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Category Archives: Gene Medicine

Patients with Severe Forms of Coronavirus Disease Could Offer Clues to Treatment – Howard Hughes Medical Institute

Posted: March 26, 2020 at 6:25 am

A new international project aims to enroll 500 COVID-19 patients to search for genetic mutations that make some people more vulnerable to severe infection.

HHMI scientists are joining many of their colleagues worldwide in working to combat the new coronavirus.Theyre developing diagnostic testing, understanding the viruss basic biology, modeling the epidemiology, and developing potential therapies or vaccines. Over the next several weeks, we will be sharing stories of some of this work.

Hundreds of clinicians worldwide are banding together in an effort to study some types ofseverecases of the new coronavirus disease.

The project, led by Howard Hughes Medical Institute (HHMI) Investigator Jean-Laurent Casanova at The Rockefeller University, seeks to identify genetic errors that make some younger patients especially vulnerable to the virus that causes COVID-19, the infectious respiratory illness also known as coronavirus disease 2019.

Casanova aims to enroll 500 patients internationally who meet three broad criteria: theyre less than 50 years old, have been diagnosed with COVID-19 and admitted to an intensive care unit, and have no serious underlying illnesses, such as diabetes, heart disease, or lung disease.

By studying these patients' DNA, scientists may pinpoint genetic mutations that make some people more susceptible to infection. Such information could one day help doctors identify people who are most at risk of developing severe coronavirus disease, says Casanova, a pediatrician at Rockefeller. It could also offer clues for scientists searching for new therapeutics. For example, if patients cells arent making enough of a particular molecule, doctors may be able to offer a supplement as treatment.

Were going to try to find the genetic basis of severe coronavirus infection in young people.

Jean-Laurent Casanova, HHMI Investigator at The Rockefeller University

That day may still be years away. This is not a short-term effort, Casanova says. Some scientists have hypothesized that COVID-19 might be a seasonal illness, with infections ebbing in the spring and summer, and then returning in the fall. But Casanovas team is optimistic. They have already begun enrolling patients and have started sequencing their exomes spelling out all of the DNA letters in every gene in a persons genome. Were going to try to find the genetic basis of severe coronavirus infection in young people.

Late last year, when the first coronavirus infections began cropping up in China, Casanova started reaching out to his colleagues there. Though the most severe cases seemed to concentrate among older adults and those with other conditions, Casanova was interested in the outliers kids and young adults hit hard by the illness who didnt have any of the usual risk factors, such as age or underlying illness.

His team kicked off a new project to study these mysterious cases, and in January just weeks after the Wuhan outbreak began enrolling patients. Clinicians mailed patient blood and DNA to his lab, and researchers there and elsewhere began processing samples the first steps needed for scientists to peer into patients genomes. Now, the project is global, and Casanova is collaborating with scientists and healthcare workers from Europe to Africa, Asia, and Oceania.

We will recruit children and adults <50 yo without risk factor admitted to ICU for idiopathic #COVID19. We will test the hypothesis that they carry inborn errors of immunity to this virus. Please refer patients to @casanova_lab and please RT. pic.twitter.com/DXPoFKieEy

Hunting for the genetic underpinnings of severe infectious diseases is nothing new for Casanovas team. What were doing with coronavirus is what my lab has been doing for 25 years with other infections, he says.

They look for weak spots in peoples immune systems small genetic changes that make people more vulnerable to disease. His group has previously searched the genomes of patients infected with viruses, bacteria, fungi, and even parasites. The infection closest to COVID-19 his team has studied is severe influenza pneumonitis, for which theyve discovered three genetic links. Theyve also identified specific genetic errors that can predispose patients with herpes to viral encephalitis. And theyve found that children with mutations in an immunity gene called IFN-gamma are vulnerable to the bacteria that cause tuberculosis. These children make low levels of the IFN-gamma protein, which is critical for fighting off bacterial infections.

Casanovas team has put these findings to use clinically. For example, the researchers have shown that tuberculosis patients with these genetic errors can benefit from treatment with IFN-gamma. Hes hoping to identify problematic genes in patients with severe coronavirus infection that can bring similar clinical gains. These genes could tell scientists which cellular defenses are crucial for warding off COVID-19 and pave the way for understanding whether such defenses are derailed in older adults or patients with an underlying medical condition.

In the US and around the world, severe coronavirus disease seems to hit older patients hardest, though scientists have reported some country-to-country variation. As of March 24, more than 44,000 confirmed and presumptive positive cases have been reported in the US. Fatality has been highest in people over 85 years old, according to a recent report from the Centers for Disease Control and Prevention (CDC). Though young people may be more susceptible than scientists once suspected,the older you are, the higher the likelihood you have a severe form of the disease, Casanova says.

Last week, Rockefeller closed all labs except those working on the coronavirus, and Casanova whittled his team to a skeleton crew of about eight people down from 35 who rotate so there is only one person per room at a time. He and his lab members are following CDC recommendations, and taking protective measures to keep themselves and others safe, including social distancing, washing hands, and disinfecting surfaces. Theyve also taken to Twitter to get the word out about their work. A tweet posted from Casanovas lab last week about recruiting new patients to their study has since been retweeted more than 400 times.

Soon, theyll be testing their genetic theory on a pandemic thats occurring in real time. Im grateful weve been able to start this new project so quickly, he says. God willing, it will be of clinical usein two or three years.

Follow the Casanova lab on Twitter (@casanova_lab) to learn the latest about their work. Doctors interested in enrolling patients in the study can contact Jean-Laurent Casanova at jean-laurent.casanova@rockefeller.edu.

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Clean your phone ‘at least once a day,’ medical experts say. Here’s howwithout damaging the screen – CNBC

Posted: at 6:25 am

The coronavirus pandemic has led to a spike in sales of products, includinghand sanitizer, toilet paper and pasta.

More than likely, however, there is no need to buy too much of anything, Dr. Amy Edwards, a pediatric infectious disease specialist at University Hospitals who works with the UH Roe Green Center for Travel Medicine & Global Health,tells Grow. "My advice would be to be vigilant, but calm, and not to panic."

To protect yourself from coronavirus, health professionals say the most important thing to do is wash your hands regularly. Don't forget to clean your phone regularly, too. "I clean my phone at least once a day," says Edwards. She advises others to do the same and many medical experts agree.

"It's often said that your phone is like a third hand because you're constantly touching it," says cleaning influencer Melissa Maker.

People take their phones out to eat, on the train, and to the bathroom. As a result, cellphones carry more than 17,000 bacterial gene copies each, according to a 2017 study. The report concluded that this "may play a role in the spread of infectious agents."

More from Grow:What to buy when your grocery store is out of pasta, beans, juiceHow a hairdresser plans to stretch her $4,000 savings while out of work3 smart ways to improve your finances while you're at home

Cleaning phones daily, at least, is smart, says Edwards."Certainly, if you are letting a lot of people use your phone, you would want to clean it to help prevent spread."

It's often said that your phone is like a third hand because you're constantly touching it.

Melissa Maker

Cleaning Influencer

If you want to clean your phone effectively, Maker says not to use a Lysolwipe or disinfectant wipe, as it may strip the coating of your phone over time. "The chemicals that are used in those disinfectant wipes are not meant to be used on electronics," she says.

Until recently, Apple advised against the use oftraditional cleaning products or compressed air.But earlier this month, Apple updated its instructions to say you can clean your phone with disinfectant wipes, as long as you wipe gently and avoid getting any liquid in charging ports.

GuidelinesforAndroidhandsets still advise steering clear of disinfectant wipes.

One alternative: Cleaning wipes that are specifically made for electronic devices. A 210-pack of individually wrapped lens- and screen-cleaning wipes is $16.99 on Amazon right now.

Maker suggests using a microfiber cloth. "Microfiber has the ability to pick up bacteria," Makers says. "Then you can launder the microfiber cloth."A six-pack of microfiber cleaning clothes is $9.99 on Amazon right now.

A damp microfiber cloth can remove microorganisms including viruses and bacteria and is more effective than a cotton rag, microbiologist Kristen Gibson told the The Wall Street Journal. It won't damage your phone the way a Lysol wipe might, either.

You can also pair onewith a homemade cleaner that is equal parts water and rubbing alcohol. Dip the cloth in the mixture, make sure it's not excessively wet, and then wipe down all parts ofyour phone. This will serve as an effective disinfectant.

And, of course, make sure you frequently wash your hands.

The article Clean Your Phone At Least Once a Day, Says Infectious Disease Specialist originally appeared on Grow by Acorns + CNBC.

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Castle Creek Biosciences Announces $75 Million Investment to Advance Development of Multiple Gene Therapy Candidates for Rare Diseases – Yahoo Finance

Posted: at 6:25 am

- Leveraging proprietary technology and manufacturing platform to develop transformativegene therapies for multiple rare diseases with high unmet needs -

- Pipeline led by Phase 3 gene therapy candidate for treatment of recessive dystrophic epidermolysis bullosa (RDEB), with a BLA filing targeted for 2021 -

- Backed by world-class group of biotech operators and investors -

EXTON, Pa., March 25, 2020 (GLOBE NEWSWIRE) -- Castle Creek Biosciences, Inc., a privately held, late-stage gene therapy company, announced that it has received a new investment of $75 million to support the advancement of its clinical development pipeline. Castle Creek Biosciences is a portfolio company of Paragon Biosciences, which led the $55 million equity investment from Fidelity Management & Research Company and Valor Equity Partners, along with a $20 million venture loan from Horizon Technology Finance Corporation (HRZN).

Castle Creek Biosciences is leveraging its proprietary technology platform and commercial-scale manufacturing infrastructure to develop personalized gene therapies for rare diseases with high unmet needs. The company plans to use the funding to advance and expand its gene therapy pipeline, led by the Phase 3 clinical development of FCX-007 (NCT04213261), its gene therapy candidate for the treatment of RDEB. It will also use the funding to expand its current good manufacturing practices (cGMP) infrastructure located in the greater Philadelphia region.

Clinical results from the ongoing Phase 1/2 clinical trial for FCX-007 continue to show positive trends in safety and wound healing in RDEB patients. Current data from this clinical trial were presented at the inaugural World Congress on Epidermolysis Bullosa held in London during January of 2020. FCX-007 was administered to 10 non-healing chronic wounds of which eight achieved complete wound closure 12 weeks post-administration (80%) vs. no wound closure in intra-patient, matched non-treated wounds (0%). FCX-007 continues to be well tolerated up to 52 weeks post administration.

We are proud to have the strategic support of world-class investors whose impact enables our efforts to transform the lives of patients and the future of medicine, said John Maslowski, Chief Executive Officer of Castle Creek Biosciences. We are steadfast in our commitment to the epidermolysis bullosa community and will continue to keep patients, caregivers and clinicians informed on the progress of our current programs, including FCX-007 and diacerein topical ointment, while we expand the scope of our gene therapy platform.

Castle Creek Biosciences is led by a strong executive leadership team with a proven record of developing innovative and potentially life-changing treatments for conditions with the greatest medical need, said Jeffery Aronin, Chairman and Chief Executive Officer of Paragon Biosciences. As investors, we are excited by the progress that the team has made and are committed to growing the Castle Creek Biosciences platform to address multiple rare genetic diseases.

About Castle Creek Biosciences, Inc. Castle Creek Biosciences is a privately held company that develops and commercializes gene therapies for patients with rare and serious genetic diseases. The companys lead gene therapy candidate, FCX-007, is being evaluated for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), the most severe and debilitating form of epidermolysis bullosa (EB). The company is also advancing clinical research evaluating a diacerein topical ointment, CCP-020, for the treatment of epidermolysis bullosa simplex (EBS) and other forms of EB. In addition, Castle Creek Biosciences is developing FCX-013, a gene therapy for the treatment of moderate to severe localized scleroderma. Castle Creek Biosciences is a portfolio company of Paragon Biosciences. For more information, visit castlecreekbio.com or follow Castle Creek on Twitter @CastleCreekBio.

About Paragon BiosciencesParagon is a life science innovator that invests in, builds, and advises bioscience companies. Our mission is to serve patients living with severe medical conditions which do not yet have adequate treatments. Paragons portfolio of independently-run bioscience companies focus on biopharmaceuticals, AI-enabled life science products, and advanced treatments such as cell and gene therapies. We help people live longer, healthier lives. For more information, please visit: ParagonBioSci.com.

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Bridging the gap study sequences Asian genomes to diversify genetic databases – University of Virginia The Cavalier Daily

Posted: at 6:25 am

Though the number of human genomes sequenced continues to rise rapidly since the completion of the Human Genome Project a scientific endeavor spanning multiple decades and countries aimed at detailing human DNA in 2003, less than 10 percent of those genomes to date correspond to individuals of Asian descent. The GenomeAsia 100K Project, a non-profit consortium, seeks to change this lack of knowledge surrounding a major portion of the worlds ethnicities. The conglomeration of researchers and private sector executives from around the world from Seoul, South Korea to the University plans to add 100,000 novel genomes from individuals of Asian ethnicity to new open-access databases.

Academic institutions and private sector companies came together in 2016 to launch the GenomeAsia 100K Project. While the research organization MedGenome and Nanyang Technological University in Singapore originally founded the non-profit consortium, representatives from other businesses and schools including Genentech, Macrogen and the University of California, San Francisco have joined the association.

Since genome sequencing can reveal the unique characteristics of each persons genetic material, it can help determine a persons ancestry and the propensity for certain medical conditions. According to GenomeAsia 100K, Asians constitute nearly half of the worlds population, and the distinct ethnicities and communities offer a relatively untapped repository of genetic diversity. The project hopes to provide new insights into inherited diseases as well as those caused by a combination of genetic and environmental factors.

Aakrosh Ratan, assistant professor of public health sciences and researcher for GenomeAsia 100K, explained that in particular, the information the initiative collects may help develop medical treatments based on peoples specific genetic makeup, instead of relying on traditional general treatments that may not target the unique root cause of each patients form of a disease.

The goal of precision medicine is to tailor treatment towards a persons genetic background, and that dream cannot be realized until you have the proper reference databases, Ratan said.

Mutations in humans DNA sequences lead to different copies of the same gene within a person and amongst ethnicities. These different versions of a gene can act as markers of diseases that are inherited or influenced by genetic makeup. For example, the disorder sickle cell anemia is caused by the change of a single point in the DNA sequence. When someone is born with copies of this particular gene from both parents contain the mutation, he or she will suffer from often debilitating pain resulting from red blood cells that cannot effectively transport oxygen.

Ratan explained that genome sequencing can highlight mutations in a persons DNA that may cause illnesses such as sickle cell anemia.

One of the ways we identify the mutations that drive a rare disease is by identifying the mutations and then prioritizing those mutations based on their prevalence in healthy populations, Ratan said. With the medical datasets we have compiled, we can actually improve such analyses for patients of Asian descent.

As of December 2019, the GenomeAsia 100K Project has completed the analysis of 1,739 genomes from 219 populations and 64 countries worldwide. Preliminary findings appeared that same month in the scientific journal Nature. The paper concluded that the sample provided a reasonable framework for sequencing practices and studying the history and health of Asian populations. Ratan and his lab at the University supervised the identification and contributed to the analysis of these genetic variants.

Once the 100,000 genomes have been collected and sequenced, the data will be publicly available as a controlled dataset. As a result, experts investigating topics from heart disease to human evolution can easily access the genome sequences.

One of the real gaps in human genetics studies of disease has been the underrepresentation of non-Europeans, Charles Farber, associate professor of public health sciences, said in an email to The Cavalier Daily. The work of the GenomeAsia 100K Consortium provided critical insight into the extent and nature of genome variation in individuals of Asian ancestry and will be critical in making disease genetic studies more inclusive of all global populations.

Ani Manichaikul, assistant professor of public health sciences in the Center for Public Health Genomics, expressed enthusiasm for the GenomeAsia 100K Project. She claimed that the additional genetic information could augment her research as part of the Multi-Ethnic Study of Atherosclerosis, a cardiovascular disease where fatty deposits accumulate and potentially block arteries. The study currently focuses on Caucasian, African American, Hispanic and Chinese American individuals.

The GenomeAsia project is very useful because there are some instances where particular genetic variants are only observed in particular genetic groups, Manichaikul said. Those markers can be unique to those sequenced through the project, which means we would not have necessarily have observed those particular variants otherwise.

Manichaikul also suggested that expanding existing repositories of hereditary statistics would improve methods of assigning people risk scores for diseases based on their DNA. The National Human Genome Research Institute describes polygenic risk score, which indicates a persons likelihood of certain diseases based on the presence of mutations known to be associated with a given disorder. Companies such as 23andMe have started to provide consumers with this metric, but without a comprehensive database of genomes from different populations, score reliability can decrease.

Since indicators of genetically-linked conditions often appear in certain alleles, or different versions of a gene, knowing whether one has a disease marker can help patients take preventative measures if need be. However, in the absence of comprehensive information on the range of disease markers that appear in different ethnicities, whole populations may lack the potential benefits of this burgeoning healthcare statistic.

The only way we can create risk prediction models that are accurate across populations is if we also have corresponding databases available with individuals that represent that diversity, Manichaikul said.

Following the findings in the preliminary study, GenomeAsia 100K Project collaborators will continue to sequence more genomes of Asian individuals. The hope is that, once researchers have access to the data, insights from 100,000 genomes will drive the development of new therapeutic strategies that will benefit people around the world.

I would like more researchers to have access to this data, Ratan said. This is a resource. Were working to establish these reference datasets, and we would definitely like them to be used.

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Coronavirus Treatment: Hundreds of Scientists Scramble to Find One – The New York Times

Posted: at 6:25 am

Working at a breakneck pace, a team of hundreds of scientists has identified 50 drugs that may be effective treatments for people infected with the coronavirus.

Many scientists are seeking drugs that attack the virus itself. But the Quantitative Biosciences Institute Coronavirus Research Group, based at the University of California, San Francisco, is testing an unusual new approach.

The researchers are looking for drugs that shield proteins in our own cells that the coronavirus depends on to thrive and reproduce.

Many of the candidate drugs are already approved to treat diseases, such as cancer, that would seem to have nothing to do with Covid-19, the illness caused by the coronavirus.

Scientists at Mount Sinai Hospital in New York and at the Pasteur Institute in Paris have already begun to test the drugs against the coronavirus growing in their labs. The far-flung research group is preparing to release its findings at the end of the week.

There is no antiviral drug proven to be effective against the virus. When people get infected, the best that doctors can offer is supportive care the patient is getting enough oxygen, managing fever and using a ventilator to push air into the lungs, if needed to give the immune system time to fight the infection.

If the research effort succeeds, it will be a significant scientific achievement: an antiviral identified in just months to treat a virus that no one knew existed until January.

Im really impressed at the speed and the scale at which theyre moving, said John Young, the global head of infectious diseases at Roche Pharma Research and Early Development, which is collaborating on some of the work.

We think this approach has real potential, he said.

Some researchers at the Q.B.I. began studying the coronavirus in January. But last month, the threat became more imminent: A woman in California was found to be infected although she had not recently traveled outside the country.

That finding suggested that the virus was already circulating in the community.

I got to the lab and said weve got to drop everything else, recalled Nevan Krogan, director of the Quantitative Biosciences Institute. Everybody has got to work around the clock on this.

Dr. Krogan and his colleagues set about finding proteins in our cells that the coronavirus uses to grow. Normally, such a project might take two years. But the working group, which includes 22 laboratories, completed it in a few weeks.

You have 30 scientists on a Zoom call its the most exhausting, amazing thing, Dr. Krogan said, referring to a teleconferencing service.

Viruses reproduce by injecting their genes inside a human cell. The cells own gene-reading machinery then manufactures viral proteins, which latch onto cellular proteins to create new viruses. They eventually escape the cell and infect others.

In 2011, Dr. Krogan and his colleagues developed a way to find all the human proteins that viruses use to manipulate our cells a map, as Dr. Krogan calls it. They created their first map for H.I.V.

That virus has 18 genes, each of which encodes a protein. The scientists eventually found that H.I.V. interacts, in one way or another, with 435 proteins in a human cell.

Dr. Krogan and his colleagues went on to make similar maps for viruses such as Ebola and dengue. Each pathogen hijacks its host cell by manipulating a different combination of proteins. Once scientists have a map, they can use it to search for new treatments.

In February, the research group synthesized genes from the coronavirus and injected them into cells. They uncovered over 400 human proteins that the virus seems to rely on.

The flulike symptoms observed in infected people are the result of the coronavirus attacking cells in the respiratory tract. The new map shows that the viruss proteins travel throughout the human cell, engaging even with proteins that do not seem to have anything to do with making new viruses.

One of the viral proteins, for example, latches onto BRD2, a human protein that tends to our DNA, switching genes on and off. Experts on proteins are now using the map to figure out why the coronavirus needs these molecules.

Kevan Shokat, a chemist at U.C.S.F., is poring through 20,000 drugs approved by the Food and Drug Administration for signs that they may interact with the proteins on the map created by Dr. Krogans lab.

Dr. Shokat and his colleagues have found 50 promising candidates. The protein BRD2, for example, can be targeted by a drug called JQ1. Researchers originally discovered JQ1 as a potential treatment for several types of cancer.

On Thursday, Dr. Shokat and his colleagues filled a box with the first 10 drugs on the list and shipped them overnight to New York to be tested against the living coronavirus.

The drugs arrived at the lab of Adolfo Garcia-Sastre, director of the Global Health and Emerging Pathogens Institute at the Icahn School of Medicine at Mount Sinai Hospital. Dr. Garcia-Sastre recently began growing the coronavirus in monkey cells.

Over the weekend, the team at the institute began treating infected cells with the drugs to see if any stop the viruses. We have started experiments, but it will take us a week to get the first data here, Dr. Garcia-Sastre said on Tuesday.

The researchers in San Francisco also sent the batch of drugs to the Pasteur Institute in Paris, where investigators also have begun testing them against coronaviruses.

If promising drugs are found, investigators plan to try them in an animal infected with the coronavirus perhaps ferrets, because theyre known to get SARS, an illness closely related to Covid-19.

Even if some of these drugs are effective treatments, scientists will still need to make sure they are safe for treating Covid-19. It may turn out, for example, that the dose needed to clear the virus from the body might also lead to dangerous side effects.

This collaboration is far from the only effort to find an antiviral drug effective against the coronavirus. One of the most closely watched efforts involves an antiviral called remdesivir.

In past studies on animals, remdesivir blocked a number of viruses. The drug works by preventing viruses from building new genes.

In February, a team of researchers found that remdesivir could eliminate the coronavirus from infected cells. Since then, five clinical trials have begun to see if the drug will be safe and effective against Covid-19 in people.

Other researchers have taken startling new approaches. On Saturday, Stanford University researchers reported using the gene-editing technology Crispr to destroy coronavirus genes in infected cells.

As the Bay Area went into lockdown on Monday, Dr. Krogan and his colleagues were finishing their map. They are now preparing a report to post online by the end of the week, while also submitting it to a journal for publication.

Their paper will include a list of drugs that the researchers consider prime candidates to treat people ill with the coronavirus.

Whoever is capable of trying them, please try them, Dr. Krogan said.

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AZ adds gene silencing tech to pipeline with Silence Therapeutics deal – – pharmaphorum

Posted: at 6:25 am

AstraZeneca has announced a collaboration with UK biotech Silence Therapeutics to develop gene silencing drugs for cardiovascular, renal, metabolic and respiratory diseases.

The deal could see AZ adding small interfering RNA (siRNA) technology to its pipeline in one of its main areas of research.

Silence Therapeutics says its technology can selectively inhibit any gene in the genome, specifically silencing the production of disease-causing proteins.

The UK biotech says it has achieved an additional level of accuracy by delivering its therapeutic RNA molecules exclusively to target cells.

siRNA is a technology that showed much promise after biologists Andrew Fire and Craig Mello received the Nobel Prize in Physiology or Medicine for discovering the technology in 2006.

But in 2010 it became apparent that it was harder to convert into a working therapy because of the challenge of delivering therapeutic RNA molecules to target tissues and big pharma quickly lost interest.

But companies like Silence have managed to overcome this hurdle and its rival Alnylam made history in 2018 when its siRNA drug Onpattro was approved by the FDA to treat hereditary transthyretin (hATTR) amyloidosis, which causes the build-up of amyloid protein in nerves and organs.

AZ will pay $60 million up front and invest $20 million in Silence, and will pay up to $400 million in milestone payments plus tiered royalties.

The companies expect to work on five targets within the first three years of the collaboration, and AstraZeneca has the option to extend it to a further five targets.

Silence has technology that can inhibit liver-expressed gene targets and the companies will collaborate to find siRNA molecules to other tissues including the heart, kidney and lung.

The UK biotech will design SiRNA molecules against gene targets selected by AZ, and will manufacture material to support toxicology and phase 1 clinical studies.

AZ and Silence will collaborate during the discovery phase, while AZ will lead clinical trials and marketing.

Silence will have the option to negotiate for co-development of two drugs of their choice starting from phase 2.

AZ will pay an option fee of $10 million for each selected target when a drug candidate is nominated, and Silence could receive up to $140 million in development milestones, and up to $250 million in market milestones.

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A coronavirus test can be developed in 24 hours. So why are some countries still struggling to diagnose? – CNN

Posted: at 6:25 am

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Andrew Cuomo said."},{"title":"How designer Christian Siriano is helping fight pandemic","duration":"03:04","sourceName":"CNN","sourceLink":"http://www.cnn.com","videoCMSUrl":"/video/data/3.0/video/style/2020/03/26/fashion-designer-christian-siriano-coronavirus-mask-support-bts-cpt-vpx.cnn/index.xml","videoId":"style/2020/03/26/fashion-designer-christian-siriano-coronavirus-mask-support-bts-cpt-vpx.cnn","videoImage":"//cdn.cnn.com/cnnnext/dam/assets/200326020912-fashion-designer-christian-siriano-coronavirus-mask-support-bts-cpt-vpx-00010101-large-169.jpg","videoUrl":"/videos/style/2020/03/26/fashion-designer-christian-siriano-coronavirus-mask-support-bts-cpt-vpx.cnn/video/playlists/coronavirus/","description":"New York Gov. Andrew Cuomo appealed for assistance in overcoming u003ca href="http://www.cnn.com/style/article/fashion-industry-masks-protective-equipment-covid-19/index.html" target="_blank">shortages of masks u003c/a>and other personal protective equipment (PPE) in the country's worst-hit state. Fashion designer Christian Siriano tells CNN's u003ca href="http://www.cnn.com/profiles/chris-cuomo-profile" target="_blank">Chris Cuomou003c/a> why he answered the call to help.","descriptionText":"New York Gov. Andrew Cuomo appealed for assistance in overcoming u003ca href="http://www.cnn.com/style/article/fashion-industry-masks-protective-equipment-covid-19/index.html" target="_blank">shortages of masks u003c/a>and other personal protective equipment (PPE) in the country's worst-hit state. Fashion designer Christian Siriano tells CNN's u003ca href="http://www.cnn.com/profiles/chris-cuomo-profile" target="_blank">Chris Cuomou003c/a> why he answered the call to help."},{"title":"Covid-19 tips: How to clean your home","duration":"01:43","sourceName":"CNN","sourceLink":"","videoCMSUrl":"/video/data/3.0/video/health/2020/03/25/coronavirus-how-to-clean-disinfect-home-orig-mg-bu-cw.cnn/index.xml","videoId":"health/2020/03/25/coronavirus-how-to-clean-disinfect-home-orig-mg-bu-cw.cnn","videoImage":"//cdn.cnn.com/cnnnext/dam/assets/200325142119-covid-19-house-cleaning-large-169.jpg","videoUrl":"/videos/health/2020/03/25/coronavirus-how-to-clean-disinfect-home-orig-mg-bu-cw.cnn/video/playlists/coronavirus/","description":"The coronavirus can live on surfaces for several days. Here's how to clean and disinfect your home properly to stay safe.","descriptionText":"The coronavirus can live on surfaces for several days. Here's how to clean and disinfect your home properly to stay safe."},{"title":"City official chews out mayor over coronavirus response","duration":"01:46","sourceName":"CNN","sourceLink":"http://www.cnn.com","videoCMSUrl":"/video/data/3.0/video/us/2020/03/24/florida-city-commissioner-mayor-confrontation-covid19-response-eg-orig.cnn/index.xml","videoId":"us/2020/03/24/florida-city-commissioner-mayor-confrontation-covid19-response-eg-orig.cnn","videoImage":"//cdn.cnn.com/cnnnext/dam/assets/200323203629-florida-official-mayor-confrontation-1-large-169.jpg","videoUrl":"/videos/us/2020/03/24/florida-city-commissioner-mayor-confrontation-covid19-response-eg-orig.cnn/video/playlists/coronavirus/","description":"Lake Worth Beach city commissioner Omari Hardy had choice words for the mayor and city manager after dozens of residents had their power shut off while staying indoors during the Covid-19 coronavirus pandemic.","descriptionText":"Lake Worth Beach city commissioner Omari Hardy had choice words for the mayor and city manager after dozens of residents had their power shut off while staying indoors during the Covid-19 coronavirus pandemic."},{"title":"Coronavirus lockdown in Italy: a US citizen's diary","duration":"02:14","sourceName":"CNN","sourceLink":"","videoCMSUrl":"/video/data/3.0/video/world/2020/03/25/coronavirus-italy-lockdown-diary-lon-orig-bks.cnn/index.xml","videoId":"world/2020/03/25/coronavirus-italy-lockdown-diary-lon-orig-bks.cnn","videoImage":"//cdn.cnn.com/cnnnext/dam/assets/200325154701-diary-thumbnail-orig-video-large-169.jpg","videoUrl":"/videos/world/2020/03/25/coronavirus-italy-lockdown-diary-lon-orig-bks.cnn/video/playlists/coronavirus/","description":"Rick Kyle, an American citizen living in Goito, Italy, has been documenting his days inside his home, as the country of 60 million is under a total coronavirus lockdown.","descriptionText":"Rick Kyle, an American citizen living in Goito, Italy, has been documenting his days inside his home, as the country of 60 million is under a total coronavirus lockdown."},{"title":"White House expert urges New Yorkers to self-quarantine","duration":"01:34","sourceName":"CNN","sourceLink":"http://www.cnn.com/","videoCMSUrl":"/video/data/3.0/video/health/2020/03/25/birx-new-york-self-quarantine-warning-sot-vpx.cnn/index.xml","videoId":"health/2020/03/25/birx-new-york-self-quarantine-warning-sot-vpx.cnn","videoImage":"//cdn.cnn.com/cnnnext/dam/assets/200324210830-deborah-birx-march-24-2020-large-169.jpg","videoUrl":"/videos/health/2020/03/25/birx-new-york-self-quarantine-warning-sot-vpx.cnn/video/playlists/coronavirus/","description":"Dr. Deborah Birx, the White House's coronavirus response coordinator, says that anyone who has recently left New York should self-quarantine for 14 days in an effort to slow the spread of the virus in the US.","descriptionText":"Dr. Deborah Birx, the White House's coronavirus response coordinator, says that anyone who has recently left New York should self-quarantine for 14 days in an effort to slow the spread of the virus in the US."},{"title":"Doctor describes dire situation in hospitals treating virus","duration":"02:11","sourceName":"CNN","sourceLink":"http://www.cnn.com/","videoCMSUrl":"/video/data/3.0/video/health/2020/03/25/craig-spencer-ny-hospital-trump-magical-thinking-sot-ac-vpx.cnn/index.xml","videoId":"health/2020/03/25/craig-spencer-ny-hospital-trump-magical-thinking-sot-ac-vpx.cnn","videoImage":"//cdn.cnn.com/cnnnext/dam/assets/200325002947-doctor-craig-spencer-may-24-2020-01-large-169.jpg","videoUrl":"/videos/health/2020/03/25/craig-spencer-ny-hospital-trump-magical-thinking-sot-ac-vpx.cnn/video/playlists/coronavirus/","description":"Columbia University Medical Center's Dr. Craig Spencer talks with CNN's u003ca href="http://www.cnn.com/profiles/anderson-cooper-profile" target="_blank">Anderson Cooper u003c/a>about the dire situation facing hospitals as they try to treat patients with coronavirus.","descriptionText":"Columbia University Medical Center's Dr. Craig Spencer talks with CNN's u003ca href="http://www.cnn.com/profiles/anderson-cooper-profile" target="_blank">Anderson Cooper u003c/a>about the dire situation facing hospitals as they try to treat patients with coronavirus."},{"title":"Nurse with coronavirus delivers powerful message on economy","duration":"02:35","sourceName":"CNN","sourceLink":"https://www.cnn.com/","videoCMSUrl":"/video/data/3.0/video/health/2020/03/25/washington-nurse-karen-goheen-coronavirus-parents-intv-cpt-vpx.cnn/index.xml","videoId":"health/2020/03/25/washington-nurse-karen-goheen-coronavirus-parents-intv-cpt-vpx.cnn","videoImage":"//cdn.cnn.com/cnnnext/dam/assets/200325000146-cuomo-nurse-split-large-169.jpg","videoUrl":"/videos/health/2020/03/25/washington-nurse-karen-goheen-coronavirus-parents-intv-cpt-vpx.cnn/video/playlists/coronavirus/","description":"Washington state nurse Karen Goheen's family was impacted by the coronavirus pandemic when both she and her parents tested positive for the virus.","descriptionText":"Washington state nurse Karen Goheen's family was impacted by the coronavirus pandemic when both she and her parents tested positive for the virus."},{"title":"US-China relationship worsens over coronavirus","duration":"02:59","sourceName":"CNN","sourceLink":"https://www.cnn.com/","videoCMSUrl":"/video/data/3.0/video/world/2020/03/25/china-us-tensions-coronavirus-covid-19-disinformation-marquardt-pkg-intl-hnk-vpx.cnn/index.xml","videoId":"world/2020/03/25/china-us-tensions-coronavirus-covid-19-disinformation-marquardt-pkg-intl-hnk-vpx.cnn","videoImage":"//cdn.cnn.com/cnnnext/dam/assets/191223124913-trump-jinping-tradewar-large-169.jpg","videoUrl":"/videos/world/2020/03/25/china-us-tensions-coronavirus-covid-19-disinformation-marquardt-pkg-intl-hnk-vpx.cnn/video/playlists/coronavirus/","description":"As the world declares war on Covid-19, the China-US relations have taken a turn for the worse as the countries are pointing fingers at each other over misinformation about the virus. u003ca href="http://www.cnn.com/profiles/alex-marquardt" target="_blank">CNN's Alex Marquardt u003c/a>reports. ","descriptionText":"As the world declares war on Covid-19, the China-US relations have taken a turn for the worse as the countries are pointing fingers at each other over misinformation about the virus. u003ca href="http://www.cnn.com/profiles/alex-marquardt" target="_blank">CNN's Alex Marquardt u003c/a>reports. "},{"title":"Some Americans still aren't practicing social distancing ","duration":"01:34","sourceName":"CNN","sourceLink":"","videoCMSUrl":"/video/data/3.0/video/us/2020/03/18/coronavirus-social-distancing-church-police-orig-jk.cnn/index.xml","videoId":"us/2020/03/18/coronavirus-social-distancing-church-police-orig-jk.cnn","videoImage":"//cdn.cnn.com/cnnnext/dam/assets/200324162919-louisiana-church-service-1000-people-large-169.jpg","videoUrl":"/videos/us/2020/03/18/coronavirus-social-distancing-church-police-orig-jk.cnn/video/playlists/coronavirus/","description":"Across the country at churches, beaches, and other gathering spaces, some Americans are ignoring pleas from officials to stay away from each other during the Covid-19 pandemic.","descriptionText":"Across the country at churches, beaches, and other gathering spaces, some Americans are ignoring pleas from officials to stay away from each other during the Covid-19 pandemic."},{"title":"Men 50% more likely to die from pandemic. Here's the data","duration":"02:53","sourceName":"CNN","sourceLink":"https://www.cnn.com","videoCMSUrl":"/video/data/3.0/video/world/2020/03/24/men-higher-gender-mortality-rate-coronavirus-foster-pkg-vpx.cnn/index.xml","videoId":"world/2020/03/24/men-higher-gender-mortality-rate-coronavirus-foster-pkg-vpx.cnn","videoImage":"//cdn.cnn.com/cnnnext/dam/assets/200324160853-men-mortality-data-large-169.jpg","videoUrl":"/videos/world/2020/03/24/men-higher-gender-mortality-rate-coronavirus-foster-pkg-vpx.cnn/video/playlists/coronavirus/","description":"Around the world, data shows that more men seem to be dying from the coronavirus pandemic than woman. CNN's Max Foster shares the reasons the US should take note. ","descriptionText":"Around the world, data shows that more men seem to be dying from the coronavirus pandemic than woman. CNN's Max Foster shares the reasons the US should take note. "},{"title":"FEMA head confirms first use of Defense Production Act","duration":"01:12","sourceName":"CNN","sourceLink":"http://www.cnn.com","videoCMSUrl":"/video/data/3.0/video/politics/2020/03/24/peter-gaynor-fema-defense-production-act-newday-vpx.cnn/index.xml","videoId":"politics/2020/03/24/peter-gaynor-fema-defense-production-act-newday-vpx.cnn","videoImage":"//cdn.cnn.com/cnnnext/dam/assets/200324114000-peter-gaynor-fema-administrator-large-169.jpg","videoUrl":"/videos/politics/2020/03/24/peter-gaynor-fema-defense-production-act-newday-vpx.cnn/video/playlists/coronavirus/","description":"FEMA Administrator Peter Gaynor says the Defense Production Act is being used to address a shortage of supplies needed to fight the novel coronavirus pandemic.","descriptionText":"FEMA Administrator Peter Gaynor says the Defense Production Act is being used to address a shortage of supplies needed to fight the novel coronavirus pandemic."}],'js-video_headline-featured-1qnmnix','',"js-video_source-featured-1qnmnix",true,true,'coronavirus');if (typeof configObj.context !== 'string' || configObj.context.length

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A coronavirus test can be developed in 24 hours. So why are some countries still struggling to diagnose? - CNN

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Vycellix and Avectas Announce Collaboration to Advance Next-Generation Solutions for the Optimized Manufacture of Cell & Gene Therapies – BioSpace

Posted: at 6:25 am

TAMPA, Fla. & DUBLIN--(BUSINESS WIRE)-- Vycellix, Inc., an immuno-discovery cell & gene therapy company, and Avectas Limited, a cell engineering technology business, today jointly announced that the companies have entered into a collaboration agreement to develop proprietary approaches for cell-based immunotherapeutic products.

The companies will collaborate on the delivery of Vycellix's novel RNA immunomodulator VY-M using Avectas' cell engineering platform, Solupore. The collaboration will address current limitations for cell-based therapies, in particular with respect to the need to accelerate the manufacturing process, reduce the cost of manufacture, and ultimately improve patient outcomes.

"We are delighted to partner with Vycellix and join forces in the development of novel cell-based products," stated Michael Maguire, Ph.D., CEO of Avectas. "We believe Solupore will play a critical role in the manufacture of cell-based therapies and will support a path towards effective patient outcomes."

According to Vycellixs President, Douglas Calder, Solupore represents a new paradigm for delivery of transgenes, and our initial studies will evaluate Solupore to deliver our product candidate, VY-M, to T cells and natural killer (NK) cells. We expect to accelerate the expansion-time of T cells and NK cells by decreasing the non-dividing lag time, resulting in much shorter vein-to-vein delivery-time to patients. The studies will be conducted at Avectas Dublin-based facility and at Karolinska Institutet, Stockholm, Sweden.

Both Vycellix and Avectas are collaborative partners within NextGenNK, a newly established competence center for development of next-generation NK cell-based cancer immunotherapies based at Karolinska Institutet, Stockholm, Sweden. It is envisioned that Vycellix and Avectas will further expand their collaboration within the NextGenNK constellation.

We are excited to see the NextGenNK Competence Center catalyzing interactions among its industrial partners to advance NK cell-based immunotherapies, said Hans-Gustaf Ljunggren, M.D., Ph.D., Director of the NextGenNK Competence Center. The present collaboration may pave the way for similar collaborations among NextGenNK partners.

About Vycellix, Inc.: Vycellix is a private, immuno-discovery, life science company at the forefront of innovation in the development of cell & gene-based therapies targeting indications in, but not limited to, hematology/oncology, autoimmunity/chronic inflammatory diseases, and organ/tissue transplantation.

The Companys portfolio of transformational platform technologies encompass novel tools urgently sought after to enable broad global adoption of advanced therapies including: 1) the ability to generate Universal Cells (VY-UC), without the need to alter expression of any of the cellular components that control self-recognition (HLA Class I or II), obviating the need for immune-suppressive drugs and redefining the path towards off-the-shelf therapies; 2) the ability to amplify cell-potency through the upregulation of internal cytotoxic mechanisms (VY-X); 3) the ability to accelerate the expansion of cells for immunotherapy by near-elimination of non-dividing lag time to leap forward to shorter vein-to-vein time with expanded cells (VY-M); and, 4) the ability to markedly enhance gene transduction levels using viral vectors with implications for autologous and allogeneic CAR-T and CAR-NK cell development (VY-OZ).

The Companys platforms were all discovered by scientists at the world-renowned Karolinska Institutet (KI) in Stockholm, Sweden. KI is globally recognized for its Nobel Assembly, which awards the Nobel Prize in Physiology or Medicine. For more information, please visit the Companys website at: http://www.Vycellix.com and follow its Twitter feed at: @Vycellix.

About Avectas Limited: Avectas is a cell engineering technology business developing a unique delivery platform to enable the ex-vivo manufacture of our partners' gene-modified cell therapy products, which will retain high in-vivo functionality. Our vision is to be a leading non-viral cell engineering technology provider, integrated into manufacturing processes for multiple autologous and allogeneic therapies, commercialized through development and license agreements. For more information, please visit the Company's website at http://www.avectas.com.

Forward Looking Statements: This press release contains forward-looking statements. All statements other than statements of historical facts are forward-looking statements, including those relating to future events. In some cases, forward-looking statements can be identified by terminology such as plan, expect, anticipate, may, might, will, should, project, believe, estimate, predict, potential, intend, or continue and other words or terms of similar meaning. These statements include, without limitation, statements related to the pre-clinical, regulatory, clinical and/or commercial development and all anticipated uses of VY-OZ, VY-X, VY-M and VY-UC, and the Companys plans for seeking out-licensing opportunities for these assets. These forward-looking statements are based on current plans, objectives, estimates, expectations and intentions, and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with immuno-discovery product development, including risks associated with advancing products to human clinical trials and/or ultimately regulatory and commercial success which is subject to the uncertainty of regulatory approval, market adoption and other risks and uncertainties affecting Vycellix and its development programs. Other risks and uncertainties of which Vycellix is not currently aware may also affect Vycellixs forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof. Vycellix undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200325005026/en/

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Vycellix and Avectas Announce Collaboration to Advance Next-Generation Solutions for the Optimized Manufacture of Cell & Gene Therapies - BioSpace

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Meet a superhero that fights breast cancer, neurofibromin – Baylor College of Medicine News

Posted: at 6:25 am

It is well known that neurofibromin (NF1), a tumor suppressor produced by the NF1 gene, keeps cancer growth in check by repressing the activity of a cancer driver gene called Ras. It then follows that when NF1 is lost, Ras can drive cancer growth by promoting treatment resistance and metastasis. NF1, however, can do more than regulate Ras.

Drs. Eric C. Chang, Matthew Ellis and Zeyi Zheng at Baylor College of Medicine and their colleagues have discovered new insights into the function of neurofibromin that improve our understanding of breast cancer resistance and suggest novel therapeutic approaches to overcome it.

The team first studied the importance of neurofibromin loss in a study they published in 2018. In this study, they sequenced tumor DNA seeking for mutations that can promote resistance to tamoxifen, a drug commonly used to prevent relapses from estrogen receptor positive (ER+) breast cancer.

When we examined the mutational patterns in NF1, we observed that poor patient outcome only occurred when neurofibromin was lost, not through mutations that selectively affect Ras regulation. This suggested that neurofibromin may have more than one function, said Chang, co-corresponding author of this work and associate professor in the Department of Molecular and Cellular Biology and a member in the Dan L Duncan Comprehensive Cancer Centers Lester and Sue Smith Breast Center.

This thought triggered studies, spearheaded by Zheng in Changs lab, into the function of neurofibromin in ER+ breast cancer cells. One of his early experiments showed that when expression of NF1 is inhibited (to mimic neurofibromin loss in tumors), the resulting ER+ breast cancer cells were stimulated by tamoxifen instead of inhibited, as it usually happens. Furthermore, these neurofibromin-depleted cells became sensitive to a very low concentration of estradiol, a form of estrogen.

The clinical relevance of these findings was immediately apparent because it suggested that tamoxifen or aromatase inhibitors, which lower estrogen levels available to the cancer cells, would be the wrong choice for treatment when neurofibromin is lost by the tumor, said Ellis, co-corresponding author and professor and director of the Lester and Sue Smith Breast Center. Dr. Ellis also is a McNair Scholar at Baylor.

Follow-up gene expression studies all strongly suggested that neurofibromin behaves like a classic ER co-repressor.

A co-repressor must bind ER directly, but the group hesitated to conduct such an experiment without more evidence because it is not trivial to do so, Chang said.

A breakthrough came when Dr. Charles Foulds, a co-author on the paper and assistant professor at the Center for Precision Environmental Health at Baylor, searched the Epicome, a massive proteomic database created by Dr. Anna Malovannaya and Dr. Jun Qin, also at Baylor. This is a part of an effort by Dr. Bert OMalley, chancellor and professor of Baylors Department of Molecular and Cellular Biology to comprehensively document all the proteins associated with ER.

Foulds found neurofibromin in the database, which encouraged the team to ultimately investigate whether estrogen receptor and neurofibromin interacted directly. However, to seriously consider NF1 as an ER co-repressor, there was still another missing piece of the puzzle.

One day Charles casually asked me whether neurofibromin had a region rich in the amino acids leucine and isoleucine, because co-repressors use these regions or motifs to bind ER, and it dawned on me that neurofibromin indeed does, Chang said. In fact, neurofibromin has two such motifs that mediate ER binding in a cooperative manner. These motifs are frequently mutated in cancer, but are not required for Ras regulation.

Since tamoxifen or aromatase inhibitors were found to be ineffective for neurofibromin-deficient ER+ breast cancer tumors, the researchers worked with animal models to determine whether the ER-degrading drug fulvestrant was still effective. However, fulvestrant only temporarily inhibited tumor growth because secondary Ras-dependent fulvestrant resistance was induced by neurofibromin loss. This Ras-dependent growth phase could be inhibited with the addition of a MEK inhibitor, which shuts off a key signaling pathway downstream of Ras.

The team validated this combination treatment strategy using a patient-derived xenograft (PDX) mouse model. In this model, a section of a human tumor taken from a patient is directly transplanted into a mouse under conditions that maintain the genomics and drug response of the original human tumor from which it was derived (Cell Reports, 2013). In this case, this PDX was derived from a patient who failed several lines of endocrine therapy and had already developed fulvestrant resistance.

The results of the combination of fulvestrant to degrade ER and a MEK inhibitor (e.g., selumetinib or binimetinib) to inhibit Ras downstream signaling, were encouraging the tumor shrunk to almost undetectable levels, Chang said.

Our next goal is to test this combination therapy in clinical trials in order to determine its therapeutic potential in the clinic.

Neurofibromin is lost in at least 10 percent of metastatic ER+ tumors. As a result of these new data, we are now working on a clinical trial that combines a MEK inhibitor with fulvestrant, said Ellis, Susan G. Komen scholar and associate director of Precision Medicine at the Dan L Duncan Comprehensive Cancer Center at Baylor. Interestingly, MEK inhibitors are also being used to control peripheral nerve tumors in patients with neurofibromatosis, where a damaged NF1 gene is inherited. Our findings contribute to an understanding of why female neurofibromatosis patients also have a much higher incidence of breast cancer.

Other contributors to this work include Meenakshi Anurag, Jonathan T. Lei, Jin Cao, Purba Singh, Jianheng Peng, Hilda Kennedy, Nhu-Chau Nguyen, Yue Chen, Philip Lavere, Jing Li, Xin-Hui Du, Burcu Cakar, Wei Song, Beom-Jun Kim, Jiejun Shi, Sinem Seker, Doug W. Chan, Guo-Qiang Zhao, Xi Chen, Kimberly C. Banks, Richard B. Lanman, Maryam Nemati Shafaee, Xiang H.-F. Zhang, Suhas Vasaikar, Bing Zhang, Susan G. Hilsenbeck, Wei Li and Charles E. Foulds. The authors are affiliated with one or more of the following institutions: Baylor College of Medicine, Chongqing Medical University, Adrienne Helis Malvin Medical Research Foundation, Zhengzhou University and Guardant Health.

This work appears in Cancer Cell,

See the publication for a complete list of the sources of support for this work.

By Ana Mara Rodrguez, Ph.D.

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Meet a superhero that fights breast cancer, neurofibromin - Baylor College of Medicine News

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Twelve Women Who Have Shaped The History of the BioHealth Capital Region – BioBuzz

Posted: at 6:25 am

The BioHealth Capital Region (BHCR) and its life science ecosystem have a rich and deep history of pioneering scientific innovation, research, development, and commercialization. The regions history has been written by life science anchor companies, scientific research universities, government research organizations, rich startup culture, and serial entrepreneurs, all of whom have played critical roles in transforming the BHCR into one of the most innovative and productive biocluster in the world.

Contributions to the BHCRs legacy of life science achievement have emerged from all staffing levels, various labs, countless executive teams, numerous entrepreneurs and biohub support organizations. Contributions have arisen from an intricate tapestry of backgrounds and cultures.

Women, in particular, have had a strong hand in shaping the history of the BHCR. In celebration of Womens History Month, were taking a closer look at the achievements of female life science leaders that have laid the groundwork for the next generation of women trailblazers in the BHCR and made the region what it is today.

Dr. Fraser is one of the most influential figures in BHCR history. In 1995, she was the first to map the complete genetic code of a free-living organism while at the Institute for Genomic Research (TIGR) in Rockville, Maryland. It was there that the automation of the DNA sequencing process made the idea of large-scale sequencing efforts tangible. As President and Director of TIGR, Fraser and her team gained worldwide public notoriety for its involvement in the Human Genome Project, which was completed in 2000 with the presentation of a working draft of the fully sequenced human genome.

As a leader, Fraser provided her researchers with the infrastructure to collaborate and apply multi-disciplinary team science and empowered them to think big. She is also most importantly known for how she challenged her team to ask the right questions, which is the root of scientific progress and success.

Her work at TIGR and as part of the Human Genome Project are foundational events in the regions history, as it marked the BHCR as the epicenter of genomic research and helped spark the regions biotech boom. In fact, it was a controversial partnership with TIGR that gave Human Genome Sciences(HGSi) the first opportunity to utilize any sequences emerging from TIGR labs. The mass of genetic information and sequences, especially that associated with diseases, that HGSi acquired catapulted them into biotech history and an important anchor company within the region.

Dr. Fraser is widely viewed as a pioneer and global leader in genomic medicine; she has published approximately 320 scientific publications and edited three books; she is also one of the most widely cited microbiology experts in the world. She founded the Institute for Genome Sciences at the University of Maryland in 1997. The institute currently holds 25 percent of the funding thats been awarded by the Human Microbiome Project and has been referred to as The Big House in genetics.

Dr. Judy Britz is yet another female life science pioneer that put the BHCR on the map. While working as a research scientist at Electro-Nucleonics Inc., Dr. Britz developed one of the first licensed blood screening tests for HIV, and launching a storied career that has spanned approximately 25 years. She is also a serial entrepreneur that has successfully raised $50M in capital and served as the top executive for two highly successful Maryland-located companies.

Dr. Britz was the first woman to lead the states biotech initiative as the first announced Executive Director of the Maryland Biotech Center. The center was launched under the Maryland Department of Commerce to deploy a strategic life science economic development plan under Governor Martin OMalleys $1.3B, 2020 Vision and to be a one-stop-shop and information center to promote and support biotechnology innovation and entrepreneurship in Maryland.

Judy was the first woman to lead Marylands life sciences initiative, bringing industry experience and perspective to the states economic development activities, a focus still maintained under Governor Hogans leadership today, shared Judy Costello, Managing Director, Economic Development BioHealth Innovation, Inc., who served as Deputy Director under Dr. Britz.

Much of the work done by Dr. Britz and her team laid the foundation and seeded the commercialization efforts that have blossomed into the thriving #4 Biotech Hub that we have today.

GeneDx was founded by Dr. Bale and Dr. John Compton in 2000. The company recently celebrated its 20th anniversary. Since its founding, GeneDx has become a global leader in genomics and patient testing. Under her leadership, the Gaithersburg, Maryland company has played an important role in the history of genetic sequencing and the rise of the BHCR as a global biohealth cluster.

GeneDx was the very first company to commercially offer NGS (Next Generation Sequencing) testing in a CLIA (Clinical Laboratory Improvement Amendments) lab and has been at the leading edge of genetic sequencing and testing for two decades. The companys whole exome sequencing program and comprehensive testing capabilities are world-renowned.

Prior to launching GeneDx, Dr. Bale spent 16 years at NIH, the last nine as Head of the Genetic Studies Section in the Laboratory of Skin Biology. She has been a pioneer during her storied career, publishing over 140 papers, chapters and books in the field. Her 35-year career includes deep experience in clinical, cytogenetic, and molecular genetics research.

Prior to being named CEO and Chair of the Board of Sequella in 1999, Dr. Nacy was the Chief Science Officer and an Executive VP at EntreMed, Inc. EntreMed was one of the most influential BHCR companies in the 1990s. EntreMed, MedImmune, Human Genome Sciences and Celera Genomics all played critical roles in creating the globally recognized, top biocluster that the BHCR has become.

After earning her Ph.D. in biology/microbiology from Catholic University, Nacy did her postdoc work at the Walter Reed Army Institute of Research in the Department of Rickettsial Diseases; her postdoc performance earned a full-time position at Walter Reed that started a 17-year career at the institute. After a highly successful run, Nacy left Walter Reed to join EntreMed.

Today, Dr. Nacy leads Rockville, Marylands Sequella, a clinical-stage pharmaceutical company focused on developing better antibiotics to fight drug-resistant bacterial, fungal and parasitic infections. Sequellas pipeline of small molecule infectious disease treatments have the potential to improve the treatment and outcomes for the over 3 billion people worldwide that are impacted by increasingly drug-resistant infectious diseases.

Emmes Corporation is the largest woman-led organization in the BHCR and is headed by Dr. Lindblad, who started her career at Emmes in 1982 as a biostatistician. She has been with Emmes for nearly 40 years, ascending to become VP in 1992, Executive VP in 2006 and ultimately the companys CEO in late summer of 2013.

Dr. Lindblad has published more than 100 publications and presentations has served as a reviewer of grant and contract applications for the National Institutes of Health (NIH) and has chaired or served on Safety and Data Monitoring Committees across multiple disease areas. Emmes is a life science anchor company for the BHCR, employing more than 600 staff globally with its headquarters in Rockville, Maryland.

Under Kings leadership, GlycoMimetics (GMI), an oncology-focused biotech, went public, secured an exclusive global licensing agreement with Pfizer and was instrumental in raising significant amounts of capital for the company. She was also the first woman Chair of Biotechnology Innovation Associations (BIO, 2013-14), where she still plays an active role on BIOs Executive Committee.

A graduate of Dartmouth College and Harvard Business School, King has had a celebrated career in both biopharma and finance. Prior to becoming CEO of GMI, King served as an Executive in Residence for New Enterprise Associates (NEA), one of the leading venture capital firms in the U.S. She has also held the position of Senior Vice President of Novartis-Corporation. King joined Novartis after a remarkable ten year run with Genetic Therapy, Inc. where she was named CEO after helping Genetic Therapy navigate the organization through various growth stages, including the companys sale to Novartis. King was named the Maryland Tech Councils Executive of the Year in 2013, the Top 10 Women in Biotech by FierceBio and has served on multiple boards across her career.

Dr. Connolly has had a pioneering career in the life sciences. She was the very first woman to graduate from Johns Hopkins Universitys Biomedical Engineering Doctoral Program in 1980. She was also a member of the first female undergraduate class entering Stevens Institute of Technology in 1971.

For decades, Dr. Connolly tirelessly worked to build up what is now known as the BHCR. In 1997, shortly before the region gained wider recognition as a biotech hub, she was the first person to be designated the state of Marylands biotechnology representative. Dr. Connollys career has spanned academia, government, and industry, including co-founding a startup and working as the Business Development Director for EntreMed, Inc., an original BHCR anchor company. She is the former Director of Maryland Industrial Partnerships Program (MIPS) and was inducted into the College of Fellows by the American Institute for Medical and Biological Engineering (AIMBE) in 2013.

Dr. Kirschstein played an enormous role in shaping the BHCR as NIH Deputy Director from 1993 to 1999 during the regions early formative years. She also served as Acting Director of NIH in 1993 and from 2000 to 2002. A pathologist by training, she received her medical degree from Tulane University in 1951 and went on to a long, successful career at the Division of Biologics Standards that lasted from 1957 to 1972.

While at the Division of Biologics Standards, Dr. Kirschstein played an important role in testing the safety of viral vaccines and helped select the Sabin polio vaccine for public use. She eventually ascended to Deputy Director of the group in 1972 and was later appointed the Deputy Associate Commissioner for Science at the FDA. In 1974 she became the Director of the National Institute of Medical Sciences at NIH and served in that role for 19 years.

Her awards and accolades are too numerous to list, but one notable honor came in 2000 when she received the Albert B. Sabin Heroes of Science Award from the Americans for Medical Progress Education Foundation.

Lastly, we want to recognize four additional women for their contributions to launching an organization that has impacted thousands of women by promoting careers, leadership, and entrepreneurship for women in the life sciences Women In Bio.

Women In Bio (WIB), one of the most important and influential support organizations for women in the life sciences, was founded in 2002 to help women entrepreneurs and executives in the Baltimore-Washington-Northern Virginia area build successful bioscience-related businesses. WIB started as a BHCR organization but has expanded its footprint to 13 chapters across the U.S. with 225 volunteer leaders and 2,600 members. The non-profit group has created a forum for female life science entrepreneurs and executives based on its core philosophy of women helping women.

WIB founders are Anne Mathias, a local venture capitalist and current Senior Strategist with Vanguard;

Elizabeth Gray, co-founder of Gabriel Pharma and current Partner at Willkie Farr & Gallagher LLP;

Robbie Melton, former Director of Entrepreneurial Innovation at TEDCO and current Director of Kauai County, Hawaiis Office of Economic Development;

and Cynthia W. Hu, COO, and General Counsel at CASI Pharmaceuticals.

In conclusion, we can not fairly capture the true history of life science and the BioHealth Capital Region without giving special recognition to Henrietta Lacks. In 1951 a Johns Hopkins researcher created the first immortal human cell line from cervical cancer cells taken from Lacks. That cell line, known as HeLa, is the oldest and most commonly used human cell line which was essential in developing the polio vaccine and has been used in scientific landmarks such as cloning, gene mapping and in vitro fertilization.

Though she was a black tobacco farmer from southern Virginia, her impact on science and medicine is unquestionable. She never knew that the Doctor took a piece of her tumor that would be used by scientists who had been trying to grow tissues in culture for decades without success. For some reason, that is still unknown, but her cells never died and the first immortal human cell line was born.

Thank you to all of the women who have been so influential in shaping the field of science, the industry of biotechnology and the BioHealth Capital Region.

Steve has over 20 years experience in copywriting, developing brand messaging and creating marketing strategies across a wide range of industries, including the biopharmaceutical, senior living, commercial real estate, IT and renewable energy sectors, among others. He is currently the Principal/Owner of StoryCore, a Frederick, Maryland-based content creation and execution consultancy focused on telling the unique stories of Maryland organizations.

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Twelve Women Who Have Shaped The History of the BioHealth Capital Region - BioBuzz

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