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Radiologist also will lead clinical services

Radiologist Debbie Lee Bennett, MD, has been named chief of the breast imaging section at Washington University School of Medicine in St. Louis.

After a national search, noted radiologist Debbie Lee Bennett, MD, has been named chief of breast imaging forMallinckrodt Institute of Radiology(MIR) at Washington University School of Medicine in St. Louis.

Bennett will oversee screening and diagnostic mammography services offered throughSiteman Cancer CenteratBarnes-Jewish Hospitaland Washington University School of Medicine, including at the Joanne Knight Breast Health Center.

Bennett comes to the university from Saint Louis University School of Medicine, where she had served on the faculty since 2014, most recently as an associate professor. There, she established the breast imaging section and began serving as its chief in 2015.

We are very excited that someone with Dr. Bennetts proven leadership and experience is joining our breast imaging faculty, said Richard L. Wahl, MD, the Elizabeth Mallinckrodt Professor of Radiology, head of the radiology department at the School of Medicine and director of MIR. She also is a welcome addition to our team of clinical experts, whose skill and experience make a difference in the lives of our patients.

Bennett serves on several committees of professional and academic organizations, including the American College of Radiology, Society of Breast Imaging, American Board of Radiology and Radiological Society of North America. She also is engaged in outreach and education, and has mentored many residents who have gone on to pursue careers in breast imaging.

After an internship in internal medicine at Vanderbilt University Medical Center, she completed a residency in diagnostic radiology and a fellowship in breast imaging, both at Massachusetts General Hospital.

She earned her medical degree from Harvard Medical School and bachelors degree from Princeton University, graduating magna cum laude from both institutions.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff ofBarnes-JewishandSt. Louis Childrenshospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked toBJC HealthCare.

Siteman Cancer Center, ranked among the top cancer treatment centers by U.S. News & World Report, also is one of only a few cancer centers to receive the highest rating of the National Cancer Institute (NCI) exceptional. Comprising the cancer research, prevention and treatment programs ofBarnes-Jewish HospitalandWashington University School of Medicinein St. Louis, Siteman treats adults at six locations and partners withSt. Louis Childrens Hospitalin the treatment of pediatric patients. Siteman is Missouris only NCI-designated Comprehensive Cancer Center and the states only member of the National Comprehensive Cancer Network. Through theSiteman Cancer Network, Siteman Cancer Center works with regional medical centers to improve the health and well-being of people and communities by expanding access to cancer prevention and control strategies, clinical studies and genomic and genetic testing, all aimed at reducing the burden of cancer.

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Bennett named chief of breast imaging section - Washington University School of Medicine in St. Louis

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Experts propose decentralized system to monitor wildlife markets, other hotspots

A juvenile saddleback tamarin is measured as part of an annual health check of a population of three primate species in southeastern Peru. In a perspective article published July 9 in Science, a team of wildlife biologists, infectious disease experts, and others propose a decentralized, global wildlife biosurveillance system to identify before the next pandemic emerges animal viruses that have the potential to cause human disease.

The virus that causes COVID-19 probably originated in wild bats that live in caves around Wuhan, China, and may have been passed to a second animal species before infecting people, according to the World Health Organization. Many of the most devastating epidemics of recent decades including Ebola, avian influenza and HIV/AIDS were triggered by animal viruses that spilled over into people. Despite the ever-present danger of a new virus emerging and sparking a worldwide pandemic, there is no global system to screen for viruses in wild animals that eventually may jump to humans.

In a perspective article published July 9 in Science, a diverse group of infectious disease experts, ecologists, wildlife biologists and other experts argue that a decentralized global system of wildlife surveillance could and must be established to identify viruses in wild animals that have the potential to infect and sicken people before another pandemic begins.

Its impossible to know how often animal viruses spill over into the human population, but coronaviruses alone have caused outbreaks in people three times in the last 20 years, said co-author Jennifer A. Philips, MD, PhD, referring to the SARS, MERS and COVID-19 epidemics. Philips is an associate professor of medicine and co-director of theDivision of Infectious Diseasesat Washington University School of Medicine in St. Louis. Even a decade ago it would have been difficult to conduct worldwide surveillance at the human-wildlife interface. But because of technological advances, it is now feasible and affordable, and it has never been more obvious how necessary it is.

Every animal has its own set of viruses, with some overlap across species. Often, an animal species and its viruses have lived together for so long that theyve adapted to one another, and the viruses cause either no symptoms or only mild to moderate disease. But when different animal species that dont normally have much contact are brought together, viruses have the opportunity to jump from one species to another. Most viruses dont have the genetic tools to infect another species. But viruses with such tools can be lethal to a newly infected species with no natural immunity.

Human activity is making such spillover events more and more likely. As the population of the world continues to grow, the demand for natural resources skyrockets. People push into wild areas to make space for new homes and businesses, and to access resources to fuel their economies and lifestyles. Wild animals are caught and sold for consumption, or as exotic pets at wildlife markets, where diverse species are jumbled together under crowded and unsanitary conditions. Wild-animal parts are shipped around the world as trinkets or ingredients for traditional or alternative medicines.

And yet there is no international system set up to screen for disease-causing viruses associated with the movement of wildlife or wildlife products.

In the lead up to this article, I spoke with friends and colleagues around the world who do wildlife research in Madagascar, Indonesia, Peru, Ecuador and asked them, Where do you take your samples for screening? said co-author Gideon Erkenswick, PhD, a postdoctoral research associate in Philips lab. Erkenswick is also the director of Field Projects International, a nonprofit organization dedicated to the study and conservation of tropical ecosystems. In almost every situation, the answer was Nowhere. Locally, there is nobody with dedicated time and resources to do this work. To find new disease-causing viruses, we have to find willing foreign collaborators, then get samples out of the country, which is difficult and expensive.

Philips, Erkenswick, and colleagues in the Wildlife Disease Surveillance Focus Group that authored the Science paper, suggest the establishment of a global surveillance network to screen wild animals and their products at hotspots such as wildlife markets. The idea would be to have local teams of researchers and technicians extract viral genomes from animal samples, rapidly sequence them on site and upload the sequences to a central database in the cloud. The cost and size of the necessary scientific equipment has dropped in recent years, making such screening affordable even in resource-limited settings where most such hotspots are located.

Theres now a genetic sequencer available that is literally the size of a USB stick, Erkenswick said. You could bring that and a few other supplies into a rainforest and analyze a sample for sequences associated with disease-causing viruses on site in a matter of hours. I mean, if you do chance upon something like the virus that causes COVID-19, do you really want to be collecting it, storing it, transporting it, risking further exposure, sample degradation, and adding months or years of delay, before you figure out what youve got? There are people with the expertise and skills to do this kind of work safely pretty much everywhere in the world, they just havent been given the tools.

Once viral sequences are uploaded, researchers around the world could help analyze them to identify animal viruses that may be a threat to people and to develop a better understanding of the universe of viruses that thrive in different environments. By comparing genomic sequence data, researchers can identify what family an unknown virus belongs to and how closely it is related to any disease-causing viruses. They can also identify whether a virus carries genes associated with the ability to cause disease in people.

By knowing the diversity out there, and tracking its evolution, we can ensure that we stay ahead of whats in wildlife populations and at the wildlife-human interface, Philips said. In the past, before modern transportation, spillover events would have been local and spread slowly, giving people elsewhere time to respond. But now the world is so small that an event in one place puts the whole world at risk. This is not someone elses problem. Its everyones problem.

Watsa M and the Wildlife Disease Surveillance Focus Group. Rigorous wildlife disease surveillance: A decentralized model could address global health risks associated with wildlife exploitation. Science. July 10, 2020. DOI: 10.1126/science.abc0017

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Global wildlife surveillance could provide early warning for next pandemic - Washington University School of Medicine in St. Louis

BOULDER, Colo.--(BUSINESS WIRE)--Edgewise Therapeutics, a biopharmaceutical company developing small molecule therapies for musculoskeletal diseases, today announced the appointment of Abby H. Bronson to the newly created position of Vice President, Patient Advocacy and External Innovation. Ms. Bronson will be responsible for leading patient advocacy and building key external relationships with the muscular dystrophy community with the goal of bringing patient insights into drug development. This comes at an important time as Edgewise prepares EDG-5506, the companys lead product candidate, for clinical development for Duchenne and Becker muscular dystrophy (DMD and BMD). Ms. Bronson brings a wealth of experience in the rare disease space, most recently serving as Senior Vice President of Research Strategy at Parent Project Muscular Dystrophy (PPMD), the largest patient centered advocacy organization devoted to finding a cure for Duchenne.

We are pleased to have Abby join our company as we advance our muscular dystrophy program and prepare EDG-5506 for clinical development, said Kevin Koch, Ph.D., President and Chief Executive Officer, Edgewise Therapeutics. Having a strong patient advocacy perspective and voice in the DMD community is integral to executing against our vision of creating novel drugs that will transform the lives of patients living with severe musculoskeletal diseases. Abby brings an extensive patient advocacy background and we are fortunate to have someone with her knowledge and passion for the patient community join our team.

Im excited to join the dedicated team at Edgewise and to support the advancement of EDG-5506, a potentially transformative product candidate in DMD, through meaningful engagement with the patient and scientific communities, said Ms. Bronson.

Ms. Bronson is a leader within the rare disease space and over her career has managed critical alliances and partnerships with academia, biopharmaceutical companies, National Institutes of Health (NIH)/federal programs, patient groups and other stakeholders. Most recently she worked at PPMD as Senior Vice President of Research Strategy where she led the Research Portfolio and built strong relationships with Duchenne academic and clinical researchers, industry and regulators to help incorporate the patient voice and improve drug development success in DMD. Prior to this, Ms. Bronson was at the NIHs Center for Advancing Translational Sciences, Division of Clinical Innovation where she was Director of Operations for the Clinical and Translational Science Awards Program. Additionally, Ms. Bronson held positions at Children's National Medical Center, where she managed the global development and execution of key translational and drug development initiatives in select rare diseases, focusing on Duchenne at the Research Center for Genetic Medicine; MedImmune (acquired by Astra-Zeneca), where she led marketing initiatives for Synagis (palivizumab) for RSV disease; Medtronic where she managed global product marketing for select medical devices; and Ciba-Geneva Pharmaceuticals (acquired by Novartis) where she was responsible for managing relationships with major managed care organizations and led sales and marketing initiatives for their cardiovascular franchise. Ms. Bronson received her M.B.A from The Wharton School, University of Pennsylvania and B.A. degree from the University of Vermont.

About Muscular Dystrophy

Muscular dystrophies are a group of genetic disorders associated with defects in the critical muscle-associated structural protein dystrophin or the sarcomere complex and are characterized by progressive muscle degeneration and weakness. In individuals with neuromuscular conditions such as Duchenne muscular dystrophy, muscle contractions lead to continued rounds of muscle breakdown that the body struggles to repair. Eventually, as patients age, fibrosis and fatty tissue accumulate in the muscle portending a steep decline in physical function that ends with mortality. There remains an unmet need for treatments that reduce muscle breakdown in patients with neuromuscular conditions. Arresting this amplified muscle response will have a dramatic effect on disease progression.

About Edgewise Therapeutics

Edgewise Therapeutics, founded in 2017 by Alan Russell, Ph.D., Peter Thompson, M.D. (Orbimed Advisors) and Badreddin Edris, Ph.D., (Springworks Inc.), is pioneering the development of first-in-class medicines for the treatment of high morbidity musculoskeletal diseases. Skeletal muscle is the largest organ system in the human body, regulating both force production to enable muscle contraction, locomotion, and postural maintenance and the metabolism of glucose, fatty and amino acids. By modulating these processes in skeletal muscle, we create therapeutic agents that will reduce muscle damage, normalize muscle function, decrease mortality and profoundly benefit our patients quality of life. To learn more, go to: http://www.edgewisetx.com

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Edgewise Therapeutics Appoints Abby H. Bronson, M.B.A., as Vice President, Patient Advocacy and External Innovation - Business Wire

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Toxoplasma infection affects about 2 billion people globally

Parasitologist L. David Sibley, PhD (standing), the Alan A. and Edith L. Wolff Distinguished Professor of Molecular Microbiology at Washington University School of Medicine in St. Louis, talks with postdoctoral researcher Alex Rozenberg, PhD, (left) and staff scientist Joshua Radke, MD. The three are part of an international effort led by Sibley to find drugs to cure toxoplasmosis, a parasitic disease characterized by vision problems and brain complications.

L. David Sibley, PhD, the Alan A. and Edith L. Wolff Distinguished Professor of Molecular Microbiology at Washington University School of Medicine in St. Louis, has spent decades unraveling the secrets of Toxoplasma gondii, a parasite spread by cats and contaminated water and food. People infected with Toxoplasma can generally control the infection, but the parasite remains in their bodies for life and can reactivate to cause toxoplasmosis, a disease characterized by vision problems and life-threatening complications in the brain.

Sibleys discoveries have put him at the forefront of the field of parasite biology. A few years ago, he was busy fielding interview requests from journalists about his latest high-profile paper when he opened an email from a woman in Heidelberg, Germany.

I would like to ask you, wrote the woman, after explaining that her husband was dying of toxoplasmosis, how far (near?) is the possibility of human therapy based on your work?

To Sibley, the email was a wake-up call.

We always say that we do basic science so that one day there might be an improvement in human health, but we dont always push hard enough to convert our discoveries into benefits for patients, Sibley said. After thinking hard about this issue, my colleagues and I came up with the idea of trying to find chemical compounds that eliminate the chronic stages of the parasite, rather than just control it, like current drug therapies do. We know a lot about the biology of this parasite. My lab has spent 30 years figuring out all the tricks the parasite uses to block the immune system. We have developed sophisticated genetic tools and animal models to monitor infection. All this has led to a pile of high-profile papers, and recognition, but has not really had an impact on people who suffer from this infection. I thought, Why not see if we can identify small molecules that might lead to a curative drug?

That plaintive email eventually led Sibley and colleagues at the California Institute for Biomedical Research (Calibr) in La Jolla, Calif.; the Broad Institute in Cambridge, Mass.; and the International Centre for Genetic Engineering and Biotechnology in New Delhi, India to launch an effort to identify chemical compounds that eliminate the chronic stages of Toxoplasma and have the potential to be developed into drugs to eradicate the infection. As principal investigator, Sibley has received a $5.5 million grant from the National Institute of Allergy and Infectious Diseases of the National Institutes of Health (NIH) to support the research.

Toxoplasma is a parasite that naturally cycles between mice and cats. An infected cat excretes millions of the parasite in its feces in a form known as oocysts, contaminating the soil and water. A mouse gets infected by eating food such as fruit or seeds contaminated with oocysts, and a cat eats the mouse, completing the cycle.

Humans and other animals are accidental participants in this process. Herbivorous animals like cows and sheep can become infected as they graze. People become infected by eating undercooked meat from such animals or unwashed vegetables, or by drinking contaminated water. Some people become infected by failing to wash their hands after cleaning cats litter boxes. Once inside a persons digestive tract, the parasite emerges from the cyst, burrows through the intestinal wall and spreads to the muscle, heart, brain and eyes. There, it develops into a cyst form and remains for the rest of the persons life.

About a quarter of the worlds population is thought to be infected with Toxoplasma. Most people do not have symptoms because a healthy immune system keeps the parasite in check. In people with compromised immune systems, though, the parasites do not stay in their cysts and instead begin to multiply, causing debilitating, sometimes fatal, damage to the brain, eyes and other organs. Women who become infected during pregnancy may pass the infection to their fetuses, resulting in severe birth defects.

Drugs for toxoplasmosis only target the parasite in the active phase, leaving cysts untouched. Since parasites may emerge from the cysts at unpredictable times, people must continue taking the drugs for prolonged periods, sometimes more than a year. Even so, the risk of relapse is high. Supplementing current therapies with a drug that eliminates the cysts not only would speed up treatment, it would cure the infection.

Nobodys ever really looked for drugs that target the latent, cyst phase, Sibley said. You cant just take drugs that work against other microbial infections and repurpose them. Thats been tried and it doesnt work very well. Its hard to kill the cyst form. Thats why they form cysts: to protect themselves when they are in an inhospitable environment. Were going to have to really dig into the biology and thats difficult and takes time. Since the potential monetary payoff will likely be small, big pharma just isnt interested. If potential drugs are going to be found, they will have to be started by academic labs.

The research project is already underway. A group led by Stuart Schreiber, PhD, a chemical biologist at the Broad Institute, screened some 80,000 small molecules for their ability to inhibit parasite growth and identified several promising leads. A group of structural biologists at the International Centre for Genetic Engineering and Biotechnology led by Amit Sharma, PhD, is analyzing how the initial leads interact with their target enzyme. A detailed understanding of the molecular structure will inform efforts to optimize the compounds. Medicinal chemist Arnab Kumar Chatterjee, PhD, leads a group at Calibr that is creating new molecules based on the promising leads but with improved potency, safety, bioavailability and other features. And Sibleys lab at the School of Medicine is responsible for the biological testing, making sure the team stays focused on compounds that actually have the capacity to treat the cyst stage.

The compounds weve started working on may not ultimately lead to a drug that works, Sibley said. There are no guarantees in this kind of work. But I think what we can do is establish a path forward. We can identify appropriate targets, establish the potency, and define the safety profile that youd need for an effective clinical candidate. Then, maybe more people will pick up on our leads and do the very difficult work that is necessary to get drug candidates evaluated in humans and get one of those candidates approved as a medicine, so people dont have to suffer and die from this devastating illness.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Search for cure for common parasitic infection focus of $5.5 million NIH grant - Washington University School of Medicine in St. Louis

LOS ANGELES, United States: QY Research has recently published a report, titled Global CRISPR And CRISPR-Associated Genes Market Size, Status and Forecast 2020-2026. The research report gives the potential headway openings that prevails in the global market. The report is amalgamated depending on research procured from primary and secondary information. The global CRISPR And CRISPR-Associated Genes market is relied upon to develop generously and succeed in volume and value during the predicted time period. Moreover, the report gives nitty gritty data on different manufacturers, region, and products which are important to totally understanding the market.

Key Companies/Manufacturers operating in the global CRISPR And CRISPR-Associated Genes market include: Thermo Fisher Scientific, Editas Medicine, Caribou Biosciences, CRISPR therapeutics, Intellia therapeutics, Inc., Cellectis, Horizon Discovery Plc, Sigma Aldrich, Precision Biosciences, Genscript, Sangamo Biosciences Inc., Lonza Group Limited, Integrated DNA Technologies, New England Biolabs, Origene Technologies CRISPR And CRISPR-Associated Genes

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CRISPR is a type of gene-editing technology that lets scientists more rapidly and accurately cut and paste genes into DNA. Market Analysis and Insights: Global CRISPR And CRISPR-Associated Genes Market The global CRISPR And CRISPR-Associated Genes market size is projected to reach US$ XX million by 2026, from US$ XX million in 2020, at a CAGR of XX%% during 2021-2026. Global CRISPR And CRISPR-Associated Genes Scope and Market Size CRISPR And CRISPR-Associated Genes market is segmented 7, and 4. Players, stakeholders, and other participants in the global CRISPR And CRISPR-Associated Genes market will be able to gain the upper hand as they use the report as a powerful resource. The segmental analysis focuses on revenue and forecast 7 and 4 in terms of revenue and forecast for the period 2015-2026. The following players are covered in this report:Thermo Fisher ScientificEditas MedicineCaribou BiosciencesCRISPR therapeuticsIntellia therapeutics, Inc.CellectisHorizon Discovery PlcSigma AldrichPrecision BiosciencesGenscriptSangamo Biosciences Inc.Lonza Group LimitedIntegrated DNA TechnologiesNew England BiolabsOrigene Technologies CRISPR And CRISPR-Associated Genes

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Biotechnology CompaniesPharmaceutical CompaniesAcademic InstitutesResearch and Development Institutes

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Key companies operating in the global CRISPR And CRISPR-Associated Genes market include Thermo Fisher Scientific, Editas Medicine, Caribou Biosciences, CRISPR therapeutics, Intellia therapeutics, Inc., Cellectis, Horizon Discovery Plc, Sigma Aldrich, Precision Biosciences, Genscript, Sangamo Biosciences Inc., Lonza Group Limited, Integrated DNA Technologies, New England Biolabs, Origene Technologies CRISPR And CRISPR-Associated Genes

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TOC

1 Report Overview1.1 Study Scope1.2 Key Market Segments1.3 Players Covered: Ranking by CRISPR And CRISPR-Associated Genes Revenue1.4 Market 71.4.1 Global CRISPR And CRISPR-Associated Genes Market Size Growth Rate 7: 2020 VS 20261.4.2 Genome Editing1.4.3 Genetic Engineering1.4.4 GRNA Database/Gene Librar1.4.5 CRISPR Plasmid1.4.6 Human Stem Cells1.4.7 Genetically Modified Organisms/Crops1.4.8 Cell Line Engineering1.5 Market by Application1.5.1 Global CRISPR And CRISPR-Associated Genes Market Share 4: 2020 VS 20261.5.2 Biotechnology Companies1.5.3 Pharmaceutical Companies1.5.4 Academic Institutes1.5.5 Research and Development Institutes1.6 Study Objectives1.7 Years Considered 2 Global Growth Trends2.1 Global CRISPR And CRISPR-Associated Genes Market Perspective (2015-2026)2.2 Global CRISPR And CRISPR-Associated Genes Growth Trends by Regions2.2.1 CRISPR And CRISPR-Associated Genes Market Size by Regions: 2015 VS 2020 VS 20262.2.2 CRISPR And CRISPR-Associated Genes Historic Market Share by Regions (2015-2020)2.2.3 CRISPR And CRISPR-Associated Genes Forecasted Market Size by Regions (2021-2026)2.3 Industry Trends and Growth Strategy2.3.1 Market Top Trends2.3.2 Market Drivers2.3.3 Market Challenges2.3.4 Porters Five Forces Analysis2.3.5 CRISPR And CRISPR-Associated Genes Market Growth Strategy2.3.6 Primary Interviews with Key CRISPR And CRISPR-Associated Genes Players (Opinion Leaders) 3 Competition Landscape by Key Players3.1 Global Top CRISPR And CRISPR-Associated Genes Players by Market Size3.1.1 Global Top CRISPR And CRISPR-Associated Genes Players by Revenue (2015-2020)3.1.2 Global CRISPR And CRISPR-Associated Genes Revenue Market Share by Players (2015-2020)3.1.3 Global CRISPR And CRISPR-Associated Genes Market Share by Company Type (Tier 1, Tier 2 and Tier 3)3.2 Global CRISPR And CRISPR-Associated Genes Market Concentration Ratio3.2.1 Global CRISPR And CRISPR-Associated Genes Market Concentration Ratio (CR5 and HHI)3.2.2 Global Top 10 and Top 5 Companies by CRISPR And CRISPR-Associated Genes Revenue in 20193.3 CRISPR And CRISPR-Associated Genes Key Players Head office and Area Served3.4 Key Players CRISPR And CRISPR-Associated Genes Product Solution and Service3.5 Date of Enter into CRISPR And CRISPR-Associated Genes Market3.6 Mergers & Acquisitions, Expansion Plans 4 Market Size 7 (2015-2026)4.1 Global CRISPR And CRISPR-Associated Genes Historic Market Size 7 (2015-2020)4.2 Global CRISPR And CRISPR-Associated Genes Forecasted Market Size 7 (2021-2026) 5 Market Size 4 (2015-2026)5.1 Global CRISPR And CRISPR-Associated Genes Market Size 4 (2015-2020)5.2 Global CRISPR And CRISPR-Associated Genes Forecasted Market Size 4 (2021-2026) 6 North America6.1 North America CRISPR And CRISPR-Associated Genes Market Size (2015-2020)6.2 CRISPR And CRISPR-Associated Genes Key Players in North America (2019-2020)6.3 North America CRISPR And CRISPR-Associated Genes Market Size 7 (2015-2020)6.4 North America CRISPR And CRISPR-Associated Genes Market Size 4 (2015-2020) 7 Europe7.1 Europe CRISPR And CRISPR-Associated Genes Market Size (2015-2020)7.2 CRISPR And CRISPR-Associated Genes Key Players in Europe (2019-2020)7.3 Europe CRISPR And CRISPR-Associated Genes Market Size 7 (2015-2020)7.4 Europe CRISPR And CRISPR-Associated Genes Market Size 4 (2015-2020) 8 China8.1 China CRISPR And CRISPR-Associated Genes Market Size (2015-2020)8.2 CRISPR And CRISPR-Associated Genes Key Players in China (2019-2020)8.3 China CRISPR And CRISPR-Associated Genes Market Size 7 (2015-2020)8.4 China CRISPR And CRISPR-Associated Genes Market Size 4 (2015-2020) 9 Japan9.1 Japan CRISPR And CRISPR-Associated Genes Market Size (2015-2020)9.2 CRISPR And CRISPR-Associated Genes Key Players in Japan (2019-2020)9.3 Japan CRISPR And CRISPR-Associated Genes Market Size 7 (2015-2020)9.4 Japan CRISPR And CRISPR-Associated Genes Market Size 4 (2015-2020) 10 Southeast Asia10.1 Southeast Asia CRISPR And CRISPR-Associated Genes Market Size (2015-2020)10.2 CRISPR And CRISPR-Associated Genes Key Players in Southeast Asia (2019-2020)10.3 Southeast Asia CRISPR And CRISPR-Associated Genes Market Size by Type (2015-2020)10.4 Southeast Asia CRISPR And CRISPR-Associated Genes Market Size by Application (2015-2020) 11 India11.1 India CRISPR And CRISPR-Associated Genes Market Size (2015-2020)11.2 CRISPR And CRISPR-Associated Genes Key Players in India (2019-2020)11.3 India CRISPR And CRISPR-Associated Genes Market Size by Type (2015-2020)11.4 India CRISPR And CRISPR-Associated Genes Market Size by Application (2015-2020) 12 Central & South America12.1 Central & South America CRISPR And CRISPR-Associated Genes Market Size (2015-2020)12.2 CRISPR And CRISPR-Associated Genes Key Players in Central & South America (2019-2020)12.3 Central & South America CRISPR And CRISPR-Associated Genes Market Size by Type (2015-2020)12.4 Central & South America CRISPR And CRISPR-Associated Genes Market Size by Application (2015-2020) 13 Key Players Profiles13.1 Thermo Fisher Scientific13.1.1 Thermo Fisher Scientific Company Details13.1.2 Thermo Fisher Scientific Business Overview13.1.3 Thermo Fisher Scientific CRISPR And CRISPR-Associated Genes Introduction13.1.4 Thermo Fisher Scientific Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020))13.1.5 Thermo Fisher Scientific Recent Development13.2 Editas Medicine13.2.1 Editas Medicine Company Details13.2.2 Editas Medicine Business Overview13.2.3 Editas Medicine CRISPR And CRISPR-Associated Genes Introduction13.2.4 Editas Medicine Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.2.5 Editas Medicine Recent Development13.3 Caribou Biosciences13.3.1 Caribou Biosciences Company Details13.3.2 Caribou Biosciences Business Overview13.3.3 Caribou Biosciences CRISPR And CRISPR-Associated Genes Introduction13.3.4 Caribou Biosciences Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.3.5 Caribou Biosciences Recent Development13.4 CRISPR therapeutics13.4.1 CRISPR therapeutics Company Details13.4.2 CRISPR therapeutics Business Overview13.4.3 CRISPR therapeutics CRISPR And CRISPR-Associated Genes Introduction13.4.4 CRISPR therapeutics Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.4.5 CRISPR therapeutics Recent Development13.5 Intellia therapeutics, Inc.13.5.1 Intellia therapeutics, Inc. Company Details13.5.2 Intellia therapeutics, Inc. Business Overview13.5.3 Intellia therapeutics, Inc. CRISPR And CRISPR-Associated Genes Introduction13.5.4 Intellia therapeutics, Inc. Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.5.5 Intellia therapeutics, Inc. Recent Development13.6 Cellectis13.6.1 Cellectis Company Details13.6.2 Cellectis Business Overview13.6.3 Cellectis CRISPR And CRISPR-Associated Genes Introduction13.6.4 Cellectis Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.6.5 Cellectis Recent Development13.7 Horizon Discovery Plc13.7.1 Horizon Discovery Plc Company Details13.7.2 Horizon Discovery Plc Business Overview13.7.3 Horizon Discovery Plc CRISPR And CRISPR-Associated Genes Introduction13.7.4 Horizon Discovery Plc Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.7.5 Horizon Discovery Plc Recent Development13.8 Sigma Aldrich13.8.1 Sigma Aldrich Company Details13.8.2 Sigma Aldrich Business Overview13.8.3 Sigma Aldrich CRISPR And CRISPR-Associated Genes Introduction13.8.4 Sigma Aldrich Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.8.5 Sigma Aldrich Recent Development13.9 Precision Biosciences13.9.1 Precision Biosciences Company Details13.9.2 Precision Biosciences Business Overview13.9.3 Precision Biosciences CRISPR And CRISPR-Associated Genes Introduction13.9.4 Precision Biosciences Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.9.5 Precision Biosciences Recent Development13.10 Genscript13.10.1 Genscript Company Details13.10.2 Genscript Business Overview13.10.3 Genscript CRISPR And CRISPR-Associated Genes Introduction13.10.4 Genscript Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)13.10.5 Genscript Recent Development13.11 Sangamo Biosciences Inc.10.11.1 Sangamo Biosciences Inc. Company Details10.11.2 Sangamo Biosciences Inc. Business Overview10.11.3 Sangamo Biosciences Inc. CRISPR And CRISPR-Associated Genes Introduction10.11.4 Sangamo Biosciences Inc. Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)10.11.5 Sangamo Biosciences Inc. Recent Development13.12 Lonza Group Limited10.12.1 Lonza Group Limited Company Details10.12.2 Lonza Group Limited Business Overview10.12.3 Lonza Group Limited CRISPR And CRISPR-Associated Genes Introduction10.12.4 Lonza Group Limited Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)10.12.5 Lonza Group Limited Recent Development13.13 Integrated DNA Technologies10.13.1 Integrated DNA Technologies Company Details10.13.2 Integrated DNA Technologies Business Overview10.13.3 Integrated DNA Technologies CRISPR And CRISPR-Associated Genes Introduction10.13.4 Integrated DNA Technologies Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)10.13.5 Integrated DNA Technologies Recent Development13.14 New England Biolabs10.14.1 New England Biolabs Company Details10.14.2 New England Biolabs Business Overview10.14.3 New England Biolabs CRISPR And CRISPR-Associated Genes Introduction10.14.4 New England Biolabs Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)10.14.5 New England Biolabs Recent Development13.15 Origene Technologies10.15.1 Origene Technologies Company Details10.15.2 Origene Technologies Business Overview10.15.3 Origene Technologies CRISPR And CRISPR-Associated Genes Introduction10.15.4 Origene Technologies Revenue in CRISPR And CRISPR-Associated Genes Business (2015-2020)10.15.5 Origene Technologies Recent Development 14 Analysts Viewpoints/Conclusions 15 Appendix15.1 Research Methodology15.1.1 Methodology/Research Approach15.1.2 Data Source15.2 Disclaimer15.3 Author Details

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BEDFORD, Mass.--(BUSINESS WIRE)--Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company pioneering a new way to treat the underlying cause of severe genetic diseases, today announced the publication of data in the journal Nature Communications that support the companys proprietary approach to precisely upregulate protein expression using TANGO (Targeted Augmentation of Nuclear Gene Output) antisense oligonucleotides (ASOs).

Stoke was founded on the idea that we could use unique insights in RNA biology to do something that has never been done before, said Isabel Aznarez, Ph.D., Co-Founder and Vice President, Head of Biology of Stoke Therapeutics and the corresponding author on the paper. Rather than address genetic diseases by replacing, repairing or editing faulty genes, we set out to increase or stoke protein output from healthy genes. These data show that we can increase full-length, fully functional protein expression from a variety of healthy genes, which supports our hypothesis and may lead to a new way of treating severe genetic diseases.

To evaluate the approach broadly, Stoke selected four gene targets that vary in type and abundance of non-productive splicing events, gene size and protein function: PCCA (propionic acidemia); SYNGAP1 (autosomal dominant mental retardation 5); CD274 (autoimmune diseases, including uveitis); and SCN1A (Dravet syndrome). Stoke designed TANGO ASOs to target the non-productive splicing events in these genes and their activity was evaluated. Dose-dependent reductions of non-productive mRNA were observed to lead to increases in both productive mRNA and protein levels for each of the target genes.

More than 10,000 genetic diseases are caused by mutations in a single gene, however, current therapeutic approaches address as few as 5% of these diseases. In the experiments published today, a proprietary bioinformatics analysis of RNA sequencing datasets was used to identify a variety of non-productive alternative-splicing events that lead to mRNA degradation and limit protein production. Stoke found 7,757 unique genes that contained at least one non-productive event, of which 16% (1,246) were associated with causing a specific disease.

A link to the publication, Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression, can be found here: https://www.nature.com/articles/s41467-020-17093-9

Pre-mRNA Splicing and TANGO

Human cells naturally regulate protein production to maintain health. Pre-mRNA splicing, including alternative splicing, is an important mechanism used to regulate how much protein and which protein variant is produced. During splicing, introns are removed and exons are joined together to generate the mRNA template that carries the code to synthesize proteins. More than one third of alternative splicing events in mammals do not produce functional proteins and lead to mRNA degradation through nonsense-mediated mRNA decay (NMD). TANGO ASOs act at the pre-mRNA level and prevent non-productive alternative splicing so that the body produces more protein-coding mRNA and thus more protein. This approach is particularly applicable to diseases that are caused by insufficient protein production.

About Stoke Therapeutics

Stoke Therapeutics, Inc. (Nasdaq: STOK), is a biotechnology company pioneering a new way to treat the underlying causes of severe genetic diseases by precisely upregulating protein expression to restore target proteins to near normal levels. Stoke aims to develop the first precision medicine platform to target the underlying cause of a broad spectrum of genetic diseases in which the patient has one healthy copy of a gene and one mutated copy that fails to produce a protein essential to health. These diseases, in which loss of approximately 50% of normal protein expression causes disease, are called autosomal dominant haploinsufficiencies. The companys lead investigational new medicine is STK-001, a proprietary antisense oligonucleotide (ASO) that has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome, a severe and progressive genetic epilepsy. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit https://www.stoketherapeutics.com/ or follow the company on Twitter at @StokeTx.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about Companys proprietary approach to precisely upregulate protein expression using TANGO ASOs and the potential benefits thereof. These forward-looking statements may be accompanied by such words as aim, anticipate, believe, could, estimate, expect, forecast, goal, intend, may, might, plan, potential, possible, will, would, and other words and terms of similar meaning. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they do not fully materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: risks related to the direct and indirect impact of COVID-19; our ability to develop, obtain regulatory approval for and commercialize current and future product candidates; the timing and results of preclinical studies and clinical trials; the risk that positive results in a clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials; the risk that regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; risks related to the occurrence of adverse safety events; risks related to failure to protect and enforce our intellectual property, and other proprietary rights; risks related to failure to successfully execute or realize the anticipated benefits of our strategic and growth initiatives; risks relating to technology failures or breaches; our dependence on collaborators and other third parties for the development, regulatory approval, and commercialization of products and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; risks associated with current and potential future healthcare reforms; risks relating to attracting and retaining key personnel; failure to comply with legal and regulatory requirements; risks relating to access to capital and credit markets; environmental risks; risks relating to the use of social media for our business; and the other risks and uncertainties that are described in the Risk Factors section of our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this press release. We do not undertake any obligation to publicly update any forward-looking statements.

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Stoke Therapeutics Announces Publication of Data in the Journal Nature Communications That Support the Company's Proprietary Approach to Addressing...

Genetic tests, also called DNA tests, are used to identify changes in DNA sequences or chromosomal structures. Genetic testing also includes measuring the consequences of genetic alterations, such as RNA analysis as an output of gene expression, and biochemical analysis to measure specific protein outputs.

The Europe Genetic testing services market is expected to reach US$ 5,840.9 Mn in 2027 from US$ 2,521.6 Mn in 2019. The market is estimated to grow with a CAGR of 11.4% from 2020-2027.

Genetic testing is a type of medical test that identifies changes in chromosomes, genes, or proteins. The results of genetic tests can help identify or rule out suspicious genetic conditions or determine the likelihood of someone developing or inheriting a genetic disorder.

A medical device is any device intended to be used for medical purposes. Medical devices benefit patients by helping health care providers diagnose and treat patients and helping patients overcome sickness or disease, improving their quality of life.

The healthcare industryis undergoing rapid transformations since a few years now. Various technological improvementshave been witnessedin the segments including diagnosis and treatment options for chronic diseases. The increase in incidences of chronic illnesses and the increasing ageing population are the primary factors fuelling the growth of healthcare segment.

The Europe Genetic Testing Servicesmarketis growing along with the healthcare industry, but the market is likely to slow down its growth due to the shortage of skilled professionals, suggests the Business Market Insights report.

The Business Market Insights subscription helps clients understand theongoingmarket trends,identifyopportunities, and make informed decisions through the reports in the Subscription Platform. The Industry reports available in the subscriptionprovidean in-depth analysis on various market topics and enable clients to line up remunerative opportunities. The reportsprovidethe market size & forecast, drivers, challenges, trends, and more.

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France has well-developed policies and strategies in place for improving the prevention of hereditary cancers. Also, France is planning to develop a national plan for personalized medicine. Genomic Medicine France 2025, which was published in 2016, which appeals for healthcare and manufacturing firms to pilot genomic sequencing platforms. By 2020 the aim is to establish a network of centers able to process around 235,000 samples for whole genome sequencing.

These factorsare expectedto offer broad growth opportunities in the healthcare industry and this is expected to cause the demand forimmunochemistryassays in the market.

Business Market Insights reports focus upon clientobjectives, use standard research methodologies and exclusive analytical models, combined with robust business acumen, which providesprecise and insightful results.

Business Market Insights reports are usefulnot only forcorporate and academic professionalsbut also forconsulting, research firms,PEVCfirms, and professional services firms.

EUROPE GENETIC TESTING SERVICES MARKET SEGMENTATION

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Europe Genetic Testing Services Market is expected to reach US$ 5840.9 Million by 2027 with CAGR of 11.4%. - Owned