StemCell Therapy

Thats the beauty of these DNA vaccines, said Wolfgang W. Leitner, the chief of the innate immunity section at the National Institute of Allergy and Infectious Diseases. They are simple and fast in terms of development.

Nor are vaccine scientists concerned about the supposed secret sauce. In fact, its quite the opposite: They are skeptical precisely because the technology behind DNA vaccines has been around for decades and has been applied toward so many infectious diseases H.I.V., the flu, malaria yet none of the vaccines have made it to market.

They believe that this approach is capable of producing immunity. Already, DNA vaccines have been licensed for use in pigs, dogs and poultry. But the big if, according to Dr. Dennis M. Klinman, a vaccine scientist who worked at the Food and Drug Administration for 18 years, is whether one will ever be able to generate strong enough an immune response in humans.

Even though Ms. Wiley had read the packet on the science of it all, the next step felt like entering uncharted territory.

Shortly after the initial injection, a nurse handed Dr. Ervin a device resembling an electric toothbrush. He pressed the head which contains three tiny needles instead of bristles over the raised skin on her arm, where shed just had a shot. Then he zapped her.

It was not painful, but its unlike anything Ive ever experienced, Ms. Wiley said.

The carefully calibrated electrical pulses basically steer the DNA into the cells by briefly opening up pores in their membrane, according to David B. Weiner, the director of the vaccine and immunotherapy center at the Wistar Institute and an adviser to Inovio.

Although it may sound fantastical, the technology, called electroporation, dates to the 1980s, when a similar approach was first used to make transgenic plants, according to Dr. Leitner.

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Guaranteed Ingredient in Any Coronavirus Vaccine? Thousands of Volunteers - The New York Times

The coronavirus pandemic has pushed health to the forefront of many peoples minds. And while the best way to avoid COVID-19 is not to catch the virus in the first place, were starting to understand why some people become seriously ill with the disease while others have only mild or no symptoms.

Age and frailty are the most important risk factors for severe COVID-19, but data from our COVID Symptom Study app, used by nearly four million people, has shown that diet-related conditions, such as obesity, heart disease and type 2 diabetes, are significant risk factors for ending up in hospital with the disease.

In the UK, around one in three adults are obese and many more are overweight. In the US, around two in five adults and nearly one in five children are obese. From generalised government nutritional guidelines to Instagram-worthy fad diets, theres no end of advice on how to lose weight. Clearly, it isnt working.

This is a complex problem to unpick. Factors such as sex, ethnicity, socioeconomic status and availability of healthy food all play a part. But on an individual level, we still understand relatively little about how each person should eat to optimise their health and weight.

In search of answers, our research team at Kings College London together with our colleagues at Massachusetts General Hospital, Stanford University and health science company ZOE launched PREDICT, the largest ongoing nutritional study of its kind in the world. Our first results have now been published in Nature Medicine.

PREDICT-1, the first phase of the PREDICT research programme, involved more than 1,000 adults (including hundreds of pairs of twins) who were continuously monitored for two weeks to discover how they respond to different foods.

Participants had an initial set-up day in hospital for detailed blood measurements and testing of responses after eating carefully designed set meals. They then carried out the rest of the study at home, following a schedule of set meals and their own free choice of foods. We measured a wide range of markers of nutritional responses and health from blood glucose, fat, insulin and inflammation levels to exercise, sleep and gut bacteria (microbiome) diversity.

This kind of detailed, ongoing analysis was made possible through the use of wearable technologies. These included continuous blood glucose monitors and digital activity trackers, which meant we could keep track of our participants blood sugar and activity levels 24/7. Simple finger-prick blood tests also allowed us to measure their blood fat levels on a regular basis.

All these measurements added up to millions of datapoints, which needed to be analysed with sophisticated machine learning techniques (a type of artificial intelligence) in order to spot patterns and make predictions.

The first thing we noticed was the wide variation in individual insulin, blood sugar and blood fat responses to the same meals, even for identical twins. For example, one twin might have healthy responses to eating carbohydrates but not fat, while the other twin is the opposite. Straight away, this tells us that we are all unique and that there is no perfect diet or correct way to eat that will work for everyone.

The observation that genetics only plays a minor role in determining how we respond to food also tells us that simple genetic tests claiming to determine the right diet for your genes are ineffective and misleading. Curiously, identical twins only shared around a third of the same gut microbe species, which may help to explain some of the variation in nutritional responses and also points towards an opportunity to improve health and weight by manipulating the microbiome.

We also discovered that the timing of meals affects nutritional responses in a personalised way. The same meal at breakfast caused a different nutritional response in some people when eaten for lunch. But in other people there was no difference, busting the myth that there are correct mealtimes that will work for all.

Another surprise was finding that the composition of meals in terms of calories, fat, carbohydrates, proteins and fibre (macronutrients or macros) also had a highly individualised effect on nutritional responses. Some people handle carbs better than fat, for example, while others have the opposite response. So prescriptive diets based on fixed calorie counts or macronutrient ratios are too simplistic and will not work for everyone.

However, despite the wide variability between participants, each persons own responses to identical meals eaten at the same times on different days were remarkably consistent. This makes it possible to predict how someone might respond to any food based on knowledge of their underlying metabolism.

Intriguingly, we found that the levels of inflammatory molecules in the blood varied by up to tenfold, even in seemingly healthy people, and that a rise in these inflammation markers was linked to having unhealthy responses to fat.

We use the term dietary inflammation to refer to these unhealthy metabolic effects that are triggered after eating. Repeatedly experiencing dietary inflammation brought on by excessive blood sugar and fat responses is linked with an increased risk of conditions such as heart disease, type 2 diabetes, non-alcoholic fatty liver disease and obesity.

On a more positive note, our findings suggest that it might be possible to improve weight management and long-term health by eating in a more personalised way designed to avoid triggering unhealthy inflammatory responses after meals.

When it comes to weight, weve traditionally put a huge emphasis on factors we have no control over, especially genetics. The fact is, while genetics plays a role, many more important factors affect how our metabolism, weight and health. Its time to move away from overly generalised guidelines, fad diets and one-size-fits-all plans and develop more personalised, scientific approaches to nutrition that understand and work together with our bodies, not against them.

For more on personalised nutrition, download and listen to our podcast, Medicine made for you, a series by The Anthill.

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Why one-size-fits-all diets don't work new study - The Conversation UK

Boston-based hearing loss gene therapy biotech Akouos is gunning for a $100 million IPO amid a near invulnerable market for public-seeking biotechs.

This comes just three months after it grabbed a $105 million series B funding round from a host of big names including the likes of 5AM Ventures, New Enterprise Associates, Novartis Venture Fund and Partners Innovation Fund as well as new participants Cowen Healthcare Investments, Polaris and Pivotal bioVenture Partners, which led the round.

Back in March, and in conjunction with its raise, the biotech also appointed Vertex and Biogen research veteran Vicki Sato, Ph.D., and Pivotal bioVenture Partners Managing Partner Heather Preston, M.D., to its board.

Brand Revitalization with RP Scherer Softgel Solutions for Analgesics

A global consumer healthcare company needed to revitalize one of their leading OTC brands competing in the analgesic category. In a series of innovation sessions, Catalent RP Scherer softgel capsules were quickly identified as the ideal solution to drive incremental growth. With a highly-successful launch and halo growth for the entire brand, the new line extension increased the brands overall market share.

Now, in the middle of a pandemic, it wants to follow many other biotechs and gun for an IPO worth $100 million to help push its lead program AK-OTOF into the clinic.

The biotech is targeting monogenic forms of sensorineural hearing loss. These forms arise when changes to single genes cause sensory cells or nerve fibers in the inner ear to malfunction.

In its SEC-1 filing, the biotech said that, after speaking with the FDA: We are designing our phase 1/2 trial to include auditory brainstem response (ABR) as an efficacy endpoint. We believe that this will enable us to quickly determine a clinical response and potentially result in rapid advancement towards a pivotal trial.

We plan to submit an investigational new drug application for AK-OTOF for OTOF-mediated hearing loss to the FDA in 2021, and we expect to report preliminary clinical data in 2022.

As it sees it, there is a great hope for gene therapy for hearing loss, but the issue is delivery: We believe genetic medicine development for hearing disorders has been hindered by the unique anatomical delivery challenges of the inner ear, it said in its filing.

To get around this, the biotech has combined a proprietary vector library of syntheticadeno-associated viruses (AAVs) that recreates the evolutionary lineage of current naturally occurring viruses, known as ancestral AAV, or AAVAnc, and a new, what it says is a minimally invasive delivery approach, that allows it to use AAV-enabled multimodal capabilities, including viral delivery, to the target cell population where the full-length transgene is split into two vectors, known as a dual vector method.

It plans to file on the Nasdaq under the ticker "AKUS."

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3 months after its B round, Akouos files an IPO to the tune of $100M - FierceBiotech

WALTHAM, Mass., June 08, 2020 (GLOBE NEWSWIRE) -- Affinia Therapeutics, an innovative gene therapy company with a platform for rationally designed adeno-associated virus (AAV) vectors and gene therapies, announced today the appointment of Elliott Sigal, M.D., Ph.D., to the companys Board of Directors. Dr. Sigal has more than 25 years of leadership experience in the biopharmaceutical industry and is the former Chief Scientific Officer and President of R&D for Bristol Myers Squibb.

As a trailblazer in the biopharmaceutical industry, Dr. Sigal has demonstrated a track record of bringing transformative medicines to patients, said Rick Modi, Chief Executive Officer at Affinia Therapeutics. We look forward to the counsel he will provide to advance our platform and investigational product candidates toward the clinic and the patients who need them most.

Dr. Sigal is a former member of the Board of Directors of Spark Therapeutics. During his tenure from 2014 to 2019, the companys lead product, LUXTURNA was approved as the first AAV gene therapy in the United States. The company was acquired by Roche in 2019.

Prior to Spark Therapeutics, Dr. Sigal was an Executive Vice President and Director of Bristol Myers Squibb. While at BMS, he led the team that established BMS at the forefront of immuno-oncology which is revolutionizing the treatment of cancer and brought fourteen new medicines to market for patients with devastating diseases in areas including oncology, hematology, cardiovascular disease, hepatitis, rheumatoid arthritis and neuropsychiatry. Dr. Sigal was instrumental in increasing R&D productivity and developing the companys strategy in biologics. In 2012, he was named the best R&D chief in the pharmaceutical industry by Scrip Intelligence.

Affinia Therapeutics is setting a new standard in gene therapy, said Dr. Elliott Sigal. I am pleased to join the companys board at such an exciting time as they pioneer and design vectors and genetic medicines to transform the applicability of gene therapies for patients in need.

Dr. Sigal received his medical degree from the University of Chicago in 1981 and trained in Internal Medicine and Pulmonary Medicine at the University of California, San Francisco (UCSF). He also holds a Bachelor of Science, Master of Science and Ph.D. in Industrial Engineering from Purdue University. Dr. Sigal currently serves as a senior advisor to the healthcare team of New Enterprise Associates and consults for select biotechnology companies including Amgen. He is co-chair of the Scientific Advisory Board of Amgen and is a member of the Scientific Steering Committee of the Sean Parker Institute for Cancer Immunotherapy. He is also a member of the Board of Directors for the biotechnology companies Adaptimmune and Surface Oncology. Dr. Sigal joined BMS in 1997 and held roles in both discovery and development before ascending to Chief Scientific Officer and President of R&D. Positions prior to BMS included a faculty appointment at UCSF, senior executive roles at Syntex/Roche and CEO of the genomics firm, Mercator Genetics.

Dr. Sigal joins Affinia Therapeutics board which includes Dave Grayzel, M.D., Partner, Atlas Venture; Ed Mathers, General Partner, New Enterprise Associates; Luk Vandenberghe, Ph.D., Associate Professor at Mass. Eye and Ear and Harvard Medical School; Rick Modi, Chief Executive Officer, Affinia Therapeutics; Robert Weisskoff, Ph.D., Partner, F-Prime Capital; and Sean Nolan, Chairman of the Board of Directors at Affinia Therapeutics.

About Affinia Therapeutics

At Affinia Therapeutics, our purpose is to develop gene therapies that can have a transformative impact on people affected by devastating genetic diseases. Our proprietary platform enables us to methodically engineer novel AAV vectors and gene therapies that have remarkable tissue targeting and other properties. We are building world-class capabilities to discover, develop, manufacture and commercialize gene therapy products with an initial focus on muscle and central nervous system (CNS) diseases with significant unmet need. http://www.affiniatx.com.

Affinia Therapeutics Contacts

Investors: investors@affiniatx.com

Media: media@affiniatx.com

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Affinia Therapeutics Announces Appointment of Elliott Sigal, MD, Ph.D. to the Company's Board of Directors - GlobeNewswire

DetailsCategory: DNA RNA and CellsPublished on Monday, 08 June 2020 18:17Hits: 336

- In post-treatment muscle biopsies, clinical trial participants in the high-dose cohort showed a dose-dependent increase in transduction and expression when compared with the low-dose cohort, with a mean of 72% beta-sarcoglycan (beta-SG) positive fibers, as measured by immunohistochemistry (IHC), substantially exceeding the pre-defined 50% measure for success ---- A mean signal intensity of 73% in the high-dose group was observed compared to normal control ---- A mean beta-sarcoglycan expression of 62% as measured by Western blot was observed in the high-dose cohort compared to normal control ---- An 89% mean reduction of creatine kinase (CK) from baseline was observed in the high-dose cohort ---- Continued functional improvement was observed in the low-dose cohort at one year --

CAMBRIDGE, MA, USA I June 08, 2020 I Sarepta Therapeutics, Inc.(NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive results from a study of SRP-9003, its investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). Results included safety and expression results from three clinical trial participants in the high-dose cohort measured at 60 days, and one-year functional data from three clinical trial participants in the low-dose cohort. SRP-9003 is in development for the treatment of LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4), a devastating monogenic neuromuscular disease caused by a lack of beta-sarcoglycan (beta-SG) proteins. SRP-9003 is a gene construct that transduces skeletal and cardiac muscle, delivering a gene that codes for the full-length beta-sarcoglycan protein, the absence of which is the sole cause of progressive degeneration and a shortened lifespan characterized by the disease.

We were very encouraged by the previously reported results from our first cohort of patients treated with a lower dose of SRP-9003, including impressive expression, good tolerability, and positive functional signals, which continue impressively at one year. We are excited to have been able to achieve even more impressive expression and other biomarkers in our higher-dose cohort for SRP-9003, along with good tolerability. The SRP-9003 gene construct, vector and promoter were designed with the goal of robustly delivering to skeletal and cardiac muscles a gene coding for the missing beta-sarcoglycan protein that causes LGMD2E. These data support the conclusion that the therapy is achieving its intended purpose, driving robust expression in the muscles where it is needed, said Doug Ingram, President and CEO, Sarepta. SRP-9003 employs the same vector, AAVrh74, and same promoter, MHCK7, as SRP-9001, our therapy in development to treat Duchenne muscular dystrophy. And Cohort 2 received a similar dose as our ongoing SRP-9001 studies for Duchenne. The safety and efficacy results with these two doses of SRP-9003 provide us with additional experience and confidence with the rh74 vector and the MHCK7 promoter as we select the dose for the pivotal trial of SRP-9003 and work to quickly develop this therapy for patients who currently have no treatment options.

The SRP-9003 study has two cohorts, each studying a different dose-per-kilogram based on the weight of the patient. Three participants in the low-dose cohort (Cohort 1) were treated with a one-time infusion of SRP-9003 dosed at 5x1013vg/kg and an additional three participants in the high-dose cohort (Cohort 2) received a one-time infusion dosed at 2x1014vg/kg. The six participants were between the ages of 4 and 13. Post-treatment biopsies were taken at 60 days. Sarepta previously shared data from Cohort 1 in 2019, including positive and robust expression and biomarker data and positive 9-month functional results.

Preliminary results from Cohort 2 (n=3) are as follows:

In Cohort 1 (low dose), at one year all three participants continued to show improvements from baseline across all functional measures, including the North Star Assessment for Limb-Girdle Muscular Dystrophies, time-to-rise, four-stair climb, 100-meter walk test and 10-meter walk test. These results are distinctly different from what an age-matched, natural history group would predict. There have been no new drug-related safety signals observed since the 9-month update, and no decreases in platelet counts outside of the normal range or signs of complement activation were observed.

LGMD2E is a devastating neuromuscular disease that causes significant disability in the children we see and currently lacks treatment options beyond tailored physical therapy, said Jerry Mendell, M.D., principal investigator at the Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Childrens Hospital and lead investigator for the study. We are pleased that these data show robust expression, similar to what we observed in the micro-dystrophin program, for the protein that is missing in children with LGMD2E, and remain hopeful that this brings us one step closer to a therapy that can help improve both prognosis and quality of life.

About SRP-9003 and the studySRP-9003 uses the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat peripheral neuromuscular diseases. AAVrh74 has lower immunogenicity rates than reported with other human AAV vectors. The MHCK7 promoter has been chosen for its ability to robustly express in the heart, which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications.

This first-in-human study is evaluating a single intravenous infusion of SRP-9003 among children with LGMD2E between the ages of four and 15 years with significant symptoms of disease. Sarepta has exclusive rights to the LGMD2E gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Childrens Hospital.

About Limb-Girdle Muscular DystrophyLimb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begin in muscles around the hips and shoulders before progressing to muscles in the arms and legs.

Patients with limb-girdle muscular dystrophy Type 2E (LGMD2E) begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10. The disease, which is an autosomal recessive subtype of LGMD, progresses to loss of ambulation in the teen years and often leads to early mortality. There is currently no treatment or cure for LGMD2E.

Sarepta has five LGMD gene therapy programs in development, including subtypes for LGMD2E, LGMD2D, LGMD2C, LGMD2B and LGMD2L, and holds an option for a sixth program for LGMD2A.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.comor follow us onTwitter,LinkedIn,InstagramandFacebook.

SOURCE: Sarepta Therapeutics

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Sarepta Therapeutics Announces Positive Expression and Functional Data From the SRP-9003 Gene Therapy Trial to Treat Limb-Girdle Muscular Dystrophy...

CINCINNATI, June 08, 2020 (GLOBE NEWSWIRE) -- Aerpio Pharmaceuticals, Inc. (Aerpio) (Nasdaq: ARPO), a biopharmaceutical company focused on developing compounds that activate Tie2 to treat ocular diseases and diabetic complications, today announced that it is hosting a key opinion leader (KOL) call on a novel mechanism for the treatment of glaucoma on Friday, June 12, 2020 at 11:30am Eastern Time.

The call will feature presentations by Dr. Paul Kaufman M.D. (University of Wisconsin) and Dr. Janey Wiggs, M.D., Ph.D. (Massachusetts Eye and Ear Infirmary and Harvard Medical School), who will discuss the current glaucoma treatment landscape and unmet medical needs, as well as the role of the Tie2 receptor in maintaining intraocular pressure. Drs. Kaufman and Wiggs will be available to answer questions at the conclusion of the event.

Aerpio's management team will also discuss its pipeline candidate, razuprotafib (formerly AKB-9778), for treating patients with glaucoma. Razuprotafib is a small molecule inhibitor that restores Tie2 activation in Schlemms canal and lowers intraocular eye pressure (IOP) via decreasing resistance to outflow from the eye. Razuprotafib has been formulated as a once or twice-daily topical eye drop and is entering a Phase 2 clinical trial in Q3:20, with top line data expected in Q1:21.

Aerpio recently announced positive and statistically significant intraocular eye pressure (IOP) reduction in a Phase 1b trial of 43 glaucoma patients, when razuprotafib was added to prostaglandin treatment. This data set is summarized here.

Paul Kaufman, M.D. is the Ernst H. Brny Emeritus Professor of Ocular Pharmacology and past Chair of the Department of Ophthalmology & Visual Sciences at the University of Wisconsin School of Medicine and Public Health, in Madison, Wisconsin. He is a physician-scientist, specializing in glaucoma and studying the mechanisms of aqueous humor formation and drainage, and the age-related loss of near vision. Dr Kaufman is a past President and past Executive Vice President of the Association for Research in Vision and Ophthalmology (ARVO), past President of the International Society for Eye Research (ISER), and has served on the US National Advisory Eye Council and numerous foundation and corporate scientific advisory boards. He has had continuous research funding from the US National Eye Institute for 40 years and from numerous private foundations, has authored over 375 original scientific articles and 75 book chapters, co-edited several textbooks including the most recent editions of Adlers Physiology of the Eye, and received numerous honors and awards including the Friedenwald Award from ARVO and the Balazs Prize from ISER. He was Editor-in-Chief of Investigative Ophthalmology & Visual Science from 2008 through 2012. Dr. Kaufman also holds an honorary Doctor of Medicine degree from Uppsala University in Sweden, where he was a post-doctoral research fellow.

Janey L. Wiggs, M.D., Ph.D. is a physician-scientist at the Massachusetts Eye and Ear Infirmary and Harvard Medical School. She is currently the Paul Austin Chandler Professor of Ophthalmology and is the Vice Chair for Clinical Research in Ophthalmology at Harvard Medical School. She also directs the CLIA-certified genetic testing laboratory at the Massachusetts Eye and Ear Infirmary and is a co-director of the Ocular Genomics Institute and co-director of the Glaucoma Center of Excellence. Dr. Wiggs received her B.A. and Ph.D. degrees in biochemistry from the University of California at Berkeley and her M.D. degree from Harvard Medical School. She did post-doctoral training in molecular genetics under the direction of Dr. Ted Dryja. Dr. Wiggs completed the ophthalmology residency at the Massachusetts Eye and Ear Infirmary and received fellowship training in glaucoma and also in medical genetics and is certified by the both the American Board of Ophthalmology and the American Board of Medical Genetics. Dr. Wiggs research program is focused on the discovery and characterization of genetic factors that contribute to the blinding eye disease glaucoma and is funded by the National Eye Institute (NEI) as well as other nonprofit foundations. She is investigating the genetic etiologies of both early-onset and adult forms of glaucoma and is the PI of the NEIGHBORHOOD consortium for gene discovery in primary open angle glaucoma and is a founding member of the International Glaucoma Genetics Consortium (IGGC). She has also participated in research programs funded by the US-INDO joint working group (NEI) and the NEI eyeGENE consortium. Dr. Wiggs was the inaugural chair of the Genetics Group for ARVO and is an ARVO gold fellow. She currently serves on the editorial boards of IOVS, JAMA Ophthalmology, Molecular Vision, Journal of Glaucoma, and Annual Reviews in Vision Science. She is a member of the scientific advisory boards for the Glaucoma Research Foundation, Research to Prevent Blindness and the Glaucoma Foundation, and is a past member of the Advisory Council of the National Eye Institute. She has received the Heed Award, the Heed/Knapp Award, the Research to Prevent Blindness Scholar Award, the AAO Honor Award, the Lew Wasserman Merit Award, the Alcon Research Award, the David L. Epstein award from the ARVO Foundation and was a winner of the NEI Audacious Goal competition. She is an elected member of the Glaucoma Research Society, the American Ophthalmological Society, the Academia Ophthalmologica Internationalis and the National Academy of Medicine.

About RazuprotafibRazuprotafib binds to and inhibits vascular endothelial protein tyrosine phosphatase (VE-PTP), an important negative regulator of Tie2. Decreased Tie2 activity contributes to vascular instability in many diseases including diabetes and more recently has been shown to contribute to the development of increased IOP and glaucoma. Razuprotafib activates the Tie2 receptor irrespective of extracellular levels of its binding ligands, angiopoietin-1 (agonist) or angiopoietin-2 (antagonist) and may be the most efficient pharmacologic approach to maintain normal Tie2 activation. Aerpio is studying a topical ocular formulation of razuprotafib in open angle glaucoma and exploring the utility of subcutaneous razuprotafib for diabetic complications, including diabetic nephropathy.

About Aerpio PharmaceuticalsAerpio Pharmaceuticals, Inc. is a biopharmaceutical company focused on developing compounds that activate Tie2 to treat ocular diseases and diabetic complications. Recently published mouse and human genetic data implicate the Angpt/Tie2 pathway in maintenance of Schlemms canal, a critical component of the conventional outflow tract. The Companys lead compound, razuprotafib (formerly AKB-9778), a first-in-class small molecule inhibitor of vascular endothelial protein tyrosine phosphatase (VE-PTP), is being developed as a potential treatment for open angle glaucoma, and the Company intends to investigate the therapeutic potential of razuprotafib in other indications. The Company is also evaluating development options for ARP-1536, a humanized monoclonal antibody, for its therapeutic potential in the treatment of diabetic vascular complications including nephropathy and diabetic macular edema (DME). The Companys third asset is a bispecific antibody that binds both VEGF and VE-PTP which is designed to inhibit VEGF activation and activate Tie2. This bispecific antibody has the potential to be an improved treatment for wet age-related macular degeneration and DME via intravitreal injection. Finally, the Company has exclusively out-licensed AKB-4924 (now called GB004), a first-in-class small molecule inhibitor of hypoxia-inducible factor-1 (HIF). GB004 is being developed by AKB-4924s exclusive licensor, Gossamer Bio, Inc. (Nasdaq: GOSS). For more information, please visit http://www.aerpio.com.

Forward Looking StatementsThis press release contains forward-looking statements. Statements in this press release that are not purely historical are forward-looking statements. Such forward-looking statements include, among other things, the Companys product candidates, including razuprotafib, ARP-1536 and the bispecific antibody asset, the clinical development plan therefor and the therapeutic potential thereof, the Companys plans and expectations with respect to razuprotafib and the development therefor and therapeutic potential thereof in addressing COVID-19 and the intended benefits from the Companys collaboration with Gossamer Bio for GB004, including the continued development of GB004 and the milestone and royalty payments related to the collaboration. Actual results could differ from those projected in any forward-looking statements due to several risk factors. Such factors include, among others, the continued development of GB004 and maintaining and deriving the intended benefits of the Companys collaboration with Gossamer Bio; ability to continue to develop razuprotafib or other product candidates, including in indications related to COVID-19; the inherent uncertainties associated with the drug development process, including uncertainties in regulatory interactions, the design of planned or future clinical trials, commencing clinical trials and enrollment of patients in clinical trials; obtaining any necessary regulatory clearances in order to commence and conduct planned or future clinical trials; the impact of the ongoing COVID-19 pandemic on the Companys business operations, including research and development efforts and the ability of the Company to commence, conduct and complete its planned clinical activities; and competition in the industry in which the Company operates and overall market conditions; and the additional factors set forth in our Annual Report on Form 10-K for the year ended December 31, 2019, as updated by our subsequent Quarterly Reports on Form 10-Q and our other subsequent filings with the SEC.

These forward-looking statements are made as of the date of this press release, and the Company assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law. Investors should consult all the information set forth herein and should also refer to the risk factor disclosure set forth in the reports and other documents the Company files with the SEC available at http://www.sec.gov.

Investors & Media:Gina MarekVP Financegmarek@aerpio.comOrInvestors:Irina KofflerLifeSci Advisorsikoffler@lifesciadvisors.com

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Aerpio Hosting Key Opinion Leader Call on a Novel Mechanism for the Treatment of Glaucoma - GlobeNewswire

While most people who contract the new coronavirus develop a mild case of Covid-19, for some the disease is deadlyand researchers are exploring whether a person's DNA may play a role in determining the disease's severity.

How Dignity Health keeps patients connected to telegeneticsduring and beyond Covid-19

Researchers already have determined that a person's age and whether they have certain underlying health conditions can affect their risk of developing a severe case of Covid-19, the disease caused by the new coronavirus. But now, some research suggests a person's blood type may be another factor in whether they have a higher risk of developing a severe case of the disease.

For example, a preprint study published Tuesday that has not been peer-reviewed examined blood samples from 1,610 Covid-19 patients who developed severe cases of Covid-19, which the researchers classified as needing oxygen or a ventilator as part of their treatment. The researchers sequenced part of each those patients' genomes, and then performed the same analysis on samples from 2,205 blood donors who did not have Covid-19 and compared the results.

The researchers found that many of the patients who had severe cases of Covid-19 possessed the same variant on a gene that determines a person's blood type. Specifically, the researchers found that having blood type A was linked with a 50% increase in the likelihood a patient would develop a severe case of Covid-19.

According to the New York Times, a separate preprint study conducted by researchers in China that hasn't yet been peer-reviewed found similar results. The study found that, out of 2,173 Covid-19 patients with different blood types, blood type A was associated with a higher risk of death from Covid-19 when compared with other blood types. The study also found that people with blood type A appeared more likely to contract the new coronavirus, whereas those with blood type O appeared to be the least likely to contract the virus.

Andre Franke, a molecular geneticist at the University of Kiel in Germany, who led the first study said he and his colleagues also identified another locus on Chromosome 3 that appeared to be linked with Covid-19. However, the researchers noted that locus hosts six different genes, and they've yet to determine which of those genes influences how Covid-19 develops.

Despite the findings, Franke said researchers are still unsure exactly how a person's blood type plays a part in how Covid-19 affects them. "That is haunting me, quite honestly," he said.

Franke said the locus that hosts the blood-type gene also contains a portion of a person's DNA that controls a gene that makes a protein that generates robust immune responses, the Times reports. According to the Times, researchers and providers have found that the new coronavirus can trigger a so-called "cytokine storm" in some patients, which occurs when a patient's immune system overreacts to a pathogen and damages a patient's organs, and it's "theoretically possible that genetic variations influence that response."

For a separate study published last month in Cell, researchers looked into how the new coronavirus affects human cells and found that, within three days of infection, the virus activates genes in the cells that produce cytokine proteins, which are the proteins that can cause cytokine storm. At the same time, the virus blocks genes in the cells that produce interferons that could constrain the virus' replicationsomething most other viruses don't do, according to Benjamin tenOever of the Icahn School of Medicine at Mount Sinai, who co-authored the study. "It's something I have never seen in my 20 years of" studying viruses, he said.

tenOever explained that, without interferons, "there is nothing to stop the virus from replicating and festering in the lungs forever."

According to STAT News, the researchers' findings could help scientists identify treatments for Covid-19. For instance, Vineet Menachery from the University of Texas Medical Branch said providing high-risk patients with interferons could potentially "allow treated cells to fend off the virus better and limit its spread."

But more research on how genetic variants might affect Covid-19 are needed, according to Jonathan Sebat, a geneticist at the University of California-San Diego who was not involved in the studies. According to Sebat, previous studies attempting to identify variances in genetic loci that are significantly more common in sick people than healthy people have failed, meaning it's possible that the variants identified in the recent studies may not play as much as a role as in how Covid-19 develops as the new findings may imply (Zimmer, New York Times, 6/3; Begley, STAT News, 5/21; Mangin, Scientific American, 4/30).

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What's your blood type? The answer could affect your risk from Covid-19. - The Daily Briefing

SAN DIEGO--(BUSINESS WIRE)--Autobahn Therapeutics is launching to create the next generation of regenerative medicines to restore hope for people affected by CNS disorders. The company has completed a $76 million Series B fundraising co-led by ARCH Venture Partners and Cowen Healthcare Investments, with participation from BVF Partners L.P., Biogen, Bristol Myers Squibb, Pfizer Ventures, Invus, Section 32, Samsara BioCapital and Alexandria Venture Investments. Proceeds will be used to advance Autobahns lead program candidate, ABX-002, a thyroid hormone receptor beta agonist therapy for the treatment of multiple sclerosis (MS) and adrenomyeloneuropathy (AMN), a rare genetic disorder, and a portfolio of transformational CNS programs leveraging the companys brain-targeting chemistry platform.

Autobahn is harnessing the regenerative power of the human body to treat both rare and prevalent CNS disorders. We are coupling our deep knowledge of thyroid hormone biology and remyelination with our brain-targeting chemistry platform to restore the brain to a healthier state, said Kevin Finney, chairman and chief executive officer of Autobahn. We stand well-positioned to advance our pipeline with funding from the highest quality investors and pharmaceutical leaders who share our mission of improving life health for people affected by these conditions.

Innovating Treatments for CNS Disorders

Autobahns scientific approach is based on the well-established role that thyroid hormone plays in the production of myelin, the protective sheath that forms around nerves. The degeneration of myelin is associated with many CNS disorders, including MS. Autobahn is developing small molecule, thyroid hormone receptor beta agonists designed to stimulate remyelination and address the progressive nature of MS and other diseases that result from demyelination. The companys strategy leverages validated human biology to de-risk and accelerate its development programs, a brain-targeting chemistry platform to maximize exposure selectively in the brain, and biomarker-driven development to establish on-target activity and proof-of-mechanism early in development.

We believe Autobahn has the insights and expertise to turn this world-class research into important medicines for patients. The work pioneered by Dr. Tom Scanlan of Oregon Health & Science University served as a strong base on which to add a highly talented team of researchers and clinical innovators to create Autobahn, said Kristina Burow, managing director with ARCH Venture Partners. We are proud to be working alongside the company to harness the transformative potential of Autobahns remyelinating therapies.

Leadership Team with Proven Track Record

Autobahn has built a team of experts in thyromimetics, drug discovery and development, clinical operations, and corporate and business development:

Autobahn is developing a differentiated portfolio of CNS therapies, backed by validated science and led by proven scientific and business leaders, said Tim Anderson, managing director of Cowen Healthcare Investments. Investing in innovative life sciences companies is at the heart of Cowen Healthcare Investments, and we are excited to join the Autobahn team to help develop products that can fundamentally change the way people with CNS disorders are treated.

Expert Board of Directors

Autobahn has established a board of directors with significant experience in company formation and scientific innovation. Directors include:

Autobahn benefits from the winning combination of validated human biology supporting its therapeutic hypothesis, clever chemistry enabling tissue-selective CNS delivery of compounds and an exceptional team to drive it forward, added Dr. Cravatt. I am highly confident in our potential to create the next generation of regenerative medicines for the brain.

About Autobahn Therapeutics

Autobahn Therapeutics is focused on improving life health for people affected by CNS disorders. Autobahn is leveraging a deep understanding of validated human biology coupled with its brain-targeting chemistry platform to develop thyroid hormone receptor beta agonist therapies that harness the regenerative power of the human body. The companys pipeline is led by ABX-002 for the treatment of multiple sclerosis and adrenomyeloneuropathy (AMN), a rare genetic disorder. Autobahn Therapeutics is based in San Diego. For more information, visit http://www.autobahntx.com.

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Autobahn Therapeutics Launches with $76 Million Series B Financing to Develop Next Generation Therapies for CNS Disorders - Business Wire

Cancer is caused by mutations that lead to uncontrolled cell division. One of the most aggressive types of cancer is glioblastoma, a form of brain tumour with a very poor prognosis. Relatively little is known about how mutations in non-coding regions drive glioblastoma. To address this knowledge gap, researchers at Uppsala University have performed whole-genome sequencing of DNA in tumour tissues from patients with glioblastoma and analysed the identified mutations.

One of our key tasks was to identify functional mutations associated with regulatory elements and potential relevance to the development of cancer cells, and to distinguish them from all random variations without presumed significance, says Professor Karin Forsberg-Nilsson at the Department of Immunology, Genetics and Pathology, Uppsala University.

The researchers assumed that DNA sequences that have remained unchanged in mammals throughout evolution are likely to have important functions. Therefore, they intersected the thousands of mutations they had found with information about evolutionary conservation of the genetic regions where the mutations lie.

The researchers validated their results using the gene SEMA3C, partly because they found a large number of mutations in non-coding regulatory regions near this gene and partly because previous findings, by others, suggest that SEMA3C is linked to a poor cancer prognosis.

We studied how mutations in non-coding regions affect SEMA3C's function and activity. Our results show that a specific, evolutionarily conserved, mutation in the vicinity of SEMA3C disrupts the binding of certain proteins whose task is to bind genes and regulate their activity, says Forsberg-Nilsson.

The study also identifies more than 200 other genes enriched for non-coding mutations in the regions concerned. These likely have regulatory potential, thus further increasing the number of genes that are relevant to the development of brain tumours.

Our results confirm the importance of the association between genetic alterations in non-coding regions, their biological function and disease pathology, concludes Forsberg-Nilsson.

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New method to identify genes that can drive development of brain tumours - ETHealthworld.com

It may seem too much to expect that a persons geographic origin can be determined from a DNA sample. But, thanks to a mathematical technique called principal component analysis, this can be done with remarkable accuracy. It works by reducing multi-dimensional data sets to just a few variables.

We live in the age of big data. Voluminous data collections are mined for information using mathematical techniques. The data may be assembled in a matrix a rectangular array of numbers with a column for each individual and a row for each variable.

For a medical database, the variables might be age, height, weight, blood-group and numerous other relevant factors, resulting in a very large matrix. We can examine small tables of numbers visually and detect interesting patterns but, with many variables, each requiring a separate dimension, simple inspection may reveal nothing of value.

A simple example illustrates dimension reduction. Suppose we let the two axes of a graph measure height and weight. Taller people are usually heavier than shorter ones, so these two variables are not independent; they are correlated. Each individual is represented by a point, and all the points form a cloud. The cloud is not round in shape, but elongated. We can fine a straight line through the centre of the cloud in the direction of elongation, so that all the points lie close to this line. Thus, the essentials of the two-dimensional cloud are captured in the one-dimensional line.

Problems are much tougher to solve in higher dimensions; this is called the curse of dimensionality. Dimension reduction is essential in big data science. Interesting features can often be captured by isolating a few key combinations of variables. What is the best way to represent data so as to highlight features of interest? Can we reduce a large data set to a much smaller one while preserving essential characteristics? Is there redundancy that can be exploited, with many variables determined by others?

Many sophisticated analysis techniques have been developed that reduce the dimensions and reveal signals buried in extraneous noise. One method of great power is called principal component analysis (PCA). From data in a high dimensional space, this method determines a small number of new variables called principal components, allowing us to spot patterns. PCA also allows us to visualize the data in a simple two-dimensional diagram that often encapsulates the essence of the problem. Clusters of points with distinct behaviour can often be detected.

PCA has many applications, in acoustics, seismology, forensic science, meteorology and medicine. An intriguing application in genetics has shown that DNA can be used to infer an individuals geographic origin with remarkable accuracy - often to within a few hundred kilometres.

A paper in the journal Nature, with lead author John Novembre of UCLA, studied the genetic variation in a sample of more than 3000 European people. Each DNA specimen was genotyped at about half a million loci. PCA was then used to drastically reduce this data set to just two dimensions and depict it on a plane graph.

The first two principal components are correlated with perpendicular combinations of longitude and latitude. With appropriate orientation, their visualization had a striking resemblance to a map of Europe (a detail is shown in the figure). Individuals from the same region cluster together so that major populations can be identified. For example, clusters corresponding to the Iberian and Italian peninsulas are clear, and the Irish and British groups are easily distinguished.

The results mean that European DNA samples contain vital information about their donors. Thus, one can place 90 per cent of individuals within about 700 km of their geographic origin.

Peter Lynch is emeritus professor at UCD School of Mathematics & Statistics he blogs at thatsmaths.com

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The geography of Europe is mapped in our genes - The Irish Times

Imagine a world in which we can produce meat without animals, cure previously incurable diseases by editing an individuals genetic fabric, and manufacture industrial chemicals in yeast factories. The foundational technologies that could make all this possible largely exist. Rapid and ever-cheaper DNA sequencing has deepened our understanding of how biology works and tools such as CRISPR are now being used to recode biology to treat diseases or make crops less vulnerable to climate change. This is what we call the Bio Revolution.

Explored in a new McKinsey Global Institute research report, which we helped co-author, the Bio Revolution is already benefiting society. A confluence of breakthroughs in biological science and ever faster and more sophisticated computing, data analytics, and artificial intelligence technologies has powered scientific responses to the Covid-19 pandemic. Scientists sequenced the virus genome in weeks rather than months, as was the case in previous outbreaks. Bio innovations are enabling the rapid introduction of clinical trials of vaccines, the search for effective therapies, and a deep investigation of the transmission patterns of the virus.

The report estimates that bio innovations could alleviate between 1% and 3% of the total global burden of disease in the next 10 to 20 years from these applications roughly the equivalent of eliminating the global disease burden of lung cancer, breast cancer, and prostate cancer combined. Over time, if the full potential is captured, 45% of the global disease burden could be addressed using science that is conceivable today.

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As much as 60% of the physical inputs to the global economy today are either biological (such as wood for construction or animals bred for food) or nonbiological (such as cement or plastics) but could, in principle, be produced over time using biology. Nylon can already be made using genetically engineered yeast instead of petrochemicals, for instance, leather is being made from mushroom roots, and bacteria have made a type of cement.

This Bio Revolution has the potential to be as transformative to business and economies as the Digital Revolution that proceeded it, creating value in every sector, disrupting value chains, and creating new business opportunities. Businesses clearly see the potential investment in a new generation of biological technologies had already surged to more than $20 billion by 2018.

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Many applications are being commercialized. We identified a visible initial pipeline of about 400 use cases, almost all scientifically feasible today, that could create a direct economic impact of $2 trillion to $4 trillion in the next 10 to 20 years more than half of which is outside health, in sectors as diverse as agriculture and textile manufacturing.

The confluence of biology and computing is already creating new capabilities. Computing is accelerating discovery and throughput in biology. An explosion of biological data due to cheaper sequencing is being used by biotech companies and research institutes that are increasingly using robotic automation and sensors in labs. Biotech company Zymergen, for example, has found that throughput in biological screening can be increased up to 10 times. Advanced analytics, more powerful computational techniques, and AI are also being deployed to generate more acute insights during the R&D process.

New biology-based manufacturing is already cutting costs, improving performance, and reducing the impact on the environment and the natural world. In cosmetics, for instance, Amyris is now making squalane, a moisturizing oil used in many skin-care products, by fermenting sugars using genetically engineered yeast instead of processing liver oil from deep-sea sharks, which was not only expensive but threatened the species with extinction. In textiles, U.S. startup Tandem Repeat is producing self-repairing, biodegradable, and recyclable fabric using proteins encoded by squid genes.

The Bio Revolution could utterly change the food business as plant-based proteins and lab-grown meat gain popularity and in the process cut greenhouse gas emissions from deforestation and animal husbandry. One study found that cultured meat could reduce greenhouse gas emissions by 80% or more compared with conventional meat if all of the energy used in manufacturing comes from carbon-free sources.

Cultured meat and seafood are made using tissue-culture technology, a lab process by which animal cells are grown in vitro. Producers still face a major technical challenge in finding a cost-effective way of growing cells. New players such as Finless Foods, Mosa Meat, Memphis Meats, and Meatable are experimenting with different approaches, including using synthetic molecules and pluripotent stem cells to replace expensive growth factors. Cultured meat and seafood could be cost-competitive with conventional animal production systems within 10 years.

In agriculture, greater understanding of the role of the microbiome offers opportunities to improve operational efficiency and output. By profiling bacteria and fungi in the soil, Trace Genomics, for one, produces insights that help choose tailored seeds and nutrients, and enables early prediction of soil diseases. In consumer markets, ongoing research into the relationship between the gut microbiome and the skin is being used to personalize skin care. Singapore-based genomics firm Imagene Lab, for instance, offers a personalized serum based on the results of its skin DNA tests that assess traits such as premature collagen breakdown.

Such examples give a sense of the breadth of applicability of bio innovation, but there is a significant caveat: risk. Biology will preserve life through innovative treatments tailored to our genomes and microbiomes, but biology could also be the greatest threat to life if it is used to create bioweapons or genetically engineered viruses that can do lasting damage to the health of humans or ecosystems. The CRISPR gene-editing tool is revolutionizing medicine and is being applied to agriculture with great effect. But consider that CRISPR kits are now available to buy on the Internet for $100 and so-called biohackers are using them at home.

Like the Digital Revolution, the Bio Revolution comes with risks but of a different order of magnitude. If citizens already have misgivings about data being gathered about their shopping habits, how much more nervous will they be about genetic data gathered from their bodies for medical treatment or ancestry tracing data that couldnt be more personal.

Another risk is that biological organisms are, by their nature, self-sustaining and self-replicating. Genetically engineered microbes, plants, and animals may be able to reproduce and sustain themselves over the long term, potentially affecting entire ecosystems. Once Pandoras box is opened and we have already cracked the lid we may have little control over what happens next.

Unless such risks are managed, it is possible that the full potential of the Bio Revolution may not materialize. We estimate that about 70% of the total potential impact could hinge on societal attitudes and the way innovation is governed under existing regulatory regimes. Yet if the risks can be managed and mitigated, the Bio Revolution can reshape our world. Scientists, in conjunction with forward-thinking companies, are now harnessing the power of nature to solve pressing problems in medicine, agriculture, and beyond, and helping craft a response to global challenges from pandemics to climate change.

Matthias Evers is a senior partner and global leader of research and development in McKinsey & Companys pharmaceuticals and medical products practice. Michael Chui is a partner at the McKinsey Global Institute, McKinseys business and economics research arm.

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The Bio Revolution is changing business and society - STAT - STAT

Knowing what cancer will do next could lessen the likelihood of it becoming resistant to treatment. A new U of T study investigates how cancer adapts its metabolism to potentially overcome therapies still in development.

Several clinical trials have failed because metabolism is such an adaptive process by which cancer cells gain drug resistance, saysMichael Aregger, a co-lead author and Research Associate working withJason Moffat, Professor of molecular genetics in the Donnelly Centre for Cellular and Biomolecular Research, who co-led the work. If you know how cells are able to adapt to perturbations, maybe we can target them more specifically to avoid resistance from developing.

The research was also led byBrenda AndrewsandCharles Boone, University Professor and Professor of molecular genetics at the Donnelly Centre, respectively, andChad Myers, a Professor of computer science at the University of Minnesota-Twin Cities.

Thestudy, published this week in the journalNature Metabolism, is the first to investigate global changes in cancerous cells as they adapt to a shortfall of critical nutrients such as fat molecules, or lipids, which make up the cells outer envelope.

When cancer cells are unable to make their own lipids, they gobble them up from their environment to ensure a steady supply of these essential building blocks, the study found. Lipids also serve as fuel and chemical signals for communication between cells, among other roles.

"If you know how cells are able to adapt to perturbations, maybe we can target them more specifically to avoid resistance from developing" - Michael Aregger, Research Associate

The switch in metabolism could be bad news for drugmakers seeking to target cancer by reducing its lipid reserves. In particular, drugs that inhibit an enzyme called FASN, forfattyacidsynthase, involved in an early step of lipid synthesis, are being explored in patient trials. Fatty acids are precursors of larger lipid molecules and their production is increased in many cancers thanks to elevated FASN levels, which are also associated with poor patient prognosis.

The U of T study suggests that the effectiveness of FASN inhibitors could be short-lived owing to cancers ability to find another way to procure lipids.

Because FASN is upregulated in many cancers, fatty acid synthesis is one of the most promising metabolic pathways to target saysKeith Lawson, a co-lead author and PhD student in Moffats lab enrolled in the Surgeon-Scientist Program at the Faculty of Medicine. Given that we know there is a lot of plasticity in metabolic processes, we wanted to identify and predict ways in which cancer cells can potentially overcome the inhibition of lipid synthesis.

To block fatty acid synthesis, the researchers employed a human cell line from which the FASN coding gene was removed. Using the genome editing tool CRISPR, they deleted from these cells all ~18,000 or so human genes, one by one, to find those that can compensate for the halt in lipid production. Such functional relationships are also referred to as genetic interactions.

Data analysis, performed byMaximilian Billmann, a co-lead author and a postdoctoral fellow in Myers lab at Minnesota-Twin Cities, revealed hundreds of genes that become essential when cells are starved of fat. Their protein products clustered into well-known metabolic pathways through which cells hoover up dietary cholesterol and other lipids from their surroundings.

Cells intake of cholesterol has become textbook knowledge since it was discovered half a century ago, winning aNobel Prizeand inspiring the blockbuster drug statin and many others. But the new study found that one component of this process remained overlooked all this time.

The gene encoding it was only known as C12orf49, named after its location on chromosome 12. The researchers re-named the gene LUR1, forlipiduptakeregulator 1, and showed that it helps switch on a set of genes directly involved in lipid import.

This was a big surprise to us that we were able to identify a new component of the process we thought we knew everything about, says Aregger. It really highlights the power of our global genetic interaction approach that allowed us to identify a new player in lipid uptake in a completely unbiased way.

By a remarkable coincidence, two groups working independently in New York and Amsterdam also linked C12orf49 to lipid metabolism, lending further support for the genes role in this process. The New York team published their findings in the same journal issue as Moffat and colleagues.

Inhibiting LUR1, or other components of lipid import, along with FASN could lead to more effective cancer treatments. Such combination therapies are thought to be less susceptible to emerging drug resistance because the cells would have to simultaneously overcome two obstaclesblocked lipid production and importwhich has a lower probability of occurring.

Therapeutic context that comes out of our work is that you should be targeting lipid uptake in addition to targeting lipid synthesis and our work highlights some specific genes that could be candidates, says Lawson.

The research was supported by the Canadian Institutes for Health Research, Ontario Research Fund, Canada Research Chairs Program and the U.S. National Institutes of Health.

Reference:Aregger, M., Lawson, K.A., Billmann, M. et al. (2020) Systematic mapping of genetic interactions for de novo fatty acid synthesis identifies C12orf49 as a regulator of lipid metabolism. Nat Metab. DOI: https://doi.org/10.1038/s42255-020-0211-z

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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When Cancer Cells Cant Produce Their Own Fat, They Import More of It - Technology Networks

With $76 million in financing, San Diego-based Autobahn Therapeutics is hitting the ground with a goal of developing regenerative medicines aimed at treating disorders of the central nervous system (CNS).

This morning, the company officially launched with a Series B funding round backed by some of the biggest names in the life sciences, including Bristol Myers Squibb, Biogen and Pfizer. The companys scientific approach is focused on the role that thyroid hormone plays in the production of myelin, the protective sheath that forms around nerves. The degeneration of myelin is associated with many CNS disorders, including multiple sclerosis (MS). Autobahn is developing small molecule, thyroid hormone receptor beta agonists designed to stimulate remyelination and address the progressive nature of MS and other diseases that result from demyelination. The companys work is built on research conducted by Thomas Scanlan, a professor of Chemical Physiology and Biochemistry at Oregon Health & Science University.

Proceeds from the Series B funding round will be used to advance the companys lead candidate, ABX-002, a thyroid hormone receptor beta agonist therapy for the treatment of multiple sclerosis (MS) and adrenomyeloneuropathy (AMN), a rare genetic disorder. Funds will also be used to develop a portfolio of transformational CNS programs leveraging the companys brain-targeting chemistry platform.

Kevin Finney, chairman and chief executive officer of Autobahn, said the company is using the bodys own regenerative power to both treat and prevent prevalent disorders of the central nervous system.

We are coupling our deep knowledge of thyroid hormone biology and remyelination with our brain-targeting chemistry platform to restore the brain to a healthier state. We stand well-positioned to advance our pipeline with funding from the highest quality investors and pharmaceutical leaders who share our mission of improving life health for people affected by these conditions, Finney said in a statement.

In addition to the pharma giants that backed Autobahns Series B, the round was led by ARCH Venture Partners and Cowen Healthcare Investments. Other supporters of the financing were BVF Partners L.P., Invus, Section 32, Samsara BioCapital and Alexandria Venture Investments.

Alongside CEO Finney, Autobahn established a leadership team with a proven track record in thyromimetics, drug discovery and development, clinical operations, and corporate and business development. Keith Lenden will serve as president and chief operating officer; Brian Stearns will serve as chief scientific officer; and Chan Beals will serve as head of translational medicine. Scanlan serves as a senior adviser to Autobahn.

Tim Anderson, managing director of Cowen Healthcare Investments and a member of the Autobahn Board of Directors, said the companys differentiated portfolio of CNS therapies is backed by validated science and led by proven scientific and business leaders.

In addition to Anderson, the board includes ARCHs Kristina Burow, Finney and Ben Cravatt, the Gilula Chair of Chemical Biology and professor in the Department of Chemistry at The Scripps Research. Institute.

Autobahn benefits from the winning combination of validated human biology supporting its therapeutic hypothesis, clever chemistry enabling tissue-selective CNS delivery of compounds and an exceptional team to drive it forward, Cravatt said in a statement. I am highly confident in our potential to create the next generation of regenerative medicines for the brain.

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Autobahn Therapeutics Takes Aim at CNS Disorders with $76 Million Series B - BioSpace

Scientists have long debated which genetic information carrier DNA or RNA started life on Earth, but a new study suggests life could have begun with a bit of both. The research, led by scientists from the Medical Research Council (MRC) Laboratory of Molecular Biology (LMB), in Cambridge, shows for the first time how some of the building blocks of both DNA and RNA could have spontaneously formed and co-existed in the primordial soup on Earth.

The work challenges one of the leading hypotheses for the advent of life the RNA world theory, which arose in the 60s and has gained wide acceptance.

Today, all known living organisms use the same genetic molecules called nucleic acids to store information. There are two sorts of nucleic acids: DNA and RNA. DNA encodes instructions in genes. Genes are turned into messages using RNA, which carries instructions to make proteins. Proteins can make structures and act as molecular machines.

In the RNA world theory, life started with RNA molecules, which can both store instructions and can act as a modest machine, potentially enabling them to self-replicate. It proposes that through evolution, life in the RNA world gave way to the era of DNA and proteins, because DNA is more stable and durable than RNA.

In the current study, published in Nature, the researchers simulated the conditions on a primordial rocky Earth with shallow ponds in the lab. They dissolved chemicals that form RNA in water, then dried them out and heated them, then they simulated the early suns rays by exposing them to UV radiation.

In this recreation of early Earth geochemistry, intermediates in the synthesis of two of the building blocks of RNA were simultaneously also converted into two of the building blocks of DNA.

It is the first demonstration that reasonable amounts of a genetic alphabet made up of four building blocks, two for RNA and two for DNA potentially sufficient to have encoded early life, which was far less complex than life today may have been available on the primordial Earth.

Professor John Sutherland from the MRC Laboratory of Molecular Biology, who led the work, says: The RNA world hypothesis suggests that life began with RNA, before a genetic takeover occurred involving primitive biosynthetic machinery and natural selection to result in DNA.

Our work suggests that in conditions consistent with shallow primordial ponds and rivulets there was a mixed genetic system with RNA and DNA building blocks co-existing at the dawn of life. This fulfills what many people think is a key precondition for the spontaneous emergence of life on Earth.

The teams experiments to simulate early Earth geochemistry showed that four of the building blocks for DNA and RNA can arise from the same reagents and conditions. They produced cytidine and uridine, two of the building blocks of RNA, and deoxyadenosine, which is one of those of DNA. Deoxyadenosine was partly converted to deoxyinosine, which can take the role of another DNA building block.

They believe that these four building blocks may have coexisted before life evolved and were the beginnings of a primitive genetic alphabet.

Professor Sutherland adds: The nucleic acids, RNA and DNA, are clearly related and this work suggests that they both derive from a hybrid ancestor, rather than one preceding the other.

Since genetic information always flows from nucleic acids to proteins, and never in reverse a principle called the central dogma of molecular biology by Francis Crick we now need to uncover how the information which can be stored and purveyed by these nucleic acids could have been first used to make to proteins.

Understanding the chemical origins of life is a fundamental aspect of natural science, and can inform the design of future synthetic biology.

Dr. Megan Dowie, head of molecular and cellular medicine at the MRC commented: This study shows that blue skies research can reveal fascinating insights into how the very beginnings of life may have emerged, and demonstrates the importance of supporting fundamental research. These underpinning discoveries in the life sciences could enable exciting future strategies for artificial biology.

Reference: Selective prebiotic formation of RNA pyrimidine and DNA purine nucleosides by Jianfeng Xu, Vclav Chmela, Nicholas J. Green, David A. Russell, Mikoaj J. Janicki, Robert W. Gra, Rafa Szabla, Andrew D. Bond & John D. Sutherland,3 June 2020, Nature.DOI: 10.1038/s41586-020-2330-9

See the article here:
Did Life Emerge in the Primordial Soup via DNA or RNA? Surprising Answer From New Research - SciTechDaily

Autobahn Therapeutics, a San Diego, CA-based developer of regenerative medicines to restore hope for people affected by CNS disorders, completed a $76m Series B funding round.

The round was co-led by Arch Venture Partners and Cowen Healthcare Investments, with participation from BVF Partners L.P., Biogen, Bristol Myers Squibb, Pfizer Ventures, Invus, Section 32, Samsara BioCapital and Alexandria Venture Investments.

The company intends to use the funds to advance its lead program candidate, ABX-002, a thyroid hormone receptor beta agonist therapy for the treatment of multiple sclerosis (MS) and adrenomyeloneuropathy (AMN), a rare genetic disorder, and a portfolio of transformational CNS programs leveraging its brain-targeting chemistry platform.

Led by Kevin Finney, chairman and chief executive officer, Autobahn Therapeutics is focused on improving life health for people affected by CNS disorders. The company is leveraging a deep understanding of validated human biology coupled with its brain-targeting chemistry platform to develop thyroid hormone receptor beta agonist therapies that harness the regenerative power of the human body. The pipeline is led by ABX-002 for the treatment of multiple sclerosis and adrenomyeloneuropathy (AMN), a rare genetic disorder.

The company has built a team of experts in thyromimetics, drug discovery and development, clinical operations, and corporate and business development:

Kevin Finney, chairman of the board of directors and chief executive officer;

Keith Lenden, co-founder, president and chief operating officer;

Brian Stearns, Ph.D., chief scientific officer;

Chan Beals, M.D., Ph.D., senior vice president of translational medicine;

Christine Maurer, senior vice president of development operations and program management;

John Borkholder, J.D., general counsel and senior vice president of administration; and

Thomas Scanlan, Ph.D., co-founder and senior advisor to Autobahn, and professor of Chemical Physiology and Biochemistry, Oregon Health & Science University.

FinSMEs

09/06/2020

Continued here:
Autobahn Therapeutics Raises $76M in Series B Financing - FinSMEs

MENLO PARK, Calif.--(BUSINESS WIRE)--Personalis, Inc. (Nasdaq: PSNL) today announced a collaboration with Sarepta Therapeutics (Nasdaq: SRPT), a leader in precision genetic medicine for rare disease. As part of this research collaboration, Sarepta will be working with the Personalis team to characterize immune response to precision genetic therapeutics, utilizing Personalis advanced proprietary analytics.

We are excited to announce this collaboration with Personalis. By bringing together Sareptas expertise in precision genetic medicine and Personalis advanced neoepitope prediction, our goal is to better characterize certain types of immune response to benefit patients with rare disease, said Dr. Tanya Teslovich, Senior Director, Genomics at Sarepta.

We are excited to work with Sarepta to apply our proprietary analytics in the rapidly emerging area of genetic medicine. This collaboration demonstrates the extensibility of our technology platform beyond cancer to additional therapy development areas, said Dr. Richard Chen, CSO at Personalis.

About Personalis, Inc.

Personalis, Inc. is a growing cancer genomics company transforming the development of next-generation therapies by providing more comprehensive molecular data about each patients cancer and immune response. The Personalis ImmunoID NeXT Platform is designed to adapt to the complex and evolving understanding of cancer, providing its biopharmaceutical customers with information on all of the approximately 20,000 human genes, together with the immune system, from a single tissue sample. Personalis also provides genomic information to the VA Million Veterans Program as part of their goal to sequence over a million veteran genomes. The Personalis Clinical Laboratory is GxP aligned as well as CLIA88-certified and CAP-accredited. For more information, please visit http://www.personalis.com and follow Personalis on Twitter (@PersonalisInc).

Forward-Looking Statements

This press release contains or may imply "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. For example, forward-looking statements include statements regarding potential positive outcomes resulting from the collaboration of Personalis and Sarepta, such as the ability of the collaboration to yield novel discoveries. These forward-looking statements are subject to risks and uncertainties, including those discussed in Personalis filings with the Securities and Exchange Commission (SEC), including in the Risk Factors and Managements Discussion and Analysis of Financial Condition and Results of Operations sections of the Companys most recently filed periodic report on Form 10-K and subsequent filings and in the documents incorporated by reference therein. Except as otherwise required by law, Personalis disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

Link:
Personalis Announces Scientific Collaboration with Sarepta Therapeutics on Immune Response to Precision Genetic Therapeutics - Business Wire

GOVERNMENT

Baker names Judy Chang undersecretary of energy

Governor Charlie Baker has a new undersecretary of energy: Judy Chang, formerly an energy economist with the Brattle Group consultancy. Changs main job in state government will be to advise Energy and Environmental Affairs Secretary Katie Theoharides on developing and implementing energy policies. Chang replaces Patrick Woodcock, who was promoted to be commissioner of the Department of Energy Resources in February. JON CHESTO

JOBS

Companies cut 2.8 milion jobs in May, far less than expected

US businesses shed 2.8 million jobs in May, significantly less than the 9.3 million job losses that were expected. The payroll company ADP reported Wednesday that businesses have let go of a combined 22.6 million jobs since March, with the bulk of the layoffs occurring in April. The virus forced employers to shutter offices, factories, gyms, and schools, while demand for gasoline, clothing, airline tickets, hotel rooms, and restaurant meals quickly vanished. The damage was concentrated in two sectors. Manufacturers cut 719,000 jobs in May. The trade, transportation, and utilities sector let go of 826,000. Other sectors that suffered as part of Aprils 19.6 million job losses saw their layoffs slow sharply. The leisure and hospitality industry which includes hotels and restaurants shed 105,000 jobs last month, down from a revised 7.7 million losses in April. The private industry report comes two days ahead of the official monthly job figures from the US Labor Department. Economists expect the Friday report will show 8 million job losses in May as the unemployment rate approaches 20 percent. ASSOCIATED PRESS

FINANCE

N.Y. regulators probe Deutsche Banks ties to Jeffrey Epstein

New York state regulators are investigating Deutsche Banks relationship with the disgraced late financier Jeffrey Epstein, adding another compliance issue to a growing list for chief executive Christian Sewing. The New York Department of Financial Services has been looking into the banks dealings with Epstein as part of a broader look into the German lenders compliance and controls, according to a person familiar with the matter. Epstein was arrested last year on federal sex-trafficking charges more than a decade after he pleaded guilty to Florida state charges of soliciting an underage girl for sex. His death in a New York City jail was ruled a suicide. BLOOMBERG NEWS

ENERGY

Germany wants major boost to offshore wind power

The German government wants to increase offshore wind power capacity fivefold by 2040 as part of its plan to wean the country off fossil fuels. The Cabinet on Wednesday agreed on a bill that would set a goal of 40 gigawatts of installed offshore wind power capacity in 20 years, from about 7.5 gigawatts at present. It also raised the target for 2030 from 15 gigawatts to 20. Economy Minister Peter Altmaier said the new offshore wind target for 2030 would help Germany achieve its goal of meeting 65 percent of its gross electricity consumption with renewable energy in a decade. ASSOCIATED PRESS

INTERNATIONAL

France blasts US probe into digital taxes

France slammed the United States over its probe into digital taxes that are being considered by a number of countries, saying it contradicts Washingtons call for unity among leading economies. There is a real contradiction between the US demanding unity within the Group of Seven which we support and the possiblity of new trade sanctions, French Finance Minister Bruno Le Maire said after a G-7 telephone conference Wednesday. The French and US government agreed a truce earlier this year in a dispute over Frances digital services tax, according to which Washington is holding back on sanctions and Paris is suspending the collection of its levy. France will resume collecting the tax at the end of the year unless there is an agreement in talks at the OECD on new global tax rules. On Tuesday, the Trump administration started investigations into digital services taxes considered by several trading partners from the European Union to India. A similar investigation into Frances tax led to the threat of tariffs. BLOOMBERG NEWS

VIDEO CONFERENCING

Zoom sales climb as virtual meetings become the norm

Zoom Video Communications Inc. demonstrated that paying customers have flocked to its virtual-meeting software, transforming the once-niche appmaker into a popular communications service and positioning it to benefit as the nature of work, school, and life is upended. Zoom reported that sales soared in the three months ending April 30, when the coronavirus pandemic spurred a wave of stay-at-home orders for millions of people worldwide. The company expects the trend to continue the rest of the year, and projected that revenue and profit will leapfrog investors earlier expectations. While security and privacy issues plagued the system early in the quarantine, Zoom has become an essential service, attracting more than 300 million participants some days, up from 10 million in December. The software maker allows gatherings of as long as 40 minutes for no charge. While Zoom has attracted more buzz than corporate rivals, the results Tuesday suggested it can attract the paying clients needed to compete against services from Microsoft, Cisco Systems, and Google. BLOOMBERG NEWS

COSMETICS

Coty wants Kim as well as Kylie

Coty Inc. cant seem to get enough of the Kardashians. The cosmetics company is in talks with Kim Kardashian West for a possible collaboration with respect to certain beauty products, according to a regulatory filing. The possible partnership comes just months after Coty closed a $600 million deal with the reality TV stars sister, Kylie Jenner. Coty agreed last month to sell Clairol and other brands for $4.3 billion in part to prioritize investment in Kylie Cosmetics. BLOOMBERG NEWS

POULTRY

CEO of 2nd largest US chicken producer charged with price fixing

The chief executive officer of Pilgrims Pride Corp., Americas second-biggest chicken producer, was charged by US prosecutors with conspiring to fix prices as part of an antitrust investigation of chicken-processing companies. Jayson Penn was indicted by a grand jury in Colorado along with Roger Austin, a former vice president of the company, the Justice Department said Wednesday. They face a statutory maximum penalty of 10 years in prison and a $1 million fine. The allegations against the leader of a top American poultry producer were the latest bombshell to hit the meat industry thats been reeling from thousands of workers sickened by COVID-19, forcing shutdowns at processing plants. The US government is also probing potential market manipulation at beef processors, who were turning big profits while farmers suffered from plant outages. BLOOMBERG NEWS

Excerpt from:
Sarepta to expand in Ohio - The Boston Globe

BEDFORD, Mass.--(BUSINESS WIRE)--Stoke Therapeutics, Inc., (Nasdaq: STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today announced the appointment of Julie Anne Smith to its Board of Directors. Ms. Smith has also been appointed to the Compensation Committee of the Board of Directors. Ms. Smith will replace Samuel Hall, Ph.D., whose term on the Board of Directors expired.

Julie brings more than two decades of experience in the life sciences industry, with a strong track record of successfully developing and commercializing medicines for rare and inherited diseases. Her expertise in drug development for neurodegenerative diseases will be particularly valued as we advance STK-001 for Dravet syndrome into the clinic later this year, said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. We thank Sam for his many important contributions to Stoke from our inception and as we matured through our successful IPO to become a public company prepared to enter the clinic with STK-001, the first potential medicine developed using our TANGO platform. We welcome Julie to the Board and look forward to her insights and contributions.

This is an exciting time for Stoke as it transitions to a clinical stage company and looks to the future, said Ms. Smith. I am pleased to work with the Board members and the executive leadership team as they advance their work in Dravet and expand the pipeline to help people who are living with severe genetic diseases and realize the potential of the TANGO platform.

Ms. Smith currently serves as President and CEO of ESCAPE Bio, Inc., a biotechnology company developing precisely targeted therapeutics for genetic forms of neurodegenerative disease. She previously served as President and CEO of Nuredis, Inc., a biotechnology company developing small-molecule therapies for nucleotide repeat disorders such as Huntingtons disease. In 2014, Ms. Smith was appointed President and CEO at Raptor Pharmaceuticals, a public biotechnology company with two commercial medicines for orphan diseases, where she served until its acquisition in 2016 (by Horizon Pharmaceuticals, Inc.). Prior to joining Raptor, Ms. Smith served as the Chief Commercial Officer at Enobia Pharmaceuticals (acquired by Alexion Pharmaceuticals, Inc.). Earlier in her career, she held positions of increasing responsibility at Jazz Pharmaceuticals plc, Genzyme, Novazyme and Bristol-Myers Squibb Company.

Ms. Smith previously served on the board of directors of Audentes Therapeutics, Inc., a genetic medicines company, and as a director on the Health and Emerging Companies Section Governing Boards of the Biotechnology Industry Organization (BIO). She currently serves on the board of directors of Exelixis, Inc., a public genomics-based drug discovery company. Ms. Smith holds a B.S. in biological and nutritional sciences from Cornell University.

About Stoke Therapeutics

Stoke Therapeutics (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. 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.

Cautionary Note Regarding 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: Stokes expectation about timing and execution of anticipated milestones with respect to STK-001, including advancement of STK-001 to the clinical stage, and expansion of the Companys pipeline. Statements including words such as plan, continue, expect, or ongoing and statements in the future tense are forward-looking statements. 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. Forward-looking statements are subject to risks and uncertainties that may cause Stokes actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to Stokes ability to develop, obtain regulatory approval for and commercialize STK-001 and its future product candidates, the timing and results of preclinical studies and clinical trials, Stokes ability to protect intellectual property; and other risks set forth in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These forward-looking statements are based on our current believes 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.

Original post:
Stoke Therapeutics Appoints Julie Anne Smith to its Board of Directors - Business Wire

HOUSTON, June 4, 2020 /PRNewswire/ -- The Golden Helix Foundation and the Pharmacogenomics Access & Reimbursement Coalition (PARC) will co-present the inaugural Pharmacogenomics Access & Reimbursement Symposium (PARS) at the National Academy of Sciences Building on October 8, 2020 in Washington D.C. to define opportunities to expand patient access to personalized medicine. Public- and private- sector members across healthcare will assemble to develop a path forward through discussions of best practices, improved economic evaluation and strategic alignment.

"Since genetic variation impacts medication responses, it is important to leverage technologies that can translate genetic information into care tailored for each patient.Market access is a critical step in achieving widespread adoption of personalized medicine." Sara Rogers, PARC Co-Chairman and Director at the American Society of Pharmacovigilance.

The symposium program includes speakers from industry, government agencies, payer organizations, health systems and health policy organizations. Thought leaders will explore the newest developments in health technology assessment, health economics and value-based payment strategies for pharmacogenomics. In addition to convening decision-makers from around the world, the symposium will develop solutions that identify actionable ways that stakeholders can work together to democratize personalized medicine.

"Defining the value of personalized medicine interventions is of utmost importance to expedite the incorporation of these innovative healthcare solutions in routine clinical practice that would directly impact patient care and quality of life." Christina Mitropoulou, The Golden Helix Foundation Managing Director, Executive Board member and Principal Investigator of the Ubiquitous Pharmacogenomics Consortium.

Organizations across health care have partnered to support the symposium, including Intermountain Precision Genomics, Medical Device Innovation Consortium (MDIC) and Pharmacogenomics Research Network (PGRN). Stakeholders are encouraged to join the discussion by registering to participate athttp://www.parcoalition.org/symposium. A live webcast will be provided to accommodate attendees who prefer to participate remotely.

About the Golden Helix Foundation The Golden Helix Foundation is an international non-profit research organization (registered London-based UK charity) aiming to advance research and education in the area of genome and personalized medicine. The Golden Helix Foundation aims to promote the development of research and the transfer and communication of knowledge from researchers and scientists in the wider scientific community through collaborative projects and conferences in the field of pharmacogenomicsand personalized medicine.

About the Pharmacogenomics Access & Reimbursement Coalition (PARC)PARC seeks to address barriers to patient access and payer coverage of Pharmacogenomics (PGx) testing by sharing resources and leveraging shared expertise in PGx. For more information, please contact [emailprotected] and follow @PGxARC

American Society of PharmacovigilanceP.O. Box 20433Houston, TX 77225www.stopADR.org

Contact: Geneva MorelDirector of Communications Email: [emailprotected] phone: 469-939-8475

SOURCE American Society of Pharmacovigilance

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The Golden Helix Foundation and PARC Co-Present International Meeting to Expand Patient Access to Personalized Medicine - PRNewswire

Mark Williams The Columbus Dispatch

TuesdayJun2,2020at5:11PM

A Massachusetts-based biopharmaceutical company plans to create 100 jobs as part of an expansion of its Columbus operations.

Sarepta Therapeutics will open an 85,000-square-foot building at 3435 Stelzer Rd. as part of its Gene Therapy Center of Excellence.

The company says it will invest more than $30 million, and that hiring for research, technician and general operations positions will begin immediately.

Employees currently working out of the companys offices in Dublin will move to the new building over time.

Sarepta, based in Cambridge, focuses on gene therapy programs to treat rare diseases.

It has two approved drugs for Duchenne muscular dystrophy and more than 40 treatments in development.

Duchenne slowly steals muscle, making children weaker and weaker as they grow older. Many died by their mid-20s. The disease afflicts mostly boys.

We are confident that gene therapy will revolutionize genetic medicine, and we chose Ohio for our Gene Therapy Center of Excellence because we believe Columbus will become a hub for genetic medicine innovation, the companys president and CEO, Doug Ingram, said in a statement.

mawilliams@dispatch.com

@BizMarkWilliams

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Sarepta to expand Columbus operations, add 100 jobs - The Columbus Dispatch