StemCell Therapy

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

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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

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

OKLAHOMA CITY, June 3, 2020 /PRNewswire/ --Progentec, a leader in next-gen diagnostics and digital technologies for the management of autoimmune diseases, announced today that Hakan Sakul, Ph.D., Vice President and Head of Diagnostics at Pfizer, has joined the Progentec Board of Directors. Dr. Sakul has a distinguished track record in the development and commercialization of companion diagnostics, precision medicine, and pharmacogenomics. His wealth of experience and knowledge will support Progentec in its mission to improve health outcomes for people living with autoimmune diseases.

"Lupus is a difficult disease to diagnose and a lab-based testing is sorely needed to confirm its diagnosis," said Dr. Sakul. "I am delighted to join Progentec's Board in this important phase of the company's diagnostics development journey."

In addition to his R&D, clinical, and product expertise, Dr. Sakul has deep experience in regulatory policy. He is currently a member of California Gov. Newsom's Precision Medicine Advisory Council, and was previously an Advisory Board member for California Gov. Brown's Precision Medicine Advisory Committee. Dr. Sakul is also on the board of directors for the Personalized Medicine Coalition and serves on Luminex Corporation's Oncology Advisory Committee.

About Progentec Diagnostics, Inc.

Progentec is committed to improving access and health outcomes for patients in therapeutic areas with a high level of unmet need by combining clinically-validated diagnostic interventions with state-of-the-art digital technologies. Through collaborations with research institutions and health practitioners around the world, Progentec is working to reduce mortality and morbidity while improving care management and service delivery for chronic health conditions.

Forward-Looking Statements:

Statements contained herein that are not historical facts are forward-looking statements that represent management's beliefs and assumptions as of the date of this news release based on currently available information. Although the Company believes that the expectations reflected in such forward-looking statements are reasonable, it cannot assure that these expectations will prove to be correct. Such statements involve risks and uncertainties that could cause actual results to differ materially from those expressed in any forward-looking statements.

Contact:

Mohan Purushothaman

(973) 885-5242

[emailprotected]

SOURCE Progentec Diagnostics, Inc.

http://www.progentec.com

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Precision Medicine and Diagnostics Expert Hakan Sakul, Ph.D., of Pfizer Joins the Progentec Board of Directors - PRNewswire

Fasten your seatbelts, folks, because the stock market has been on one heck of an emotional roller-coaster over much of the past four months. Initially, panic and uncertainty tied to the coronavirus disease 2019 (COVID-19) pandemic sent the broad-based S&P 500 down 34% in less than five weeks. But in the subsequent 11 weeks, we've witnessed roughly 80% of this initial drop clawed back by equities, with Wall Street appearing to enter a new bull market.

Although swoons in the stock market are inevitable, history has decisively shown that opportunistic long-term investors who buy during these periods of weakness tend to make money. It's never really a question of whether you should invest when a new bull market emerges, but where you should park your capital.

Assuming a new bull market has been established, the following four industries look set to thrive (and dominate) for a long time to come.

Image source: Getty Images.

One of the bigger beneficiaries of the COVID-19 pandemic is cybersecurity. To be perfectly clear, cybersecurity was already growing at a healthy pace well before the coronavirus shut down nonessential businesses across much of the country. But with more employees being forced to work remotely than ever before, the need to secure cloud-based data is taking on added importance. Essentially, COVID-19 took an existing trend and gave it a shot of adrenaline.

Hands down, my favorite company in this space is Palo Alto Networks (NYSE:PANW), which I pegged on April 9, 2020, as one of five stocks that could be a 10-bagger (i.e., deliver 1,000%-plus returns) by 2030.

Aside from the fact that enterprise cloud demand continues to pick up, Palo Alto benefits from the fact that it's pushing subscription and support-based solutionsas opposed to hardware. Subscriptions generate much better margins than hardware, and cash flow is far more predictable as Palo Alto is unlikely to see customer churn for what's effectively a basic-need service for any business.

Over the past year, Palo Alto's subscription and support revenue has grown to account for 68% of total sales, up from approximately 62% in the prior-year quarter.

Image source: Getty Images.

Building off of cybersecurity, the hottest thing since sliced bread in the new bull market is going to be anything having to do with cloud computing. This could mean the infrastructure players that are responsible for aiding businesses in creating their cloud, platform-as-a-service providers that allow enterprises to build, manage, and deploy applications, and software-as-a-service companies, which operate and sell ready-to-use apps for businesses and consumers.

Interestingly, some of the most prominent names in the cloud space are the United States' largest publicly traded companies: Amazon (NASDAQ:AMZN) with Amazon Web Services (AWS), Microsoft with Azure, and Alphabet with Google Cloud.

I don't see how investors can't get excited about Amazon in the new bull market. Even putting aside its e-commerce dominance, AWS continues to grow at about twice the pace of its retail/ad operations, and it produces considerably better margins. With Amazon's cloud services now accounting for 13.5% of total sales in Q1 2020 and a majority of its operating income, it's no wonder Wall Street expects a near-tripling in the company's operating cash flow by 2023.

Image source: Getty Images.

The new bull market will also feature an ongoing push toward personalized medicine. By personalized medicine, I'm referring to the idea of tailoring treatment for each individual rather than offering generalized medical solutions. Most personalized medicine leans on the increased use of technology, the cloud, and innovative new devices.

For example, another 10-bagger stock I singled out in April is Livongo Health (NASDAQ:LVGO), a provider of personalized health solutions. Using mountains of collected data and artificial intelligence, Livongo sends tips to members with diabetes in order to help them make meaningful behavioral changes that result in them living longer and healthier lives. It also doesn't hurt that Livongo's solutions can potentially work hand in hand with other wireless diabetes devices (e.g., insulin pumps), meaning patients don't have to leave the comfort of their homes to convey sensitive data to their physicians.

Maybe the best thing about Livongo Health and the personalized medicine push is that it's not all talk. Livongo's Diabetes member count doubled from the prior-year quarter to more than 328,000, and it's already generated two consecutive quarters of profitability despite significant reinvestment into its solutions.

If Livongo is already turning the corner to profitability with less than 1% of U.S. diabetes market share, just imagine its potential when it expands to treat other chronic illnesses, like hypertension.

Image source: Getty Images.

Finally, look for the Internet of Things (IoT) to finally realize its full potential in the new bull market. By IoT, I'm talking about wireless devices being able to connect and communicate with one another and data centers. Examples might include a smart thermostat that learns your heating and cooling preferences to conserve energy usage in your home, or an automobile that automatically reorders a defective or soon-to-be-defective component.

It's not uncommon for Wall Street and investors to overestimate the impact of new technology and be left disappointed. This is sort of what happened with IoT stocks in the latter half of the 2010s. But with an increased reliance on work-from-home, the cloud, and even personalized medicine, the value of IoT is rising like never before.

To build off of the previous industry (personalized medicine), DexCom (NASDAQ:DXCM) is the perfect example of IoT in action. Dexcom, which happens to have a deal in place with Livongo Health, is a producer of continuous glucose monitoring devices for diabetics. DexCom's devices can provide patients with real-time blood-glucose levels, communicate with insulin pumps to administer or hold off on the administration of insulin, and aid in producing reports that can be sent to a primary care provider.

IoT has applications in virtually every industry, and the new bull market should showcase that fact.

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4 Industries That'll Thrive in the New Bull Market - Motley Fool

New York, June 09, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global Humanized Mouse Model Market 2020-2024" - https://www.reportlinker.com/p05048523/?utm_source=GNW Our reports on humanized mouse model market provides a holistic analysis, market size and forecast, trends, growth drivers, and challenges, as well as vendor analysis covering around 25 vendors. The report offers an up-to-date analysis regarding the current global market scenario, latest trends and drivers, and the overall market environment. The market is driven by the rising focus on preventing the spread of pandemics and innovative applications. In addition, rising focus on preventing the spread of pandemics is anticipated to boost the growth of the market as well. The humanized mouse model market analysis includes product segment and geographic landscapes

The humanized mouse model market is segmented as below: By Product Genetically humanized mouse model Cell-based humanized mouse model

By Geographic Landscapes North America Europe Asia ROW

This study identifies growing adoption of personalized medicine as one of the prime reasons driving the humanized mouse model market growth during the next few years. "The analyst presents a detailed picture of the market by the way of study, synthesis, and summation of data from multiple sources by an analysis of key parameters. Our humanized mouse model market covers the following areas: Humanized mouse model market sizing Humanized mouse model market forecast Humanized mouse model market industry analysis"

Read the full report: https://www.reportlinker.com/p05048523/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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The Global Humanized Mouse Model Market is expected to grow by $ 74.72 million during 2020-2024 progressing at a CAGR of 12% during the forecast...

The following discussion and analysis should be read together with our unauditedcondensed consolidated financial statements and related notes thereto set forthin this Quarterly Report on Form 10-Q as well as our Annual Report on Form 10-Kfor the year ended December 31, 2019.This Form 10-Q contains "forward-looking statements" that indicate certain risksand uncertainties, many of which are beyond our control. Actual results coulddiffer materially and adversely from those anticipated in such forward-lookingstatements as a result of certain factors, including those set forth below andelsewhere in this report. Important factors that may cause actual results todiffer from projections include: We may not be able to continue operating without additional financing; Current negative operating cash flows; The terms of any further financing, which may be highly dilutive and may include onerous terms; Significant debt repayments due between June and September 2020, which the Company will likely need to extend or

restructure, with

associated with

pandemic, which

Our precision medicine business, conducted in our Helomics division, iscommitted to improving the effectiveness of cancer therapy using ourproprietary, multi-omic tumor profiling platform, one-of-a-kind database ofhistorical tumor data, and the power of AI to build predictive models of tumordrug response.

Contract Research Organization (CRO) and AI-Driven Business

Patient enrichment & selection for trials

Clinical trial optimization

Via our Helomics subsidiary, we offer a group of clinically relevant,cancer-related tumor profiling and biomarker tests for gynecological cancersthat determine how likely the patient is to respond to various types ofchemotherapy and which therapies might be indicated by relevant tumorbiomarkers.

Skyline Medical - The STREAMWAY System

STREAMWAY System Product Sales

We sell our medical device products directly to hospitals and other medicalfacilities using employed sales representatives, independent contractors anddistributors.

Comparison of three-month periods ended March 31, 2020 and March 31, 2019

Operations expense. Operations expense primarily consists of expenses related toproduct development and prototyping and testing in our current stage.

Liquidity and Capital Resources

Net cash used in operating activities was $2,972,981 and $2,048,653 for thethree months ended March 31, 2020 and March 31, 2019, respectively. Cash used inoperating activities increased in the 2020 period primarily because of theincrease in total operating expense and the additional costs related to theHelomics business.

Liquidity, Plan of Financing, and Going Concern Qualification

We have funded our operations through a combination of debt and equityinstruments including short term borrowings, and a variety of debt and equityofferings.

February 2020 Convertible Note

March 2020 Private Placement of Common Stock and Warrants

March 2020 Amendments to and Extensions of Promissory Notes

April 2020 Paycheck Protection Program

May 2020 Registered Direct Offering of Common Stock and Concurrent PrivatePlacement of Warrants

Off-Balance Sheet Arrangements

We have not engaged in any off-balance sheet activities as defined in Item303(a)(4) of Regulation S-K.

Accounting Standards and Recent Accounting Developments

See Note 1 - Summary of Significant Accounting Policies to the unaudited,Condensed Consolidated Financial Statements of this Quarterly Report on Form10-Q for a discussion of recent accounting developments.

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Chev

But the Original Jews did NOT challenge Jesus, it was Jews such as the National group that stemmed from a movement prior to the time of Jesus. The Hellene Movement. So, Jesus was challenged by mixed-bred movement of people that were elevated above the Original Jews!!!

Therefore, they were probably telling the truth in that they had never been oppressed in Egypt.

YOU DO NOT UNDERSTAND WHAT THE PROBLEM WAS DURING THAT TIME! Geez!

You shouldn't be so quick to assume what I do and do not understand....lol.

I know there was a struggle between the Sadducees and Pharisees during the time of Jesus.What you're calling the Hellene Movement for the Jews to become more like the Greeks was led by the Pharisees -who were Persian overseers sent to Jersusalem to monitor the Jews when they returned from exile.

The Sadducees (sons of Zadok) wanted to maintain the religion the Moses gave them when he civilized them but the Pharisees wanted to move them more in line with the rest of the Caucasians called Gentiles or Greeks.

Get outta here! That was the book you used as a reference

Go back and look at my quotes.You will find NO QUOTE of me referencing a "Hebrew Standard Version".

I say there is the Hebrew Bible, and the various English translations like King James, American Revised, ect.....

The date of that translation was hundreds of years prior to the scriptures translated just before the time of the CRUSADES, of which became the basis for the

KJV which was translated by Hebrew Israelites.

YOur version is NOT received by Christians today for specific reasons.

But, I will add that I do know some of the terms you referenced is in an Ethiopian version [GEEZ]. But however, scholars today

do NOT agree with those terms for major reasons. and today, the people speak Amharic.

I'm not sure what you mean by "my" version.I don't have too much faith in ANY of them.....lol.But when discussing (or in this case arguing...lol) the Bible I like to get the PURIST source possible to find out exactly what the authors meant, so I usually used the HEBREW Bible.

I haven't quoted from the Hebrew Bible on this site because I have to post the text of it along with the translation and I'm not sure how to do that here.The texts is in both Hebrew and English letters.But that's the one I read from and use the most.

No. The very reference you quoted states that LOT was deceived; he had no idea.

OK?Whether he KNEW it or not, INCEST was still performed!

I said according to the Bible INCEST was commited, I didn't say Lot was willing to do it or started it or loved it or enjoyed it or was tricked into it....I said according to the Bible he DID it.And again, you can't find ANY scriptures where the Kenaani, Kemeti, Sumarian, or other Original peoples committed incest......drunk or sober.

Today, there are numerous findings that attest to the violence in that land byway of the Canaanites. They were BAAL PRIEST, that means HEADHUNTERS

Baal was a Kenaani deityZedek was anotherSalem (of which Jersusalem was named after) was another.

Most of these deities had "priests" or "Kohens" who served them in their dedicated temples.That doesn't mean they were killing people or cutting off heads for them.Melchizedek of the Bible was a Canaanite priest who came out to meet Abraham, was HE a head hunter?

As I told you before, the Kenaani were a very sexual people and this usually means violence was kept to a minimum.What credible source can you reference to say that the priests of Baal were headhunters?

The only record we have of people KILLING others and even chopping off heads (in the case of David) for their "god" are the Israelites who went around slaughtering the Original peoples of the land in the name of Yahweh....according to the Bible.

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Troy

You are equating Black primarily with skin color, with no real relationship to culture.

Exactly

Still, it is not clear given your definition why Obama is not Black. He is basically the same complexion as I am; am I not Black?

I've seen only limited picturesof you but you don't look Obama's complexion to me.Obama is a light brown complexion and you seem to be medium brown.

Also, his features are different than yours.His lips are thinner and his head is more "angular"....larger dome with a smaller jaw line...like most Europeans.

Like most AfroAmericans I'm sure you're mixed with White and Native American ancestry from way back, but sinceyour African features dominate I consider you Black.You don't have to be 100% just to be of a certain race, just be predominant in those traits.

But Obama's African features don't "dominate".....he's pretty even between both races phenotypically speaking.

Look Troy, the thing we must keep in mind is the fact that while Obama is DIRECTLY mixed....meaing one of his parents is actually White....many so-called "Black" people in the United States are INDIRECTLY mixed. Meaning they have a lot of White (and Native American) ancestry and it comes out in their complexion and facial features just like his does.

So when you point to another AfroAmericans and say:"Well THEY are Black andObama looks like THEM so Obama must be Black too!"

Really, all you're doing is comparing 2 mixed raced people!NEITHER ONE is truly"Black" racially speaking but the only difference is one has an immediateWhite parent and the other has Whites ancestorsfurtherback.

Which leads me to my NEXT response....................

My mother and sister are much lighter than Obama, are they also not Black? Let me know

I don't know, I would have to look at them to make that judgment.As I said before, although complexion/skin color is one of the BIGGEST factors in determining race, it's not the ONLY one....other features go into it.

There are groups of Africans in southern Africa known as San and Khoi people who are YELLOW in complexion with slanted eyes but are still "Black" racially speaking.

Speaking of southern Africa.........

If you're sitting back shaking your head and laughing at what I'm saying -Just remember that in South Africa you have millions of people just like us here in the United States but unlike us they all don't consider themselves "Black".

One group calls themselves Black and the other call themselves "Colored".Now the Coloreds look just like many AfroAmericans, light skinned with curly hair and some European features but they don't consider themselves Black!

So don't trip or think it's so ridiculous at my suggesting that many fellow AfroAmericans like Beyonce or Al B Sure or even Prince shouldn't be considered "Black",if this were any other nation besides the United States....trust me they probably wouldn't consider THEMSELVES Black either!

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what does it mean to be black - Culture, Race & Economy ...

Writing for children is a joyful endeavor. It takes heart, passion, and one endearing story that yearns to be told. As with all things, commit to putting your own ideas on paper. A story left untold will never blossom or touch the heart of a reader. Christine Taylor Butler

Christine Taylor Butler grew up in Ohio surrounded by books and literature. She developed an overwhelming passion for reading and writing at an early age that defined her purpose and who she has become today. She read every opportunity that she got and even made up story filled adventures of her own. Sadly even though she was a gifted and prolific writer she was missing one ingredient in order to step out on her dreams of becoming a writer, and that was courage. Fear gripped her heart and to alleviate the fear of stepping out she became preoccupied with other productive tasks. Some entail a New England boarding school, two MIT degrees (Engineering and Art & Design)and a series of jobs that included working for a start-up software company. Following thereafter was several years at Harvard University and more than a decade as a Graphic Arts Manager at Hallmark Cards. Although she was productive the overwhelming desire to write kept knocking on the doors of her heart until she eventually opened up.

One of the main reasons for her courageously stepping out in pursuit of a career as an author was the love that her two daughters had for reading childrens books. But it saddened her to see their urban peers did not. She came to the conclusion that sadly multicolored children dont see themselves as being relevant in todays society or as having the potential to make a difference. And even though more books are now like never before presenting children of color in a more positive light Christine wanted to really ensure that this continued. As a result of her lending her voice and being an agent of change her writing career soared. Since then she has released 65 commercially published books.(2018)

Christine Taylor Butler is a force to be reckoned with. She genuinely enjoys the journey of childrens book writing and prides in the fact that she was courageous enough to answer the call to write. She has received a countless amount of awards, ranging from Best Childrens Book of the year by Bank Street College Of Education. Best Books Of The Year by Nebraska Library Association, Best Childrens Book of the year by Barnes and Noble Review. As well as Best Multicultural Books list just to name a few. Her accolades attest to the fact that writing, inspiring and captivating the hearts of children through writing is what she was destined to do.

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