoT, 3D printing, robotics and drones, blockchain) took over a globalized world. Now we have entered a third era of the agritech revolution, where exponential technologies merge to change forever the way we live and grow food. This technical progression unsettles and scares many people who view the technological future with great distrust and angst.

Then SARS-Cov-2 appeared (not as a surprise to many scientists) and changed everything, for better or worse. I personally believe it will be for the better in the long term. For a start, people everywhere are interested in learning a little about biology and virology, including IT programmers who could be inspired to learn the flawless biological designs of nature and incorporate them into their work.

In the 2020s, the approach of synthetic biology and the increasing involvement of newly trained young bioengineers in both industrialized and developing countries will create a domestication and democratization of biotechnology. It is imperative not to tie the hands of this second generation of biotechnologists with ill-conceived ideas of risk and precautionary policies. Instead, it is key to craft robust, pragmatic, science-based and coherent biotechnology policies, for the successful development of the bioeconomy and to maintain human, animal and environmental health.

The mantra that there is enough food and we only have a problem of distribution is not valid today. The tools of biotech and infotech have already been developed and deployed in certain regions to address the unique challenges they face. They will be able to grow food efficiently in future times of crisis, provided they arent made to face more regulatory hurdles. Do we envisage a better and fairer post-COVID world, or will we return to business as usual and keep the status quo of social inequality? The choice is ours, more now than ever before.

Maria Mercedes Roca is a Latin-American/British virologist and biotechnologist. She splits her time between Mexico and Bolivia and wrote this article during lockdown from the UK.

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Viewpoint: COVID-19 food shortagesWhy the pandemic is a warning to embrace agricultural technology - Genetic Literacy Project

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UNITY Biotechnology, Inc. to Announce First Quarter 2020 Financial Results – ForexTV.com

Posted: May 5, 2020 at 7:52 am

SAN FRANCISCO, May 04, 2020 (GLOBE NEWSWIRE) UNITY Biotechnology, Inc. (UNITY) [NASDAQ:UBX], a biotechnology company developing therapeutics to extend healthspan by slowing, halting or reversing diseases of aging, today announced it will report financial results for the first quarter ended March 31, 2020, on Thursday, May 7, 2020 after NASDAQ market close.

UNITY will not conduct a conference call in conjunction with the financial results press release.

About UNITYUNITY is developing therapeutics to extend healthspan with an initial focus on cellular senescence. UNITY believes that the accumulation of senescent cells is a fundamental mechanism of aging and a driver of many common age-related diseases. Cellular senescence is a natural biological state in which a cell permanently halts division. As senescent cells accumulate with age, they begin secreting inflammatory factors, proteases, fibrotic factors, and growth factors, that disturb the tissue micro-environment. This collection of secreted proteins is referred to as the Senescence Associated Secretory Phenotype, or SASP. UNITY is developing senolytic medicines to eliminate senescent cells and thereby stop the production of the SASP, which UNITY believes addresses a root cause of age-related diseases. By stopping the production of the SASP at its source, UNITY believes senolytic medicines could slow, halt, or reverse diseases such as osteoarthritis and age-related eye diseases. More information is available atwww.unitybiotechnology.comor follow us onTwitter.

InvestorsEndurance AdvisorsPeter Rahmer / Mike Zanoni[emailprotected] / [emailprotected]

MediaCanale CommunicationsJason Spark[emailprotected]

GlobeNewswire,is one of the world's largest newswire distribution networks, specializing in the delivery of corporate press releases financial disclosures and multimedia content to the media, investment community, individual investors and the general public.

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Covid-19 has shuttered labs. It could put a generation of researchers at risk – STAT

Posted: May 5, 2020 at 7:49 am

Scientists are skilled at tackling unexpected problems that threaten the integrity of their experiments it comes with the territory. But the coronavirus pandemic poses a new and entirely unprecedented challenge.

The global health emergency has shut down scientific research labs across the country in a crisis that has left some scientists scrambling to save their work and has left others grieving the loss of experiments they had dedicated months or even years to carrying out. Many are grappling with an overwhelming sense of uncertainty about how theyll continue their work.

The situation has hit early-career researchers particularly hard. Their funding and their futures depend on quickly gathering data to publish in prestigious journals. Without additional financial support and an extension of tenure clocks, some scientists who have just started their own labs fear the delays to their studies may be too disruptive to overcome.

Early-career scientists will be very vulnerable, said Cullen Taniguchi, assistant professor at University of Texas MD Anderson Cancer Center. Taniguchi said it will be crucial to properly support researchers when labs reopen or, he warned, we may lose a whole generation of researchers because of this.

Despite these struggles, many researchers say that shutting down the labs was necessary to stem the spread of the virus. And some labs are still up and running, though not all are doing so at full capacity. But for scientists whose work has been deferred, the closures have fueled a devastating ripple effect of consequences, both big and small.

Even when laboratories are reopened, it may take months to a year for research to resume as normal.

I have [new hires] in the lab that havent even met each other physically, said Alice Soragni, a cancer researcher and assistant professor who runs a lab at the University of California, Los Angeles. There is a lot of training that needs to have happened that hasnt happened, she added.

STAT spoke to scientists across the country to better understand the wide-ranging impacts of lab shutdowns.

Scientists have transitioned from long hours in the laboratory to working from home but the abrupt halt to their research projects has left a lingering sense of disorientation for researchers like Kathleen Beeson, a sixth-year graduate student at Oregon Health and Science University.

Like many of her colleagues, Beeson was caught off guard by her labs closure.

We were given a weeks notice,she said. Immediately, I and others were in a race to finish experiments, collect any data that we could, and get the lab prepared for a minimum of six weeks of shutdown.

Beeson had been completing a final experiment for a publication she needs to earn her Ph.D. and move onto a postdoc research position at Harvard Medical School.

The shutdown has upended Beesons research, which involves measuring electrical activity in the brains of genetically engineered mice. Her work aims to describe how proteins at the junction of nerve cells help transmit chemical signals an important step in understanding neurological dysfunctions such as epilepsy.

While other scientists were able to freeze cells or preserve tissue samples in formaldehyde, Beesons research relied on analyzing freshly dissected brain tissue. Because she could no longer come into the lab, she had to sacrifice most of her mouse colony, which she had painstakingly raised from one male and one female to approximately 200 animals.

In the end, I found myself euthanizing mice by the masses in the university basement, she said. It was the punctuation on a sad and disorienting week.

Beeson said it will likely take her months to raise enough animals for experiments again. In the meantime, she has been working on her Ph.D. dissertation and a second publication from home although not at the pace that she had hoped for.

I applaud anyone making any progress, on anything, during this time, she said. Sometimes my progress is processing my grief.

Disruptions to research and long startup times pose an especially daunting challenge to early-career scientists who have just a few years to establish themselves as experts in their fields and obtain critical funding for their laboratories.

With experiments on hold, some early-career scientists cant collect the kind of preliminary data that is crucial for them to compete with more established researchers who have a decade or more of experimental findings to build on.

[All researchers] are impacted but I think there are exquisite challenges for early-career investigators like myself, said UCLAs Soragni.

To protect early-career scientists, the NIH has extended the time frame for which researchers can be considered early stage investigators a status that helps government institutes and centers prioritize funding for scientists running new laboratories. The agency has also relaxed some of the eligibility requirements for maintaining grants and added additional flexibility for spending funds.

Despite these welcome efforts, early-career researchers especially those lacking data needed to apply for new grants remain in a precarious position. Soragni and others said they hoped the NIH would take the impact of Covid-19 into account and temporarily adjust its criteria for reviewing applications. However, the agency has recommended that scientists without enough preliminary data submit their applications at a later date.

For Soragni, the most difficult challenge has been the uncertainty.

You are kind of left not knowing what you should do. Should you be ramping up completely? But what if you are switched down again?

Alice Soragni, UCLA cancer researcher

We really dont know if we are going to have a second wave of infections and what will be the consequences, she said. You are kind of left not knowing what you should do. Should you be ramping up completely? But what if you are switched down again? Should you be hiring? Will the economy bounce back? What is going to happen to your grants?

We are just at a more vulnerable stage of our career, Soragni said. I believe we may lose some laboratories to this, so that will be very painful to witness.

The shutdowns have taken a toll not only research, but also on the close professional relationships at the heart of scientific collaboration.

For Stephanie Campos, Covid-19 meant that she would not complete her research or be able to say goodbye to her mentor, Walter Wilczynski, in person. Campos had come to Georgia State University for a postdoctoral fellowship with Wilczynski, a pioneer in the field of behavioral neuroscience and the first director of the universitys Neuroscience Institute. But after 37 years of research, the lab was scheduled to close this summer after Wilczynskis cancer, once in remission, returned.

Campos and her colleagues were wrapping up their research a study of the brain activity in lizards aimed at unraveling the neural underpinnings of social behaviors when the pandemic hit. The lab shuttered earlier than expected.

With the laboratory closed, Campos has been limited to writing manuscripts from home and analyzing old videos of lizard behavior. She cant see Wilczynski who is immunocompromised again in person before she moves to a new role as a visiting assistant professor at Swarthmore College.

[This experience] has really affected me emotionally in the way that I knew I was going to be his last student, Campos said. And so I had really wanted to get as much as I could.

With Georgia easing restrictions on social distancing, there is a possibility that Campos could return to the lab late in the summer, but she is still unsure if returning to work would be socially responsible. Instead, she is planning on mailing the bulk of her delayed research project which involves 68 lizard brains preserved in vials of paraformaldehyde to Pennsylvania, where she will begin work in August.

Campos credits Wilczynski, who was at times too fatigued to read papers, for guiding her through the gauntlet of an academic job search and giving her the confidence to continue in academia.

His kindness during this time is what Ill remember the most, Campos said. For me it is all about the personal connection, how well your mentors make you feel. Those are the things that I will take away.

Waiting for their labs to reopen, principal investigators are steeling themselves for the months of effort that will be needed to reestablish the rhythms of a productive laboratory.

Theres a mountain of work to muddle through before experiments can get off the ground again.

We will have to first retest [our equipment] to make sure it is working, regrow our [bacterial] cultures, which takes a while, before we can even consider doing an experiment, said Eric Rubin, an immunology and infectious diseases researcher and professor at the Harvard T.H. Chan School of Public Health. Rubin also the editor-in-chief the New England Journal of Medicine.

Regrowing bacteria in Rubins laboratory is not a job for the impatient. The focus of his studies, Mycobacterium tuberculosis, causes tuberculosis and kills more people worldwide than any other infectious pathogen. M. tuberculosis also grows approximately 50 times more slowly than other microorganisms. Experiments that would take a day with other commonly studied bacteria typically take weeks in the Rubin lab.

When laboratories closed, Rubins team was in the midst of testing a batch of promising drug compounds for the ability to kill the bacteria. To resume the study, researchers will have to thaw out stocks of frozen bacteria and coax them to replicate in liquid broth.

We normally always have things growing so that we can grab them and do our next experiment, said Rubin. [But now] it will likely take four months before we will have enough cells to do experiments at full tilt again.

Restarting research may take even longer up to a year for those working with laboratory animals, such as Subhash Kulkarni, a scientist and assistant professor at Johns Hopkins University School of Medicine.

In 2017, Kulkarni showed that, contrary to established dogma, nerve cells lining the intestines continue to grow and divide in adult animals. To understand how this discovery could lead to new treatments for digestive disorders, Kulkarni had begun analyzing how neurons behaved over the lifespan of a mouse. This project required raising genetically engineered mice at staggered times to have enough of each age group at the start of the study.

With his lab closed, the entire effort will have to be repeated once Kulkarni is allowed to work again. That timeline is daunting.

Think of this as the time when the planets are in perfect alignment, Kulkarni said. Once that time is lost, making the next time requires [new] breedings, which can take anywhere from six to 12 months.

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CytoDyn Reports Strong Results from eIND COVID-19 Patients Treated with Leronlimab; Majority of Patients Have Demonstrated Remarkable Recoveries -…

Posted: May 5, 2020 at 7:49 am

54 eINDs approved by FDA and 49 patients have been treated with leronlimab thus far

VANCOUVER, Washington, April 30, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (CYDY), (CytoDyn or the Company"), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, today announced updates on 49 COVID-19 patients who have received leronlimab under the U.S. Food and Drug Administrations (FDA) emergency Investigational New Drug (eIND) program:

Eleven (11) Patients in NY hospital: All treated patients were in Intensive Care Units (ICU) because of acute respiratory failure, eight of whom were intubated (placed on mechanical ventilation). One patient was not intubated because of poor baseline pulmonary status (history of lung cancer and had undergone bilateral upper lobectomy). Seven patients were organ-transplant recipients (six patients were renal-transplant recipients and one patient had a history of heart transplant) and were on immunosuppressive regimen. Ten patients were on dialysis and nine were on vasopressors during hospitalization. Despite their pre-existing and severe conditions, we believe we were able to save the lives of four patients. All patient blood samples were evaluated and important powerful results from the effect of leronlimab were demonstrated in almost all of these patients. This data has been submitted to a prestigious journal and we expect the publication on Friday, May 1.

Twenty-three (23) patients in Southern California hospital: Six patients were in critical condition (intubated) and 17 patients were severely-ill, needing oxygen support. No death was reported. Out of 6 critical patients, all were intubated patients, 3 were extubated (taken off ventilator), 2 patients remain relatively stable and still breathing with the assistance of a ventilator and one patient has shown deterioration in respiratory parameters. Of 17 severe condition (but not critical) patients, 11 patients demonstrated improvement in respiratory parameters (8 of them were discharged from hospital, including one patient in the news, Samantha Mottet), 2 patients remain relatively stable, 2 have shown deterioration in respiratory parameters and information is pending for 2 recently treated patients.

Three (3) patients in Georgia hospital: All three ICU patients were intubated and two of them had renal failure at the start of leronlimab treatment. Of these 3 patients, 2 were extubated (taken off ventilator) and 1 patient remains on a ventilator but improving.

One (1) patient in another NY hospital: Patient was taken off oxygen and discharged from hospital after leronlimab treatment.

One (1) patient in Northern California hospital: Patient is now weaning from ventilator and transferred to rehabilitation hospital.

Updates are pending for 10 other patients. Five additional patients have been approved to receive leronlimab under eINDs, which increases the total eINDs approved by the FDA to 54 patients.

Bruce Patterson, M.D., Chief Executive Officer and founder of IncellDx, a diagnostics company and an advisor to CytoDyn, expanded on these findings by stating, We are excited that patients are responding extremely well to leronlimab as expected from the novel mechanism of COVID-19 pathogenesis we discovered and will be reporting in the coming days.

Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn said, We believe these results, although anecdotal, are very impressive and the number of patients treated under eIND is rapidly increasing. The enrollment for our Phase 2 double-blind and Phase 2b/3 trials is moving along rapidly and we believe the results from both studies will be very powerful due to the mechanism of action (MOA) of affecting the viral load and restoring the immune system. With our first major paper very close to publication, we expect to have a second paper published shortly thereafter, as our MOA is as unique as our results.

About Coronavirus Disease 2019CytoDyn is currently enrolling patients in two clinical trials for COVID-19, a Phase 2 randomized clinical trial for mild-to-moderate COVID-19 population in the U.S. and a Phase 2b/3 randomized clinical trial for severe and critically ill COVID-19 population in several hospitals throughout the country.

Story continues

SARS-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 completed the filing of its BLA in April 2020 to seek FDA approval for leronlimab as a combination therapy for highly treatment experienced HIV patients. 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. Forward-looking statements specifically include statements about leronlimab, its ability to have positive health outcomes, the possible results of clinical trials, studies or other programs or ability to continue those programs, the ability to obtain regulatory approval for commercial sales, and the market for actual commercial sales. 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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CytoDyn Reports Strong Results from eIND COVID-19 Patients Treated with Leronlimab; Majority of Patients Have Demonstrated Remarkable Recoveries -...

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CytoDyn (OTC: CYDY) Reports Strong Results from eIND COVID-19 Patients Treated with Leronlimab; Majority of Patients Have Demonstrated Remarkable…

Posted: May 5, 2020 at 7:49 am

VANCOUVER, Washington, Apr 30, 2020 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, today announced updates on 49 COVID-19 patients who have received leronlimab under the U.S. Food and Drug Administrations (FDA) emergency Investigational New Drug (eIND) program:

One (1) patient in another NY hospital: Patient was taken off oxygen and discharged from hospital after leronlimab treatment.

One (1) patient in Northern California hospital: Patient is now weaning from ventilator and transferred to rehabilitation hospital.

Updates are pending for 10 other patients. Five additional patients have been approved to receive leronlimab under eINDs, which increases the total eINDs approved by the FDA to 54 patients.

About Coronavirus Disease 2019

CytoDyn is currently enrolling patients in two clinical trials for COVID-19, a Phase 2 randomized clinical trial for mild-to-moderate COVID-19 population in the U.S. and a Phase 2b/3 randomized clinical trial for severe and critically ill COVID-19 population in several hospitals throughout the country.

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 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 a Phase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.

About CytoDyn

CytoDyn 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 completed the filing of its BLA in April 2020 to seek FDA approval for leronlimab as a combination therapy for highly treatment experienced HIV patients. 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 at http://www.cytodyn.com.

Forward-Looking Statements

This press release contains 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. Forward-looking statements specifically include statements about leronlimab, its ability to have positive health outcomes, the possible results of clinical trials, studies or other programs or ability to continue those programs, the ability to obtain regulatory approval for commercial sales, and the market for actual commercial sales. 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 Form 10-K, and any risk factors or cautionary statements included in any subsequent Form 10-Q or Form 8-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.

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BioMarin broadens its gene therapy horizons with a new R&D alliance in rare cardio cases – Endpoints News

Posted: May 4, 2020 at 4:45 pm

Hours after Gilead announced that an NIH trial testing their antiviral drug remdesivir in Covid-19 patients had succeeded, NIAID director Anthony Fauci sat on a couch in the Oval Office and gave the world the top-line readout.

The drug induced a 31% improvement on the primary endpoint of time to recovery: 11 days in the drug arm compared to 15 days in the placebo arm, he said, adding that patients taking the drug appeared less likely to die, with an 8% mortality rate in the drug arm compared to 11% in patients given the placebo.

The mortality data were not yet statistically significant, he cautioned but were trending in the right direction. Fauci, surrounded by President Trump, Vice President Mike Pence and several other advisors, said the news was a very optimistic sign in the hunt for treatments to fight the virus.

Although a 31% improvement doesnt seem like a knockout 100%, it is a very important proof of concept, he said. Because what it has proven, is that a drug has blocked this virus.

Fauci said more details would come and that the study would be submitted to a peer-reviewed journal. Trump, who deferred to Fauci in giving the readout, echoed Faucis commentary.

Its a beginning, that means you build on it, Trump said. But its a very positive event.

Shortly after the briefing, the New York Times reported that the FDA was preparing to issue an emergency use authorization for the drugs use in Covid-19. In an email to Endpoints News, the FDA did not confirm or deny the Times report, but a spokesperson said the agency has been engaged in sustained and ongoing discussions with Gilead Sciences regarding making remdesivir available to patients as quickly as possible, as appropriate.

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BioMarin broadens its gene therapy horizons with a new R&D alliance in rare cardio cases - Endpoints News

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BioMarin Extends Gene Therapy Leadership with DiNAQOR in a Preclinical Collaboration and License Agreement to Develop Gene Therapies for Rare Genetic…

Posted: May 4, 2020 at 4:45 pm

SAN RAFAEL, Calif., May 3, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the company has entered into a preclinical collaboration and license agreement with DiNAQOR AG (DiNAQOR), a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. DiNAQOR will receive an undisclosed upfront payment and is eligible to receive development, regulatory and commercial milestones on product sales in addition to tiered royalties on worldwide sales. The company did not disclose financial terms. BioMarin management reiterated its 2020 GAAP net income guidance of $20 to $80 million, inclusive of this collaboration.

The license initially covers DiNAQOR's lead program, DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM). Additionally, the companies will collaborate on several of DiNAQOR's other pipeline programs, and BioMarin has the option to extend the license to include these additional programs on similar terms. Reflecting the long-term commitment to the collaboration, BioMarin is simultaneously investing in DiNAQOR.

"With this agreement, BioMarin is continuing to apply its gene therapy know-how and manufacturing expertise in new areas like cardiology," said Jean-Jacques Bienaim, Chairman and Chief Executive Officer at BioMarin. "This collaboration extends our global leadership position in gene therapy and boosts our potential to transform the lives of patients worldwide with rare genetic cardiomyopathies."

"We are thrilled to collaborate with the researchers at DiNAQOR to conduct this pioneering work on the development of gene therapies for inherited cardiomyophathies," said Lon Cardon, Chief Scientific Strategy Officer and Senior Vice President at BioMarin. "We believe there is tremendous potential in combining our experience in gene therapy research and development with DiNAQOR's in-depth knowledge of genetic heart diseases."

DiNAQOR was founded and is led by several leading pharmaceutical and biotechnology executives and academics with deep cardiology and gene therapy expertise. The company's holistic approach to gene therapy is focused on gene therapies for the heart that deliver a medical solution that can safely deliver gene therapies to the heart muscle, ensure transduction of the cardiac cells, and limit the exposure of the therapy to other organs.

"BioMarin is a global leader in rare disease research, development and commercialization, and their commitment to DiNA-001 is a powerful validation of DiNAQOR's gene therapy platform," said Dr. Johannes Holzmeister, Co-Founder, Chairman and CEO at DiNAQOR. "We believe our platform has many potential applications and this milestone agreement will enable us to invest in expanding our genetic medicine pipeline."

"Momentum for gene therapies continues to build, and BioMarin has demonstrated tremendous scientific, clinical, and manufacturing leadership and expertise in the space," said Thomas Voit, M.D., Ph.D., Co-Founder and Chief Scientific Officer at DiNAQOR and Director of the Biomedical Research Centre at the Great Ormond Street Hospital and the UCL Institute of Child Health, University College London. "We are looking forward to combining our strengths to expand the promise of gene therapy treatments by targeting the heart muscle to treat rare genetic cardiomyopathies."

About HCM and MYPBC3

Hypertrophic cardiomyopathy (HCM) is one of the most common genetic heart diseases, with about 500,000 patients diagnosed with HCM worldwide. Up to 60% of HCM cases have a genetic origin, and it is estimated that 40% of those have mutations in MYBPC3, the gene that encodes cardiac myosin-binding protein C (MyBP-C).

HCM affects the heart muscle, causing the muscle to enlarge. HCM patients have an increased risk of developing heart failure and life-threatening arrhythmias. There are no approved pharmacological treatment options available that address the underlying disease biology of HCM and invasive surgery or heart transplantation may be the only options available for patients with advanced disease.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare genetic diseases. The Company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit http://www.biomarin.com. Information on BioMarin's website is not incorporated by reference into this press release.

About DiNAQOR

Founded in 2019, DiNAQOR AG is a global gene therapy platform company focused on advancing novel solutions for patients suffering from heart disease. The company's lead preclinical program, DiNA-001 is focused on the treatment of MYBPC3-linked cardiomyopathy. DiNAQOR is headquartered in Pfffikon, Switzerland, with additional presence in London, England and Boston, Massachusetts (US). For more information visit http://www.dinaqor.com.

Forward Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: BioMarin's expectations regarding the announced collaboration, the prospects for the lead and follow on pipeline products and it's 2020 GAAP profitability. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning the programs; the ability to manufacture the product candidates, BioMarin's revenue for 2020, especially with the possible impact of COVID-19, and those other risks detailed from time to time under the caption "Risk Factors" and elsewhere in BioMarin's Securities and Exchange Commission (SEC) filings, including BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, and future filings and reports by BioMarin. BioMarin undertakes no duty or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

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BioMarin Pharmaceutical Inc.

(415) 455-7558

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SOURCE BioMarin Pharmaceutical Inc.

Company Codes: NASDAQ-NMS:BMRN

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BioMarin Extends Gene Therapy Leadership with DiNAQOR in a Preclinical Collaboration and License Agreement to Develop Gene Therapies for Rare Genetic...

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LifeArc, MRC create 16m fund to set up gene therapy hubs – PharmaTimes

Posted: May 4, 2020 at 4:45 pm

The Medical Research Council (MRC) and independent medical research charity LifeArc are streaming 16 million into establishing a network of gene therapy innovation hubs.

The centres will offer clinical grade viral vectors as well as translational and regulatory guidance to support academic-led patient trials of new gene therapies.

Operating as 'centrally coordinated facilities', the hubs aim to address challenges faced by academics as they seek to advance novel gene therapy research into early stage clinical trials, such as a shortage of viral vector production capacity and a complex and evolving translational pathway for gene therapies.

LifeArc and the MRC said they will create the network by providing UK-based research organisations with grants for up to five years, to support the costs associated with expanding or repurposing existing viral vector production centres.

The selected centres, or hubs, will also have access to LifeArcs translation advice and support.

We hope that through this unique collaboration with the MRC, LifeArc can offer its funding and expertise in technology transfer and translational science to support the progression of promising gene therapies, said Dr Melanie Lee, the charity's chief executive. Translation of advanced therapies will be a core focus of LifeArcs future strategy for delivering significant new patient benefits.

MRC executive chair Professor Fiona Watt added: Through this partnership, we aim to support clinical development of the most exciting gene therapy projects from the UKs world-leading academic researchers. This investment will streamline and accelerate progress towards a new generation of genetic medicines for patients.

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Taysha Gene Therapies hits the ground running with $30M, 15 programs – FierceBiotech

Posted: May 4, 2020 at 4:45 pm

The team that developed Zolgensma is back for round two. A group of former AveXis executives and investors unveiled a new gene therapy company, and theyre wasting no time. With 15 programs, $30 million in seed funding and an unrivaled partnership with UT Southwestern Medical Center, Taysha Gene Therapies plans to be in the clinic by the end of the year.

Its working on adeno-associated vector (AAV) gene therapies for monogenic diseasesthat is, diseases caused by a defect in a single geneof the central nervous system. Tayshas lead program targets GM2 gangliosidosis, a very rare disorder that progressively destroys nerve cells in the brain and spinal cord, but the companys portfolio includes conditions that are more prevalent.

The company plans to start clinical trials for three more programs by the end of 2021: treatments for Rett syndrome, a neurodevelopmental disorder; SURF1 deficiency, the most frequent cause of Leigh syndrome; and a SLC6A1 genetic epilepsy, which is similar to Dravet syndrome, Taysha CEO and co-founder R. A. Session II told FierceBiotech.

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And thats not allTaysha has the option to pick up four more prospects from UT Southwestern.

The reason why we can tackle 15 programs with the option to four additional programs is were able to focus on what we do best and were able to allow our collaborators to focus on what they do best, Session said.

Under their partnership, UT Southwestern is working on discovery and preclinical work all the way through IND-enabling studies. It has its own GMP viral manufacturing site to support that work, as well as clinical development, which Taysha will pick up. The company will also take care of regulatory strategy, commercial manufacturing and commercialization. Its a skill set that Tayshas management team honed at AveXis as itdeveloped the gene therapy that eventually became Novartis spinal muscular atrophy treatment Zolgensma.

We essentially flew the plane and built it at the same time when we were developing AveXis We have people with the experience of being able to develop, manufacture and commercialize a gene therapy program and were marrying that with a best-in-class academic research institution, Session said.

RELATED: FDA lets Novartis off the hook in Zolgensma data manipulation

Dividing the labor creates this engine for innovation that allows the partners to advance many programs in parallel, Session added. With about 50 people in its gene therapy unit, UT Southwestern can develop a capsidthe protein shell of a virusfor treatment delivery or get a candidate into animal models much more quickly than a biotech going it alone, he said. The same goes for late-stage development and commercialization on Tayshas side.

For its initial programs, Taysha is focusing on AAV gene therapies because the team knows they work.

AAV9 is the best way we have to treat monogenic CNS disease. If its not broke, dont fix it, Session said. We know how to effectively dose AAV9; we know its safe, effective and efficient. And we know its scalable. These are problems we had to solve at our previous company.

But, moving forward, Taysha and UT Southwestern are working on new technologies, including an AAV delivery platform that would allow for the redosing of gene therapies as well as an AAV capsid platform aimed at improving target delivery.

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Taysha Gene Therapies hits the ground running with $30M, 15 programs - FierceBiotech

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AskBio Takes Over French Gene Therapy Company to Treat Alzheimers and… – Labiotech.eu

Posted: May 4, 2020 at 4:45 pm

The Paris-based BrainVectis, a biotech developing gene therapies for Huntingtons disease and Alzheimers disease, has been acquired by the US clinical-stage gene therapy company AskBio.

AskBios gene therapy experience and manufacturing capacity will help to accelerate BrainVectis lead candidate for Huntingtons into phase I testing. In return, AskBio gets to expand its list of target indications.

AskBio recognized the scientific merit of the work at BrainVectis as an opportunity to strengthen our central nervous system clinical pipeline, Robin Fastenau, VP of Communications for AskBio, told me. No financial details about the acquisition were disclosed.

BrainVectis lead candidate focuses on increasing the expression of a protein called CYP46A1. This enzyme is key for turning excess cholesterol into a derivative that can be cleared from the brain into the blood. It is also reduced in Huntingtons and Alzheimers patients, allowing toxic levels of cholesterol to build up in the brain. By increasing the levels of this enzyme, BrainVectis aims to restore normal cholesterol metabolism and improve the clinical outcome.

So far, BrainVectis lead gene therapy candidate has shown proof-of-concept in animal models of Huntingtons. It also received orphan drug designation from the European Commission last year.

According to Nathalie Cartier-Lacave, CEO and Founder of BrainVectis, AskBio offers a strong cell-line manufacturing process. In particular, it can manufacture a range of viral vectors, including BrainVectis vector of choice: the adeno-associated virus.

This powerful adeno-associated virus technology and Askbios expertise in clinical applications will allow us to rapidly go to clinical application in patients, Cartier-Lacave told me.

There are currently no approved treatments able to slow down the progress of Huntingtons and Alzheimers. Combined with aging populations in developed countries, these debilitating diseases are creating a healthcare challenge. Many companies are trying and sometimes failing to develop drugs able to stop the progression of Alzheimers, for example, as such a drug could make a huge impact on society.

According to Cartier-Lacave, Huntingtons disease is the first target for the company going forward. The disease is caused by a mutation in a gene called HTT that is important for the function of nerve cells. Its a very severe disease for which we think the treatment may not only decrease the toxic mutated protein, but also preserve neurons from death, she added.

Gene therapies are becoming ever more popular in the biotech industry for their potential to tackle previously incurable conditions. BrainVectis is one of several companies aiming gene therapies at the brain; another is the French company Lysogene. However, there are major challenges with developing gene therapies for the brain, for example, getting the therapy past the blood-brain barrier and into the brain tissue.

AskBio might be up to the challenge, as it has taken several gene therapies to the clinic. Its proprietary treatment for the neuromuscular indication Pompe disease is currently in phase I/II, and it has licensed its technology to several big pharmaceutical companies. Those currently in clinical development include treatments for Duchenne muscular dystrophy (Pfizer), hemophilia (Takeda), and spinal muscular atrophy (AveXis), which was approved by the FDA last year, and is awaiting marketing approval by the EMA.

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AskBio Takes Over French Gene Therapy Company to Treat Alzheimers and... - Labiotech.eu

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Monthly Archives: May 2020

Viewpoint: COVID-19 food shortagesWhy the pandemic is a warning to embrace agricultural technology – Genetic Literacy Project

UNITY Biotechnology, Inc. to Announce First Quarter 2020 Financial Results – ForexTV.com

Covid-19 has shuttered labs. It could put a generation of researchers at risk – STAT

CytoDyn Reports Strong Results from eIND COVID-19 Patients Treated with Leronlimab; Majority of Patients Have Demonstrated Remarkable Recoveries -…

CytoDyn (OTC: CYDY) Reports Strong Results from eIND COVID-19 Patients Treated with Leronlimab; Majority of Patients Have Demonstrated Remarkable…

BioMarin broadens its gene therapy horizons with a new R&D alliance in rare cardio cases – Endpoints News

BioMarin Extends Gene Therapy Leadership with DiNAQOR in a Preclinical Collaboration and License Agreement to Develop Gene Therapies for Rare Genetic…

LifeArc, MRC create 16m fund to set up gene therapy hubs – PharmaTimes

Taysha Gene Therapies hits the ground running with $30M, 15 programs – FierceBiotech

AskBio Takes Over French Gene Therapy Company to Treat Alzheimers and… – Labiotech.eu

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