Daily Archives: February 15, 2022

Later this year, the now-Nobel prize-winning paper authored by Jennifer Doudna and Emmanuelle Charpentier in which they described how a primordial immune system in bacteria could be harnessed to edit the genomes of other organisms will turn 10 years old. The discovery that CRISPR could be turned into an easily programmable tool for rewriting DNA launched biomedical research into warp drive.

In the 10 years leading up to 2012, 200 papers mentioned CRISPR. In 2020 alone, there were more than 6,000. The last decade has seen scientists use CRISPR to cure mice of progeria, fix muscular dystrophy in dogs, and eliminate symptoms for people with genetic blood disorders. Currently, there are more than two dozen human trials of the technology underway around the world.

STAT has created a new tracker of milestone CRISPR studies, and found that the explosion in interest created a positive feedback loop, accelerating the movement of new and better gene editing approaches toward the clinic. For CRISPR 1.0 therapies those using the original Cas9 cutting enzyme described in the Doudna paper four-and-a-half years passed, on average, between the first studies in cells and the first public data in non-human primates. Base editing, or CRISPR 2.0, got it down to three years, according to the CRISPR TRACKR.

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This time-shaving trend is evident in other ways, too. Last November, Beam Therapeutics announced it had gotten the green light to test its base editing technology in humans for treating sickle cell disease. If it begins dosing patients this year, that will put Beam just a few years behind the CRISPR 1.0 companies Intellia, Editas Medicine, and Crispr Therapeutics which began clinical studies of therapies for various genetic disorders in 2021, 2020, and 2019, respectively, effectively shortening the development time from an average of eight years to six.

Were now seeing a real acceleration in progress, said Kiran Musunuru, a gene editing researcher at the University of Pennsylvania and the co-founder of Verve Therapeutics. As the challenges are worked out for version 1.0, it just makes it much much easier to substitute in version 2.0 and then 3.0 and then whatever is next.

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The first five years after Doudna and Charpentiers (and Feng Zhangs and George Churchs) seminal papers were published, the field was consumed with fine-tuning how CRISPR-Cas9 worked in different kinds of cells, setting records for how many cuts it could make, and finding medically relevant applications for its targeted gene-breaking abilities.

The next five, driven by a gold rush in finding, engineering, or evolving new CRISPR proteins, saw the gene editing toolbox expand rapidly outward. These newer, shinier, crisper versions of CRISPR pushed forward faster toward the clinic, propelled by all the groundwork that had been laid by its older, clunkier cousin.

The thing thats frankly exhilarating to me, as a gray-haired veteran of editing, is how rich the overall ecosystem has become, said Fyodor Urnov, scientific director of the Innovative Genomics Institute at the University of California, Berkeley, which is headed by Doudna.

Urnov compared the 2000s, when he and others were working on pre-CRISPR versions of genome editing, to medieval times, with a few labs toiling away in their fiefdoms, separated by large tracts of no-mans land. The tools were few, and difficult to come by. Today, Urnov said, dialing up a gene editing experiment is more like clicking open the app store on your smartphone. Not only will you find options for different kinds of editors and modes for delivering them, but each comes with ratings and reviews too.

Ten years ago, the ability to just walk into this enormous smorgasbord of offerings simply didnt exist, he said.

For many of these tools, its still too early to say which ones will take off into components of blockbuster therapies and which ones will burn out upon takeoff. But with the field moving at warp speed, it wont be long before we know.

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Drugs based on next-generation gene editing are moving toward the clinic faster than CRISPR 1.0 - STAT

BRAZIL - 2021/02/18: In this photo illustration an Editas Medicine logo seen displayed on a ... [+] smartphone with the stock market graphic in the background. (Photo Illustration by Rafael Henrique/SOPA Images/LightRocket via Getty Images)

Our theme of Gene Editing stocks continues to underperform, declining by about 16% year-to-date in 2022, compared to the S&P 500 which is down by about 6% over the same period. The theme also declined by about 11% in 2021. With interest rates rising and monetary policy set to get tighter, the markets are souring on high-growth and futuristic stocks. Gene editing stocks have been hit particularly badly as most players dont generate meaningful revenue as yet and remain deeply unprofitable. Some of the companies in the theme have also witnessed clinical setbacks or seen mixed data from their clinical trials over the last year (see updates below).

So whats the outlook like for the theme? The sector clearly remains out of favor with the market and could see more volatility through 2022 if investors continue to move out of riskier assets amid rising interest rates. Liquidity could also be an issue for smaller players such as Editas Medicine which have been burning through cash, meaning that potentially dilutive fundraises are a possibility.

However, with market capitalizations of gene editing stocks remaining depressed following the sell-off over the last year, some companies could be attractive acquisition targets for larger pharma companies looking for expertise and pipelines in the gene-editing space. The long-term outlook for gene editing as a larger theme also appears promising, given the potentially revolutionary drugs under development, that could cure conditions from cancer to rare genetic disorders that currently lack treatments, to more chronic conditions such as diabetes. Considering this, the theme could see upside in the long term and the recent correction could be a buying opportunity.

Within our theme, Editas Medicine has been the worst performer, declining by about 30% year-to-date in 2022. On the other side, Vertex Pharmaceuticals has been the best performer, with its stock up around 10% year-to-date.

Below youll find our previous coverage of the Gene Editing theme where you can track our view over time.

[8/13/2021] Will Modernas Interest Boost Gene Editing Stocks?

Our indicative theme of Gene Editing stocks has returned about 11% year-to-date, compared to the S&P 500 which is up by about 19% over the same period. However, the gains have overwhelmingly come from a single stock, Intellia Therapeutics, which is up by about 3x year-to-date, after the company announced positive results from early-stage clinical trials for its experimental treatment for transthyretin amyloidosis, marking the first time genome editing was carried out inside the human body to treat disease. The five other stocks in our theme remain down year-to-date. For instance, Editas Medicine remains down by about 6.8%, while bluebird bio remains down by about 56%.

That being said, we think the outlook for gene-editing stocks is looking better. Intellias progress bodes well for the broader gene-editing space, as it validates that gene-editing technology works in humans and also that it remains safe. As more of these companies move candidates into clinical stages and provide readouts, we could see movements in stock prices across the theme. Moreover, gene-editing companies could be ripe for buyouts. For instance, Covid-19 vaccine behemoth Modernas management indicated that it was interested in expanding into other areas, including gene editing. Considering that a majority of gene-editing stocks are small to mid-cap companies, they could easily be acquired by larger players such as Moderna.

[7/1/2021] Gene Editing Stocks Are Worth A Look After Intellias Big Breakthrough

Intellia Therapeutics - a gene-editing company co-founded by CRISPR pioneer and Nobel prize winner Jennifer Doudna - indicated that NTLA-2001, its experimental treatment for transthyretin amyloidosis provided very promising results in an early state trial. Although the study was small, including just six patients, the company noted that there were significant reductions in levels of a harmful liver protein that is associated with the disease after a single infusion. Intellia stock has rallied by almost 80% over the last three trading days following the news.

Now, we think that this could be a big deal for the broader gene editing sector, as well. This was the first report from a clinical trial of genome editing carried out inside the human body to treat disease, and the results should broadly validate that gene-editing technology works in humans and also that it remains safe. Our indicative theme of Gene Editing stocks has rallied considerably over the last week, and remains up by roughly 20% year-to-date, compared to the S&P 500 which is up by about 15% over the same period. That said, the gains are primarily driven by Intellia stock, which is up by almost 3x year-to-date, and the five other stocks in our theme have actually underperformed the market, or declined this year. For example, CRISPR Therapeutics is up by just about 6%, while Vertex Pharmaceuticals and Editas Medicine are down by 15% and 19%, respectively. Sangamo Therapeutics is down 23% (chart, 10-k), while bluebird bio is down by 26%. As more of these companies move candidates into clinical stages and provide readouts, we could see gains in stock prices across the theme.

[6/14/2021] Should You Add Gene Editing Stocks To Your Portfolio?

Our indicative theme of Gene Editing stocks is down by about 12% year-to-date, compared to the S&P 500 which is up by over 13% over the same period. The decline comes as investors move money from high-growth and futuristic sectors to more cyclical and value stocks to ride the post-Covid surge in economic activity over the next few quarters. Gene Editing players have been particularly badly hit by this shift, given that they are mostly clinical or pre-clinical stage biotechs with little or no revenues. Now, although most of the companies in our theme are currently losing money, and are presently out of favor with the market, the longer-term upside could be sizable, given that they are working on potentially revolutionary drugs that could cure conditions from cancer to rare genetic disorders that currently lack treatments, to chronic conditions such as diabetes.

Within our theme, Intellia Therapeutics was the strongest performer, rising by about 57% year-to-date, due to favorable views from brokerages and anticipation surrounding the companys NTLA-2001 drug, which is a single-course, potentially curative therapy for transthyretin amyloidosis. A data readout from the phase 1 study on the drug is due later this month. On the other side, Editas Medicine has been the worst performer in our theme, declining by about -47% year to date, partly due to its big rally late last year, multiple analyst downgrades, and some changes at the top management level.

[3/29/2021] Gene Editing Stocks Have Corrected. What Next?

Our indicative theme of Gene Editing stocks is down by about 19% year-to-date, compared to the S&P 500 which is up by about 6% over the same period. With the economic recovery expected to gather pace, on the back of declining Covid-19 cases and higher vaccination rates, bond yields have been trending higher, causing investors to move funds from highly valued growth names to more cyclical and value bets. Gene Editing players have been particularly badly hit by this shift, given that they are mostly clinical or pre-clinical stage biotechs with little or no revenues. That said, we think that this could be a good time to take a look at the sector, considering that these companies are working on potentially revolutionary developments that could cure conditions from cancer to rare genetic disorders.

Within our theme, Intellia Therapeutics was the strongest performer, rising by about 19% year-to-date. Last November, the company began dosing under its phase 1 study is to evaluate its drug NTLA-2001 which is a single-course, potentially curative therapy for transthyretin amyloidosis. A data readout is due sometime in the next several months. On the other side, Editas Medicine has been the worst performer, declining by about 42% year to date, partly due to its big rally late last year, multiple analyst downgrades, and some changes at the top management level. See our earlier updates below for a detailed look at the components of our Gene Editing stocks theme.

[2/10/2021] Gene Editing Stocks To Watch

Our indicative theme of Gene Editing Stocks is up by about 187% since the end of 2018 and by about 5% year-to-date. Gene editing has received more attention this year, as scientists used the technology to cure progeria syndrome in mice, raising hopes for therapy in humans as well. Progeria is a very rare genetic condition that causes premature aging in children, shortening their lifespan to approximately 14 years. Investors also remain interested in the sector, given that it could revolutionize medicine and also due to the fact that absolute valuations arent too high, with most of the companies remaining in the mid-cap space.

Within our theme, Intellia Therapeutics (NASDAQ: NTLA) has been the strongest performer year-to-date, rising by around 35% since early January. The company recently outlined strategic priorities for 2021, which include the continued advancement of a phase 1 study for a single-course therapy for protein misfolding disorder and the planned submission of regulatory applications for the treatment of acute myeloid leukemia and hereditary angioedema this year. On the other side, Vertex Pharmaceuticals, has declined by about 10% year to date, driven partly by weaker than expected Q4 2020 results. See our updates below for a detailed look at the components in our theme.

[1/27/2021] How Are Gene Editing Stocks Faring?

Gene-editing technology is used to insert, edit, or delete a gene from an organisms genome, and shows promise in treating medical conditions ranging from cancer to rare genetic conditions. Our indicative theme on Gene Editing Stocks has returned over 170% since the end of 2018, compared to the broader S&P 500 which is up by about 54% over the same period. The theme has returned about 2.4% year-to-date. Investor interest in gene-editing remains high, given the upside potential of the sector and considering that absolute valuations arent too high, with most of the stocks remaining in the mid-cap space. Intellia Therapeutics (NASDAQ: NTLA) has been the strongest performer in our theme this year so far, rising 18% since early January. The gains come as the company has outlined strategic priorities for 2021, which include the continued advancement of a phase 1 study for a single-course therapy for protein misfolding disorder and the planned submission of a regulatory application for the treatment of acute myeloid leukemia. [1] On the other side, Editas Medicine has declined by about 13% year to date, after the company indicated that it plans to raise additional capital, issuing about 3.5 million shares at $66 per share. See our update below for a detailed look at the components in our theme.

[1/8/2021] Gene Editing Stocks

Gene editing has emerged as a promising biotech theme. The technology is used to insert, edit, or delete a gene from an organisms genome, helping to replace the defective genes responsible for a medical condition with healthy versions. This technology is being used to develop treatments for a range of diseases from cancer to rare genetic conditions, that are otherwise hard to treat, and is also being considered for diagnostic purposes. While there are broadly three gene-editing technologies, clustered regularly interspaced short palindromic repeats or CRISPR, as it is popularly known, has emerged as the method of choice with most companies, considering that it is relatively inexpensive, simpler, and more flexible compared to other tools such as ZFN and TALEN.

While most gene-editing players remain in the clinical stage with a limited financial track record, funding has risen meaningfully and larger pharma companies are also partnering with these companies, considering that the treatments could be lucrative and the broader technologies may be highly scalable. While the upside remains large, investing in these companies is risky. Being a new technology that has never been used in humans before, there are risks of significant side effects or of the therapies not being effective. The economics of producing and selling these drugs also remains uncertain. These stocks are also volatile, seeing big swings as any new research or data on their potential or risk is outlined. Our indicative theme on Gene Editing Stocks - which includes names such as CRISPR Therapeutics, Editas Medicine, and others - has returned about 230% over the past 2 years, compared to the broader S&P 500 which is up by about 52% over the same period. Below is a bit more about these companies.

CRISPR Therapeutics AG is one of the best-known names in the gene-editing space. The company is working with Vertex Pharmaceuticals to co-develop CTX001, an experimental gene therapy that has provided promising results for people with sickle cell disease, and transfusion-dependent beta-thalassemia - disorders that affect the oxygen-carrying cells in human blood. The company is also developing cancer therapy candidates independently. The company was profitable last year, due to collaboration revenues from Vertex.

CRSP

Editas Medicine, another leading CRISPR-focused biotech company, with a flagship program, EDIT-101 is targeting the treatment of hereditary blindness. The company recently finished dosing for its first group of patients in earlier-stage human trials. The company also recently filed a request with the U.S. FDA to commence phase 1/2 study of EDIT-301 in treating sickle cell disease. The company also has multiple other pre-clinical drugs focused on genetic diseases.

Intellia Therapeutics is developing a drug for a rare and fatal disease known as transthyretin amyloidosis in collaboration with Regeneron. The drug is in phase 1 trials currently. The company is also working on ex-vivo Sickle Cell Anemia treatment with Novartis that involves editing cells outside the body before infusing them into the patient. The candidate is entering Phase 1/2 trails. While the company has 8 other candidates, they are still in the research or pre-clinical stages. [2]

Sangamo BioSciences focuses on multiple areas in the genomic medicine space, including gene therapy, cell therapy, in vivo genome editing, and in vivo genome regulation. The company pioneered the zinc finger nuclease gene-editing method. The companys most advanced development is a treatment for Hemophilia A, which is being developed with Pfizer and is in phase 3 trials. The company also has 4 candidates in the phase 1/2 stage and 13 in the Preclinical stage. [3]

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The Sell Off In Gene Editing Stocks Continues. Time To Buy? - Forbes

Today, its clear that our genes not only cause many diseases, but also hold potential cures. But that wasnt always the case. It wasnt until 1949 that scientists first found the molecular culprit of a disease its roots in the genetic code. The disease was the blood disorder known as sickle cell disease, an inherited disorder that causes severe and debilitating pain. Now, nearly 75 years later, researchers are developing gene therapies to cure it.

Sickle cell disease results from a change in a key protein in hemoglobin, which helps transport oxygen in red blood cells. Hemoglobin normally allows red blood cells to be very floppy and pliable, and slip and slide through the blood vessels easily, says pediatrician Erica Esrick. But a mutation in a single gene, the HBB gene, makes hemoglobin stack in long strings inside blood cells, giving them an inflexible, sickle shape. Instead of being squishy, the stiff red blood cells get stuck inside blood vessels, blocking blood flow.

Sickle cell affects millions of people around the world, particularly those whose ancestors come from sub-Saharan Africa, parts of the Middle East and Southeast Asia. In the United States, for instance, approximately 100,000 people live with the disease, most of them Black or Latino. People with sickle cell disease have a shortened life expectancy, living only into their late 40s on average, in large part due to strokes or organ damage from blocked blood vessels. Esrick, of Boston Childrens Hospital and Harvard Medical School, and others are trying to fight the disease through gene therapy.

Gene therapies seek to manipulate the very information of life by replacing, inactivating or fixing missing or broken genes and so curing patients. But the journey to todays handful of approved gene therapies, including for diseases like severe combined immunodeficiency syndrome, or SCID, certain blood cancers and spinal muscular atrophy, has been rocky. Early clinical trials in the 1990s werent effective, and the 2000s brought unintended and sometimes deadly consequences, including a leukemia-like illness.

Despite gene therapys challenges, many researchers believe sickle cell is a good target because the molecular pathways are well understood and straightforward. Whats more, every copy of the gene doesnt need to be mended to have an effect. (Individuals who inherit the mutated gene from only one parent, for example, dont develop sickle cell disease.)

Esrick is co-leading a clinical trial testing a gene therapy that attempts to encourage the body to make more of a healthy type of hemoglobin produced by fetuses and young babies but not adults called fetal hemoglobin. DNA for making a short string of genetic material called a microRNA is delivered by a virus into cells from a patients bone marrow. The virus, called a vector, permanently inserts the DNA into the cells genetic blueprint. The microRNA then interferes with the production of a protein that prevents fetal hemoglobin from being made. Once that protein is blocked, fetal hemoglobin production turns back on. Like turning on a faucet, a steady stream of the healthy hemoglobin can flow into the bloodstream, making up for the faulty form.

Preliminary data released in January 2021 showed that the treatment helped six sickle cell patients make fetal hemoglobin, Esrick and colleagues reported in the New England Journal of Medicine. During the follow-up period, ranging from several months to more than two years, the patients symptoms were reduced or eliminated. The team has expanded the trial to include more patients and further test the treatment.

Scientists are testing other ways to tackle sickle cell via gene therapy, too. A biotechnology company called bluebird bio is testing an approach that delivers a functional copy of the HBB gene to patients. Another team is preparing to begin a trial that will edit that gene directly using CRISPR/Cas9.

Science News staff writer Erin Garcia de Jess spoke with Esrick about the ongoing fetal hemoglobin clinical trial, including the hurdles and the hope. The conversation has been edited for length and clarity.

Garcia de Jess: What tools do we currently have to treat sickle cell?

Esrick: The only curative treatment is a bone marrow transplant. The bone marrow is like the factory for the blood cells. If you can get bone marrow from somebody who doesnt have sickle cell disease, then you can grow your own healthy red blood cells that dont sickle. But that is a major procedure, and its really only standard if you have whats called a matched sibling [a brother or sister without sickle cell whose key white blood cell proteins match yours].

Less than 20 percent of people with sickle cell have a matched sibling available. If a matched sibling is available, then thats a really good potential treatment option, but it is still a risky procedure. It comes along with some up-front risk of mortality and a lot of potential side effects, such as graft-versus-host disease and a higher risk of infection because of immunosuppressive drugs.

Then there are medications to treat sickle cell. The most well-established and long-lasting is called hydroxyurea. It increases fetal hemoglobin. In many people, it increases the fetal hemoglobin by a lot; thats why it works so well. Its been available since the 90s, and has been moving gradually to younger and younger ages.

Now it is a very clear recommendation that essentially every child with sickle cell should be on it. But not everyone has access to specialized hematology care, and its a medication that has to be taken daily. Some people have adverse effects and cant take it. It also doesnt work for everybody.

Garcia de Jess: How many people are in your teams trial and what results have you seen so far?

Esrick: Nine patients have been treated. We anticipate the 10th patient will be treated soon. The preliminary data from the first six patients was published about a year ago. Additional data from subsequent patients has been largely quite similar except for one patient whose fetal hemoglobin response was unfortunately not as robust.

Garcia de Jess: What is the process like for the trial participants?

Esrick: Patients have to get their cells collected [the cells live in the bone marrow and give rise to blood cells], which takes a three-day hospital admission and sometimes has to be repeated a few times. Its through IV, basically. Then the cells get taken off to the lab.

When we get word from the lab, OK, we have a good product [meaning the virus got the DNA into enough cells], then the patient comes back and is admitted to the hospital for a month or so. Its a long and arduous hospital admission because they need to receive chemotherapy.

The reason they need chemotherapy is because the bone marrow cells that havent been collected need to get nearly wiped out in order to give the advantage to the cells that are being given back [also through IV] to set up shop and produce.

Chemotherapy comes with a lot of the side effects and risks associated with gene therapy, including acute short-term risks like hearing loss and nausea. And it also comes with some of the long-term risks, including infertility and a risk of blood cancers.

Garcia de Jess: Why choose gene therapy over a bone marrow transplant if both require chemotherapy?

Esrick: With gene therapy, theres no issue with immunosuppression, because its your own cells. People who get a transplant from another person have to be on immunosuppressive medications for a period of months after the transplant. Theres a risk of graft rejection because of the mismatch between the donor and the recipient.

The other risk in a bone marrow transplant from another person is graft-versus-host disease, where the graft and donated cells reject the recipient. That can cause severe disease. With gene therapy, thats not a risk at all.

Garcia de Jess: Last year, a clinical trial run by a company called bluebird bio announced that a trial participant developed leukemia. Cancer is obviously a huge concern and has thwarted previous gene therapy trials. What do we know so far about that?

Esrick: This was, of course, of major concern to the field. It was actually the second case of leukemia in that trial. The first one was published a couple of years ago as a case report.

If theres ever a case of leukemia or any preleukemia in a gene therapy trial, we always ask: Was it caused because the vector stuck a gene into a spot that was dangerous?

It does not look like thats the case. In the first patient in the bluebird bio trial who developed leukemia, the leukemia cells didnt even have the transferred gene in them. So, the thought was that was probably just an example of chemotherapy causing leukemia, which we know can happen in a small percent of people who receive chemotherapy.

But the second case, in February 2021, really raised a red flag. Why is that happening two times in a trial of only 40-something patients? Its still not exactly clear. There are some studies that suggest that people with sickle cell disease may have an increased risk of leukemia. But the [U.S. Food and Drug Administration] placed the bluebird bio trial on hold while some investigations were done. When it became pretty clear that it wasnt directly related to the vector, the trial was allowed to reopen.

Our trial, which has many similarities to the bluebird bio trial, was not put on hold by the FDA but was put on hold by our funder, the National Heart, Lung and Blood Institute while they looked at the data. That hold was recently lifted.

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Garcia de Jess: Have there been any cases of leukemia in your teams trial?

Esrick: Fortunately, no.

But you know when anything like that happens in the field, its a big deal. I called all of the patients who we had treated in our trial to let them know. [The bluebird bio cases] happened in patients who had been treated three and five years prior. The longest-treated patient in our trial was almost three and a half years ago, and the most recently treated was about eight or nine months ago. I hope we see no concerning signs for any new development like that, but its too early to say.

Garcia de Jess: What are some of the biggest challenges that sickle cell has had to overcome?

Esrick: For the longest time, there were no new therapies at all. These technologies took a long time because they are based on basic science discoveries that were being worked on. But also, the patient population with sickle cell is a population that has historically been underserved and without a lot of power.

In the United States, its primarily Black and Latino patients, and across the board those populations have suffered from health inequality. I think that if there were a disease that caused this degree of morbidity and mortality and pain in other parts of the population, it may have been speedier.

Garcia de Jess: What gives you hope? What do you find exciting?

Esrick: I find myself bending over backwards to make sure that Im not coming across as, We have a cure! But that said, it is really exciting that this is a treatment that is theoretically possible for everyone without needing to find a [bone marrow] match. Thats a huge difference from classic bone marrow transplants.

The speed at which new [gene therapy] treatments are being developed is amazing. I think the horizon is very bright in terms of one or maybe many of these therapies being really effective and safe. Ive talked to so many patients and families who have reached out interested in our trial or other trials. Theres such a huge unmet need. The fact that there are a lot of these new treatments that are being developed is an encouragement to these families.

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Gene therapies for sickle cell disease come with hope and challenges - Science News Magazine

Six researchers from Washington University in St. Louis have been named senior members of the National Academy of Inventors (NAI).

Richard Axelbaum, PhD, at the McKelvey School of Engineering, along with five researchers at the School of Medicine David T. Curiel, MD, PhD; James W. Janetka, PhD; Gregory M. Lanza, MD, PhD; Robi D. Mitra, PhD; and Jennifer N. Silva, MD are being recognized for their success in patents, licensing and commercialization, and for producing technologies that have the potential to have a significant impact on the welfare of society.

They are among 83 new senior members who will be honored in June at the NAI annual meeting in Phoenix.

Axelbaum, a professor of energy, environmental and chemical engineering and the Stifel & Quinette Jens Professor of Environmental Engineering Science, researches combustion in its many forms and in different environments including in microgravity on the International Space Station. He uses his understanding of fossil fuel combustion and its resulting pollutants to address concerns over carbon dioxide emissions, notably by developing novel approaches to carbon capture and storage. Axelbaum holds 10 patents and founded the startup company AP Material Inc., which commercialized a flame-synthesis technology to manufacture high-purity nanopowders.

Curiel, a professor of radiation oncology and director of the Biologic Therapeutics Center at the School of Medicine, has harnessed gene therapy and viral vectors to develop therapeutics and vaccines for a number of diseases, including cancer, genetic disorders and COVID-19. More recently he has pioneered gene editing strategies toward gene therapy cures. He has co-founded a number of biotechnology startups, including Altimmune, DNAtrix, Unleash Immuno Oncoloytics and Precision Virologics. Most recently, he co-developed a nasal vaccine against COVID-19 that is in phase 3 human clinical trials in India.

Janetka, a professor of biochemistry and molecular biophysics, is a medicinal chemist who is developing small-molecule therapeutics to treat cancer and infectious diseases. He has expertise in rational, structure-based drug design. Janetkas team is working on novel drugs for treatment of a broad scope of infections caused by bacteria, parasitic worms, toxoplasma and viruses. His team has discovered broad-spectrum new anticancer and antiviral drugs for the treatment of COVID-19. He is a co-founder of two pharmaceutical startups, Fimbrion Therapeutics and ProteXase Therapeutics.

Lanza, a cardiologist and professor of medicine, of biomedical engineering, and of biology and biomedical sciences, has developed nanotechnologies with wide applications in medicine, from cardiovascular imaging to cancer therapy. He was the co-founder, chief scientific officer and a board member of Kereos Inc., a biotechnology startup focused on developing molecular imaging agents and therapeutics for cardiovascular disease and cancer. He co-founded and is chief scientific officer of Capella Imaging Inc., a startup focused on biomedical imaging, and is in partnership with NorthStar Medical Radioisotopes, with a particular focus on detecting blood clots in the heart and in operating ventricular assist devices.

Mitra, a professor of genetics and the Alvin Goldfarb Distinguished Professor of Computational Biology, develops new technologies for analyzing the genome. He and his lab have pioneered new methods for efficient DNA sequencing, analyzing the binding of transcription factors, studying single-molecule proteomics, analyzing single-cell genomics and capturing specific regions of the genome. Mitra played key roles in the founding of a number of genomic resources for the School of Medicine, including the universitys Genomic Technology Access Center, the Genomics and Pathology Services Lab, and the Genome Engineering and iPSC Core facility.

Silva, a pediatric cardiologist and professor of pediatrics, is an electrophysiologist who treats children with disorders of the heart, including those that cause life-threatening arrhythmias. She has developed a 3D imaging system using virtual-reality technology to help cardiologists better visualize the electrical circuits that are misfiring. She co-founded the startup SentiAR to develop a headset that can display a hologram of a patients heart and show a real-time 3D map of what is happening as the patient undergoes a catheter ablation procedure, in which the tissue causing the arrhythmia is burned to stop the erratic signals.

Link:
Six innovators named National Academy of Inventors senior members - The Source - Washington University in St. Louis - Washington University in St....

PARIS, FRANCE (February 8, 2022) Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency.

"These results confirm the stability and good tolerance of the lentiviral vector as a tool for gene transfer into blood stem cells, said Anne Galy, Ph.D., Inserm Research Director at Genethon. Our teams have carried this project from translational research to a clinical trial by working with the best clinical and research teams internationally. We are delighted with the results of this trial which now show the long-term efficacy and safety of this approach for this rare and severe immune deficiency.

Frederic Revah, Ph.D., CEO of Genethon, added, I would like to congratulate Anne Galy and her team who have been working on this project for more than 15 years in the service of patients and their families. This trial for Wiskott-Aldrich syndrome was the first international trial launched by our laboratory, and today 12 clinical trials are being conducted worldwide for products stemming from our R&D. From basic research to clinical development, Genethon has developed a unique expertise in the field of gene therapy for different families of rare diseases.

The long-term results of the WAS clinical trial, sponsored by Genethon and conducted by colleagues in France and England, were published in Nature Medicine, in a paper titled Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for WiskottAldrich syndrome. The vector used in the study was designed, developed and manufactured by Genethon.

WAS, a rare and severe complex immune deficiency, is caused by a mutation in the WAS gene in hematopoietic progenitor cells, which are blood forming cells. The inherited disease affects only boys and results in hemorrhages, repeated severe infections, severe eczema and, in some patients, autoimmune reactions and development of cancers. The only treatment currently available is bone marrow transplantation, which requires a compatible donor and can cause serious complications. Symptoms of the disease emerge at 6 months old and life expectancy for severe forms is 3.5 years without treatment.

Genethons gene therapy involves extracting from patients the blood stem cells carrying the genetic abnormality, correcting them in the laboratory with a healthy WAS gene and transplanting the cells back into the patients. Initial results from the clinical trial, published in the Journal of the American Medical Association in 2015 showed safety and efficacy along with stabilized engraftment of the blood cells 9 months to 42 months after the treatment.

Results from the longer term follow-up of eight patients for a median of 7.6 years confirm the stability of the transplanted genetically modified cells and their safety and efficacy. The gene therapy corrected major disease symptoms, improved or eliminated bleeding and signs of autoimmunity, and restored T-cell (or immune system) function. In addition, a 30-year-old patient was treated in the trial, demonstrating efficacy in adult patients whose thymus gland, which makes T-cells, was thought to be low- to non-functioning after many years of illness. Platelet levels remain low but gene therapy alleviates the need for platelet transfusions and prevents the occurrence of spontaneous hemorrhages.

The WAS gene therapy clinical trial was sponsored by Genethon and conducted in collaboration with Inserm (the French National Institute of Health and Medical Research) and NeckerEnfants Malades Hospital in France; and the University College of London, Great Ormond Street Hospital and Royal Free London Hospital in England.

About Genethon

A pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a unique non-profit organization created by a patient association, the AFM-Telethon. A first gene therapy drug, to which Genethon contributed, has obtained marketing for spinal muscular atrophy. With 200+ scientists and professionals, Genethon is pursuing its mission to bring life-changing therapies to patients suffering from rare genetic diseases. 12 products resulting from Genethons research are in clinical trials for eye, liver, blood, immune system and muscle diseases. A further 7 products are in the preparation phase for clinical trials over the next five years. Find out more: genethon.com

Contacts:

Dan Eramian

Opus Biotech Communicationshttp://opusbiotech.com/425-306-8716

danieleramian@comcast.net

Charles Craig

Opus Biotech Communications

http://opusbiotech.com/404-245-0591

charles.s.craig@gmail.com

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Genethon's Lentiviral vector-based gene therapy demonstrates long-term safety and efficacy for Wiskott-Aldrich Syndrome - EurekAlert

NEW HAVEN, Conn. and NEW YORK - February 14, 2022 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) and Pfizer Inc. (NYSE: PFE), today announced positive top-line results from an Asia-Pacific, Phase 3 clinical trial of rimegepant in 1,431 adults for the acute treatment of migraine. Led by BioShin Limited, a subsidiary of Biohaven in China and South Korea, the randomized, regional, multi-center study met the co-primary endpoints evaluating the efficacy and safety of the orally dissolving tablet (ODT) formulation of rimegepant, an oral calcitonin gene-related peptide (CGRP) receptor antagonist.

This is the fourth positive Phase 3 study of rimegepant for the acute treatment of migraine and the first to be conducted in Asia Pacific. The study met its co-primary endpoints of freedom from pain (p<0.0001) and freedom from most bothersome migraineassociated symptom (MBS) including nausea, phonophobia or photophobia (p<0.0001) at 2-hours following a single oral dose of rimegepant. In the study, a single oral dose of rimegepant 75 mg provided significant relief of migraine symptoms and return to normal function at 2 hours and delivered sustained efficacy that lasted up to 48 hours for many patients. Rimegepant showed a favorable safety and tolerability profile among study participants that was consistent with prior clinical trial results in the United States. Detailed data from the study will be presented at future medical meetings to help inform ongoing and future research.

Under the terms of the collaboration agreement between Biohaven and Pfizer, Pfizer has commercialization rights to rimegepant in markets outside of the U.S. Biohaven continues to lead research and development globally and retains the U.S. market. Rimegepant is commercialized as Nurtec ODT in the U.S. and is the only oral CGRP receptor antagonist approved for both the acute and preventive treatment of migraine in adults. An application for the approval of rimegepant is currently under review by the European Medicines Agency with a decision expected in the first half of 2022. Rimegepant is approved for the acute treatment of migraine in Kuwait and the United Arab Emirates, and for the acute and preventive treatment of migraine in Israel.

Vlad Coric, M.D., Chief Executive Officer and Chairman of the Board of Biohaven commented, "These top-line trial results clearly show the consistent clinical profile of rimegepant to relieve migraine symptoms and return patients to normal function. Through our partnership with Pfizer, we are committed to rapidly expanding the availability of rimegepant to patients around the world, particularly in Asia Pacific where migraine is a common disease and a leading cause of disability."

"It is very exciting to see the completion and positive results of the first Phase 3 study of rimegepant in Asia Pacific, said Nick Lagunowich, Global President, Pfizer Internal Medicine. "With millions of patients in the region impacted by this debilitating neurological disease, these results provide hope for a potentially new effective acute treatment for patients in need. We are moving as quickly as possible in our effort to get this potential treatment into the hands of patients, and we look forward to working with regulatory agencies around the world to do so.

Professor Shengyuan Yu, Principal Investigator of the study and Director of the Department of Neurology, Chinese PLA General Hospital, said, We need new, effective and safe treatment options to help improve the lives of our migraine patients in Asia and are encouraged by the positive results of this study.

Donnie McGrath, M.D., Executive Chairman of Biohavens wholly-owned subsidiary in China, BioShin, added, The results from this study demonstrate the effectiveness of rimegepant and highlight the potential impact for patients in Asia Pacific, if approved. Im so proud of the BioShin R&D team who executed this study.

About RimegepantRimegepant targets a key component of migraine by reversibly blocking CGRP receptors, thereby inhibiting the biologic cascade that results in a migraine attack. Rimegepant was approved by the U.S. Food and Drug Administration (FDA) under the trade name Nurtec ODT for the acute treatment of migraine in February 2020 and for the preventive treatment of episodic migraine in May 2021. A single dose of 75 mg Nurtec ODT provides fast pain relief, significant pain reduction and return to normal function, and has a lasting effect of up to 48 hours in many patients. Nurtec ODT is taken orally as needed, up to 18 doses/month to stop migraine attacks or taken every other day to help prevent migraine attacks and reduce the number of monthly migraine days. Nurtec ODT does not have addiction potential and is not associated with medication overuse headache or rebound headache.

About NURTEC ODTNURTEC ODT (rimegepant) is the first and only calcitonin gene-related peptide (CGRP) receptor antagonist available in a quick-dissolve ODT formulation that is approved by the U.S. Food and Drug Administration (FDA) for the acute treatment of migraine with or without aura and the preventive treatment of episodic migraine in adults. The activity of the neuropeptide CGRP is thought to play a causal role in migraine pathophysiology. NURTEC ODT is a CGRP receptor antagonist that works by reversibly blocking CGRP receptors, thereby inhibiting the biologic activity of the CGRP neuropeptide. For more information about NURTEC ODT, visit http://www.nurtec.com.

IndicationNURTEC ODT orally disintegrating tablets is a prescription medicine that is used to treat migraine in adults. It is for the acute treatment of migraine attacks with or without aura and the preventive treatment of episodic migraine. It is not known if NURTEC ODT is safe and effective in children.

Important Safety InformationDo not take NURTEC ODT if you are allergic to NURTEC ODT (rimegepant) or any of its ingredients. Before you take NURTEC ODT, tell your healthcare provider (HCP) about all your medical conditions, including if you: have liver problems, have kidney problems, are pregnant or plan to become pregnant, breastfeeding or plan to breastfeed.

Tell your HCP about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

NURTEC ODT may cause serious side effects including allergic reactions, trouble breathing and rash. This can happen days after you take NURTEC ODT. Call your HCP or get emergency help right away if you have swelling of the face, mouth, tongue, or throat or trouble breathing. This occurred in less than 1% of patients treated with NURTEC ODT.

The most common side effects of NURTEC ODT were nausea (2.7%) and stomach pain/indigestion (2.4%). These are not the only possible side effects of NURTEC ODT. Tell your HCP if you have any side effects.

You are encouraged to report side effects of prescription drugs to the FDA. Visit http://www.fda.gov/medwatch or call 1800FDA1088 or report side effects to Biohaven at 18334NURTEC.

See full Prescribing Information and Patient Information.

About MigraineMore than one billion people worldwide suffer from migraine and the World Health Organization classifies migraine as one of the 10 most disabling medical illnesses. Migraine is characterized by debilitating attacks lasting four to 72 hours with multiple symptoms, including pulsating headaches of moderate to severe pain intensity that can be associated with nausea or vomiting, and/or sensitivity to sound (phonophobia) and sensitivity to light (photophobia). There is a significant unmet need for new treatments as more than 90 percent of people with migraine are unable to work or function normally during an attack.

CGRP Receptor AntagonismSmall molecule CGRP receptor antagonists represent a novel class of drugs for the treatment of migraine. CGRP receptor antagonists work by reversibly blocking CGRP receptors, thereby inhibiting the biologic activity of the CGRP neuropeptide. For acute treatment, this unique mode of action potentially offers an alternative to other agents, particularly for patients who have contraindications to the use of triptans or who have a poor response to triptans or are intolerant to them. CGRP signal-blocking therapies have not been associated with medication overuse headache (MOH) or rebound headaches which limits the clinical utility of other acute treatments due to increases in migraine attacks that result from frequent use.

About Pfizer: Breakthroughs That Change Patients LivesAt Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.Pfizer.com. In addition, to learn more, please visit us on http://www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Pfizer Disclosure NoticeThe information contained in this release is as of February 14, 2022. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about rimegepant, and a collaboration agreement between Pfizer and Biohaven for commercialization of rimegepant outside the U.S., including their potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from the clinical studies; whether and when any applications may be filed for rimegepant in any jurisdictions; whether and when regulatory authorities may approve any potential applications that may be pending or filed for rimegepant in any jurisdictions (including the application for rimegepant pending with the European Medicines Agency), which will depend on myriad factors, including making a determination as to whether the products benefits outweigh its known risks and determination of the products efficacy and, if approved, whether rimegepant will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of rimegepant; whether the collaboration between Pfizer and Biohaven will be successful; uncertainties regarding the impact of COVID-19 on Pfizers business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizers Annual Report on Form 10-K for the fiscal year ended December 31, 2020 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information and Factors That May Affect Future Results, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

About BiohavenBiohaven is a commercial-stage biopharmaceutical company with a portfolio of innovative, best-in-class therapies to improve the lives of patients with debilitating neurological and neuropsychiatric diseases, including rare disorders. Biohaven's Neuroinnovation portfolio includes FDA-approved NURTEC ODT (rimegepant) for the acute and preventive treatment of migraine and a broad pipeline of late-stage product candidates across three distinct mechanistic platforms: CGRP receptor antagonism for the acute and preventive treatment of migraine; glutamate modulation for obsessive-compulsive disorder, Alzheimer's disease, and spinocerebellar ataxia; and MPO inhibition for amyotrophic lateral sclerosis. More information about Biohaven is available at http://www.biohavenpharma.com.

Forward-Looking StatementsThis news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements involve substantial risks and uncertainties, including statements regarding the future development, timing and potential marketing approval and commercialization of NURTEC ODT (rimegepant). Various important factors could cause actual results or events to differ materially from those that may be expressed or implied by our forward-looking statements. Additional important factors to be considered in connection with forward-looking statements are described in the "Risk Factors" section of Biohaven's Annual Report on Form 10-K for the year ended December 31, 2020, filed with the Securities and Exchange Commission on March 1, 2021, and Biohaven's subsequent filings with the Securities and Exchange Commission. The forward-looking statements are made as of this date and Biohaven does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

NURTEC and NURTEC ODT are registered trademarks of Biohaven Pharmaceutical Ireland DAC. Neuroinnovation is a trademark of Biohaven Pharmaceutical Holding Company Ltd.

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Biohaven and Pfizer Announce Positive Top-Line Results of Pivotal Trial of Rimegepant for the Acute Treatment of Migraine in China and South Korea -...