Category Archives: Transhuman News

ALISO VIEJO, Calif., Feb. 10, 2021 /PRNewswire-PRWeb/ -- RARE-X today announced three new board members who will help support the nonprofit's work in structured patient data collection, responsible data sharing, and the promise of its Federated Data Sharing Platform for data sharing and analysis. The new board members are Cynthia Grossman, PhD, director at Biogen; Jason Colquitt, CEO of Across Healthcare; and Simon Frost, CEO of Tiber Capital Group.

"The additions of Cynthia Grossman, PhD, Jason Colquitt, and Simon Frost to the board are very strategic. All bring a depth of knowledge in patient advocacy, health tech, scaling-up organizations, and operational excellence," said Nicole Boice, RARE-X Co-Founder/Executive Director. "We are honored to have them join an already extraordinary board and thrilled to channel their expertise, talent, and energy into helping RARE-X build towards the future."

Cynthia Grossman, PhD, is a director at Biogen, leading the MS PATHS program, a collaborative research network aimed at generating evidence to improve outcomes for patients living with Multiple Sclerosis. Prior to joining Biogen, Cynthia was director at FasterCures, a center of the Milken Institute. Before joining FasterCures, she was chief of the HIV Care Engagement and Secondary Prevention Program in the Division of AIDS Research (DAR) at the National Institute of Mental Health (NIMH). Cynthia has spent her career working to improve health by expanding opportunities for patients' perspectives to shape the processes by which new therapies are discovered, developed, and delivered. Cynthia graduated Phi Beta Kappa from Earlham College with a B.A. in psychology and biology and earned her Ph.D. in clinical psychology from the University of Vermont. She has been the recipient of a National Science Foundation Incentives for Excellence Scholarship, an NIH Ruth L. Kirschstein National Research Services Award, and a Postdoctoral Fellowship in Pediatric Psychology at the Warren Alpert Medical School of Brown University.

Jason Colquitt is CEO of Across Healthcare, a company he founded in 2012, leveraging his 20+ years in the healthcare technology field. His work has caused positive disruption within the healthcare industry as he has partnered with many organizations ranging from small start-ups to some of the world's largest health companies including Greenway Health, Walgreens Boots Alliance, Quintiles, IQVIA, Cystic Fibrosis Foundation, Muscular Dystrophy Association, American College of Surgeons, and American Heart Association. Jason has worked directly with patients, caregivers, physicians, regulators, and researchers. Jason was diagnosed with Carnitine Palmitoyltransferase II Deficiency (CPT II), a rare mitochondrial disease. He has used his experiences and technical background to help the rare disease community. Jason holds a Bachelor's degree in Applied Mathematics from Auburn University.

Simon Frost is the CEO of Tiber Capital Group. Before joining Tiber Capital Group, he was the chief investment officer of Greencourt Capital, a public company with approximately $1 billion in real estate assets. Before joining Greencourt Capital, Simon was president and COO of Key Properties. He was also the co-founder of The American Home, one of the largest single-family rental aggregators in the United States. Simon holds Bachelor's and Master's degrees in economics from Cambridge University in England, and a Bachelor's degree in finance from the University of South Africa. Simon serves as director of both Cure AHC and Hope For Annabel, charities dedicated to finding therapies for Alternating Hemiplegia of Childhood.

The current RARE-X Board of Directors includes: Betsy Bogard, head of program and alliance management within the 4:59 Initiative at 5AM Ventures; Nicole Boice, co-founder and executive director of RARE-X; Jason Colquitt, CEO of Across Healthcare; Wendy Erler, vice president of Patient Experience, STAR and Advocacy at Alexion Pharmaceuticals; Simon Frost, CEO of Tiber Capital Group; Peter Goodhand, CEO of Global Alliance for Genomics and Health; Cynthia Grossman, PhD, director at Biogen; Walt Kowtoniuk, PhD, COO of MOMA Therapeutics and venture partner at Third Rock Ventures; Craig Martin, president of Rithm Health and interim CEO at Global Genes; Katherine Maynard, principal at PWR; Angeli Moeller, PhD, head of Pharma Informatics International at Roche; David Pearce, PhD, president of Innovation and Research for Sanford Health; Anthony Philippakis, MD, PhD, chief data officer at Broad Institute; John Reynders, PhD, chief data scientist at Reynders Consulting; Morrie Ruffin, co-founder and board member of ARM Foundation for Cell and Gene Medicine and managing partner, Adjuvant Partners; Alvin Shih, MD, president and CEO at Catamaran Bio.

ABOUT RARE-X

RARE-X is a 501(c)(3) patient advocacy organization focused on supporting the acceleration and development of life-altering treatments and future cures for patients impacted by rare disease. Enabled by best-in-class technology, patients, researchers, and other technology vendors, RARE-X will gather structured, fit-for-purpose data to share broadly, benefitting from 21st-century governance, consent, and federated data sharing technology. RARE-X is building the largest collaborative patient-driven, open-data access project for rare diseases globally. For more information, visit http://www.rare-x.org.

Media Contact:

Tom Hume, Marketing Communications RARE-X

tomh@rare-x.org

Media Contact

Tom Hume, RARE-X, 7602144863, tomh@rare-x.org

SOURCE RARE-X

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RARE-X Announces the Expansion of its Board of Directors to Support the Organization's Growth and Launch Efforts - WFMZ Allentown

Key Points:Question: Can a genomic biomarker estimate the risk of prostate cancer clinical endpoints in men who received salvage radiation for rising prostate-specific antigen levels after surgery?

Findings: In this ancillary study of 352 men randomized to placebo or hormone therapy in the NRG/RTOG 9601 clinical trial of salvage radiation, the Decipher genomic classifier was independently associated with the risk of metastasis, prostate cancerspecific mortality, and overall survival.

Meaning: These findings suggest that the Decipher genomic classifier is a promising biomarker to risk stratify men to better enable hormone therapy treatment decisions for biochemical recurrence of their prostate cancer after surgery.

Abstract:Importance: Decipher (Decipher Biosciences Inc) is a genomic classifier (GC) developed to estimate the risk of distant metastasis (DM) after radical prostatectomy (RP) in patients with prostate cancer.

Objective: To validate the GC in the context of a randomized phase 3 trial.

Design, Setting, and Participants: This ancillary study used RP specimens from the phase 3 placebo-controlled NRG/RTOG 9601 randomized clinical trial conducted from March 1998 to March 2003. The specimens were centrally reviewed, and RNA was extracted from the highest-grade tumor available in 2019 with a median follow-up of 13 years. Clinical-grade whole transcriptomes from samples passing quality control were assigned GC scores (scale, 0-1). A National Clinical Trials Networkapproved prespecified statistical plan included the primary objective of validating the independent prognostic ability of GC for DM, with secondary endpoints of prostate cancer-specific mortality (PCSM) and overall survival (OS). Data were analyzed from September 2019 to December 2019.

Intervention: Salvage radiotherapy (sRT) with or without 2 years of bicalutamide.

Main Outcomes and Measures: The preplanned primary endpoint of this study was the independent association of the GC with the development of DM.

Results: In this ancillary study of specimens from a phase 3 randomized clinical trial, GC scores were generated from 486 of 760 randomized patients with a median follow-up of 13 years; samples from a total of 352 men (median [interquartile range] age, 64.5 (60-70) years; 314 White [89.2%] participants) passed microarray quality control and comprised the final cohort for analysis. On multivariable analysis, the GC (continuous variable, per 0.1 unit) was independently associated with DM (hazard ratio [HR], 1.17; 95% CI, 1.05-1.32; P = .006), PCSM (HR, 1.39; 95% CI, 1.20-1.63; P < .001), and OS (HR, 1.17; 95% CI, 1.06-1.29; P = .002) after adjusting for age, race/ethnicity, Gleason score, T stage, margin status, entry prostate-specific antigen, and treatment arm. Although the originally planned analysis was not powered to detect a treatment effect interaction by GC score, the estimated absolute effect of bicalutamide on 12-year OS was less when comparing patients with lower vs higher GC scores (2.4% vs 8.9%), which was further demonstrated in men receiving early sRT at a prostate-specific antigen level lower than 0.7 ng/mL (7.8% vs 4.6%).

Conclusions and Relevance: This ancillary validation study of the Decipher GC in a randomized trial cohort demonstrated association of the GC with DM, PCSM, and OS independent of standard clinicopathologic variables. These results suggest that not all men with biochemically recurrent prostate cancer after surgery benefit equally from the addition of hormone therapy to sRT.

Trial Registration ClinicalTrials.gov identifier: NCT00002874

Authors: Felix Y. Feng, MD; Huei-Chung Huang, MA; Daniel E. Spratt, MD; Shuang (George) Zhao, MD; Howard M. Sandler, MD; Jeffry P. Simko, MD, PhD; Elai Davicioni, PhD; Paul L. Nguyen, MD; Alan Pollack, MD, PhD; Jason A. Efstathiou, MD, PhD; Adam P. Dicker, MD, PhD; Tamara Todorovic, MSc; Jennifer Margrave, BSc; Yang (Seagle) Liu, PhD; Bashar Dabbas, MD; Darby J. S. Thompson, PhD; Rajdeep Das, MD, PhD; James J. Dignam, PhD; Christopher Sweeney, MD; Gerhardt Attard, PhD; Jean-Paul Bahary, MD; Himanshu R. Lukka, MD; William A. Hall, MD; Thomas M. Pisansky, MD; Amit B. Shah, MD; Stephanie L. Pugh, PhD; William U. Shipley, MD; Phuoc T. Tran, MD, PhD

Department of Radiation Oncology, UCSF Medical Center, San Francisco, California (Feng, Das); Department of Medicine, UCSF Medical Center, San Francisco, California (Feng, Das); Department of Urology, UCSF Medical Center, San Francisco, California (Feng, Das); Decipher Biosciences, San Diego, California (Huang, Davicioni, Todorovic, Margrave, Liu, Dabbas); Department of Radiation Oncology, University of Michigan, Ann Arbor (Spratt, Zhao); Department of Radiation Oncology, Cedars-Sinai Medical Center, Los Angeles, California (Sandler); NRG Biorepository, Department of Pathology, UCSF Medical Center, San Francisco, California (Simko); Department of Radiation Oncology, Brigham and Women's Hospital, Boston, Massachusetts (Nguyen); Department of Radiation Oncology, University of Miami Miller School of Medicine, Miami, Florida (Pollack); Department of Radiation Oncology, Massachusetts General Hospital, Boston, Massachusetts (Efstathiou, Shipley); Department of Radiation Oncology, Thomas Jefferson University Hospital, Philadelphia, Pennsylvania (Dicker); Emmes Canada, Vancouver, British Columbia, Canada (Thompson); NRG Oncology Statistics and Data Management Center, Philadelphia, Pennsylvania (Dignam, Pugh); Department of Public Health, University of Chicago, Chicago, Illinois (Dignam); Department of Medicine, Dana-Farber/Harvard Cancer Center, Boston, Massachusetts (Sweeney); Department of Oncology, University College London, London, United Kingdom (Attard); Department of Radiation Oncology, Centre Hospitalier de lUniversit de Montral-Notre Dame, Montreal, Quebec, Canada (Bahary); Department of Radiation Oncology, Juravinski Cancer Centre at Hamilton Health Sciences, Hamilton, Ontario, Canada (Lukka); Department of Radiation Oncology, Froedtert and the Medical College of Wisconsin, Madison, Wisconsin (Hall); Department of Radiation Oncology, Mayo Clinic, Rochester, Minnesota (Pisansky); Department of Radiation Oncology, WellSpan Health-York Cancer Center accruals under Thomas Jefferson University Hospital, Philadelphia, Pennsylvania (Shah); Department of Radiation Oncology, Johns Hopkins University, Baltimore, Maryland (Tran); Department of Oncology, Johns Hopkins University, Baltimore, Maryland (Tran); Department of Urology, Johns Hopkins University, Baltimore, Maryland (Tran).

Source: Feng F, Huang HC, Spratt D et al."Validation of a 22-Gene Genomic Classifier in Patients With Recurrent Prostate Cancer An Ancillary Study of the NRG/RTOG 9601 Randomized Clinical Trial." JAMA Oncology. 2021.2020.7671.

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Genomic Test Helps Guide Precision Medicine to Estimate Risk of Prostate Cancer Metastasis, Death

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Validation of a 22-Gene Genomic Classifier in Patients With Recurrent Prostate Cancer An Ancillary Study of... - UroToday

BOSTON--(BUSINESS WIRE)--Ensoma, a company expanding the curative power of genomic medicine by pioneering a next-generation in vivo approach, today launched with a $70 million Series A financing led by co-founder and seed investor 5AM Ventures, with participation from F-Prime Capital, Takeda Ventures, Viking Global Investors, Cormorant Asset Management, RIT Capital Partners, Symbiosis II, LLC, and Alexandria Venture Investments. In addition to an equity investment of $10 million in the Series A financing, Takeda Pharmaceutical Company Limited (Takeda) and Ensoma have entered into a strategic collaboration with the potential for upfront and preclinical research payments totaling $100 million as part of a strategic collaboration worth up to $1.25 billion, announced in a separate press release this morning.

The foundation of the companys platform its Engenious vectors is based on over two decades of academic and clinical research generated by scientific co-founders and renowned experts, Hans-Peter Kiem, M.D., Ph.D., of Fred Hutchinson Cancer Research Center, and Andr Lieber, M.D., Ph.D., of University of Washington School of Medicine. The company will be led by biotechnology industry veterans with demonstrated track records in innovative therapeutic modalities, including gene therapy and editing, across an array of disease areas, including rare disease, hematology and oncology.

Ensomas Engenious vectors are designed to deliver a diverse range of genome modification technologies including those that require a high level of packaging capacity directly to hematopoietic stem cells (HSCs) or the various cell types that arise from these cells, such as T cells, B cells and myeloid cells. The companys vectors are optimized to work without the need for stem cell collection or prior myeloablative conditioning (e.g., chemotherapy). As a result, Ensomas therapies will be designed to be delivered via single injection in diverse environments, including outpatient and areas where access to sophisticated healthcare systems may be limited.

With the launch of Ensoma, we aspire to bring innovative new treatments to patients in a way that is accessible for all, said Paula Soteropoulos, executive chairman of Ensoma. Because our in vivo therapies do not require prior conditioning or stem cell donors, we hope to deliver them as off-the-shelf treatments to address diseases both rare and common dramatically simplifying the logistics of scaling production and reducing patient and healthcare-system burden. Every person, no matter where they are in the world, should have access to the innovative technologies that are changing the way we treat disease.

Engenious Vectors

Ensomas Engenious vectors are specially engineered adenovirus vectors devoid of any viral genome and minimal pre-existing immunity, thus minimizing the chance of an immune response and freeing up ample storage space up to 35 kilobases (kb) of DNA packaging capacity to deliver a diverse range of genome modification technologies. Also known as therapeutic cargo, these technologies may include, separately and in combination, the following:

These approaches enable Engenious vectors to engineer various erythroid, lymphoid (e.g., T cells, B cells) and myeloid (e.g., macrophages, microglia) cell types, with great precision and vast therapeutic potential. Addressable indications range from rare monogenic diseases to broader diseases such as oncology, autoimmunity and infectious diseases via precision, off-the-shelf engineering of the immune system.

Given the highly specific nature of these technologies, Ensomas Engenious vectors enable preferential targeting of HSCs inside the body. Additionally, Ensomas founders have developed an in vivo selection system that can increase the population of genetically modified HSCs, if needed. This proprietary approach enables precise titration to lasting therapeutic levels without the need to re-dose patients, bypassing the immunogenic challenges associated with re-dosing for some other gene therapy modalities.

Ensomas Engenious platform has been extensively validated in numerous preclinical models with a range of genome editing technologies, demonstrating robust genetic modification of bone marrow HSCs and stable long-term expression of therapeutic proteins in small and large preclinical models.

There have been tremendous advancements in technologies to precisely target, genetically edit and modify human disease. However, many of these tools pose delivery challenges; some lack the ability to reach the right cells within the body, while others lack the ability to broadly reach significant numbers of patients due to complex procedures and supply chain challenges, said Kush M. Parmar, M.D., Ph.D., founding chief executive officer of Ensoma. Ensomas scientific approach allows us to do what hasnt been done beforeto make the curative power of genomic medicine and stem cell technology portable so they may be administered in low-resource and outpatient settings for the very first time.

Leadership & Scientific Founders

Ensoma was founded by and incubated within the 4:59 Initiative, the company creation engine of 5AM Ventures. The companys scientific co-founders include Dr. Hans-Peter Kiem, an oncologist and world-renowned pioneer in gene-editing technologies, including stem cell and gene therapies, from Fred Hutch, who also serves as vice president of the American Society of Gene & Cell Therapy and chief scientific and clinical advisor for Ensoma; and Dr. Andr Lieber, an accomplished academic researcher and professor of medicine, Division of Medical Genetics, UW School of Medicine, who has studied the biological and translational aspects of human adenoviruses for more than two decades. Ensoma is based on an exclusively licensed portfolio of technologies developed by the Fred Hutch lab of Dr. Kiem and the University of Washington lab of Dr. Lieber that enable in vivo genome engineering and gene therapy advances of HSCs for therapeutic use in blood diseases.

Following more than 20 years of academic and clinical research, Ensoma has assembled an exceptional team to boldly forge a new era of genomic medicine in vivo, said Bihua Chen, founder and portfolio manager at Cormorant Asset Management. The company is moving swiftly to accelerate and broaden the therapeutic potential of its approach, and I am confident they have the right team and the right technology to potentially bring life-changing, curative therapies within reach for people all over the world.

Additional details surrounding company leadership, including its board of directors, are as follows:

Ensoma has also named its scientific advisory board, which may be viewed here.

About 5AM Ventures

Founded in 2002, 5AM actively invests in next-generation biotech companies. With approximately $1.5 billion raised since inception, 5AM has invested in 89 companies. For more information, please visit http://www.5amventures.com.

About the 4:59 Initiative

The 4:59 Initiative is the internal company creation engine at 5AM Ventures that helps discover, incubate, and fund breakthrough science. The 4:59 team provides hands-on scientific, strategic, and operational support, working closely with academics and entrepreneurs to advance breakthrough science and establish proof-of-concept data to enable a clear path to transformative therapies for patients.

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Ensoma Launches to Pioneer Next-Generation In Vivo Approach to Deliver First Off-the-shelf Genomic Medicines - Business Wire

LONDON and LAUPHEIM, Germany, Feb. 11, 2021 (GLOBE NEWSWIRE) -- The Cell and Gene Therapy Catapult (CGT Catapult), an independent centre of excellence in innovation advancing the UKs cell and gene therapy industry, and Rentschler Biopharma SE, a leading global contract development and manufacturing organisation (CDMO) for biopharmaceuticals, have announced today that Rentschler Biopharma will establish their manufacturing capability in Advanced Therapy Medicinal Products (ATMPs), including Adeno-Associated Virus (AAV) Vectors for clinical trial supply, at the CGT Catapult site in Stevenage.

Under the terms of the agreement, Rentschler Biopharma will make a significant investment at the site over the next five years to set up their manufacturing capabilities, benefitting from the expertise and unique collaborative model provided by the CGT Catapult. The companys investment is expected to make a major contribution to meeting the demand from UK and international researchers for suitable manufacturing capability. This development will further strengthen the UK ecosystem through the addition of Rentschler Biopharmas more than 40 years of experience and solid reputation in the development and manufacturing of biologics for both clinical and commercial supply. The company will leverage the CGT Catapults expertise in ATMP manufacturing setup and technology development, as well as the cell and gene therapy cluster and ecosystem that has developed around Stevenage and across the UK.

Dr. Frank Mathias, CEO of Rentschler Biopharma, said:We are excited to take this next big step in our evolution and address the growing industry demand for ATMP manufacturing capacity and viral vector supply. With the largest industry cluster for cell and gene therapies outside the US, the UK is an ideal location for us to establish our Center of Excellence for cell and gene therapy. We look forward to working with the CGT Catapult as we invest in this growing field. They are well established in this important market, enabling us to immediately tap into the organisations network and utilisethe UKs strong expertise and supply chain in cell and gene therapy manufacturing.

Matthew Durdy, CEO of the Cell and Gene Therapy Catapult, commented:We are very pleased that Rentschler Biopharma, a global CDMO, has chosen to build their ATMP capacity in the UK, bringing in their expertise and investment. This will build new capacity to benefit the international ATMP supply chain and meet growing academic and commercial demand across the industry. As more companies from around the globe come to the UK, it demonstrates and enhances the attractiveness of its cell and gene therapy ecosystem as a place to develop new technologies and capabilities.

The investment in the UK cell and gene therapy industry announced today is expected to further accelerate the development of the vital infrastructure and skilled jobs needed to meet the rising demand for manufacturing capacity in the UK and globally, as well as streamline the supply chain for these advanced therapies. Currently, 27% of European ATMP companies are operating in the UK, and there are more than 90 advanced therapy developers. The last year has also seen a 50% increase in the number of ATMP clinical trials being run in the UK, accounting for 12% of global ATMP clinical trials, and these numbers are predicted to increase further.

The CGT Catapult manufacturing centre has been backed by over 75m of funding, including investment from the UK Governments Industrial Strategy Challenge Fund, the Department for Business, Energy and Industrial Strategy, Innovate UK and from the European Regional Development Fund. Since it was announced, there has been over 1.1bn of investment in the ATMP industry in its vicinity.

About Rentschler Biopharma SE

Rentschler Biopharma is a leading contract development and manufacturing organization (CDMO), focused exclusively on client projects. The company offers process development and manufacturing of biopharmaceuticals as well as related consulting activities, including project management and regulatory support. Rentschler Biopharma's high quality is proven by its long-standing experience and excellence as a solution partner for its clients. A high-level quality management system, a well-established operational excellence philosophy and advanced technologies ensure product quality and productivity at each development and manufacturing step. In order to offer best-in-class formulation development along the biopharmaceutical value chain, the company has entered into a strategic alliance with Leukocare AG. Rentschler Biopharma is a family-owned company with about 1,000 employees, headquartered in Laupheim, Germany, with a second site in Milford, MA, USA. In Stevenage, UK, Rentschler Biopharma launched a company dedicated to cell and gene therapies, Rentschler ATMP Ltd.

For further information, please visit http://www.rentschler-biopharma.com. Follow Rentschler Biopharma on LinkedIn and Facebook.

About the Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 330 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK.

For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

About the European Regional Development Fund

This project has received 3.36m of funding from the England European Regional Development Fund as part of the European Structural and Investment Funds Growth Programme 2014-2020. The Ministry of Housing, Communities and Local Government (and in London the intermediate body Greater London Authority) is the Managing Authority for European Regional Development Fund. Established by the European Union, the European Regional Development Fund helps local areas stimulate their economic development by investing in projects which will support innovation, businesses, create jobs and local community regenerations. For more information visit https://www.gov.uk/european-growth-funding.

About the Industrial Strategy Challenge Fund

This project has received 12m of funding from the Industrial Strategy Challenge Fund, part of the governments modern Industrial Strategy. The Industrial Strategy Challenge Fund is a four-year, 1 billion investment in cutting-edge technology designed to create jobs and improve living standards, built on guidance from business and the academic community. Healthcare and Medicine is one of three core areas for investment under the programme.

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Rentschler Biopharma to build new cell and gene therapy capabilities in the UK - BioSpace

If theres one thing Ive learned over the years as a health and wellness writer, its that information is power. The flip side of that is the fact that not having key information available to you can be deeply disempowering. Like millions of other Americans, Im adopted, which means I havent been able to find out a lot about important health information that most people have readily available to them: family health history and genetic health information.

Family health history is essentially just that: knowing the health histories of members of your biological family. This kind of information can help doctors pinpoint whether you are at risk for certain health conditions that can run in families or be determined by genetics. Family history is a strong clue for chronic disease risks you may face, such as heart disease, stroke, cancer, and diabetes, says Latha Palaniappan, MD, the scientific director of Genomics and Pharmacogenomics in Primary Care at Stanford Medicine. The Centers for Disease Control and Prevention (CDC) CDC recommends documenting as much as you can about your familys health history in order to share with your doctor, and ask for additional testing if youre concerned about your risk for a specific disease.

While Ive always valued a healthy lifestyleI try to eat well, sleep enough, exercise, and manage stress as much as possibleIve wondered recently if my inclination towards healthy living has been driven in part by fear, specifically the fear of what I dont know about my health and genetics. Since I dont know what could be in my genes, at least I do have some control over my lifestyle now, and that counts for a lot, right?

Thankfully, Dr. Palaniappan assures me that family history is not the end-all, be-all of what will happen with your health.Family history is probabilistic, not predictive, she says. (Basically, it can educate you about your odds of experiencing a certain health outcome, but not predict it outright.) But if you do have access to that information, use it, since family history provides important clues about your health risks, says Dr. Palaniappan.

So if you dont have access to this information, should you be worried? And what else can you do, besides actually going out to try to find your biological relatives information (which is a hugely personal choice, and not possible for some)? There are some other things you can do to help you gather more information about your health and feel more empowered about your future.

Honestly, I didnt think about my family health history too much until I started approaching 30. As the mystery surrounding family health information came up a bit more for me, I talked to my mom and my sister about my concerns surrounding what we dont know. When my mom got me a 23andMe DNA test (which start at $199 for the Health + Ancestry test) for Christmas one year, I was excitedand kind of anxiousto have the chance to take a deeper look into my health information.

23andMe is just one example of a direct-to-consumer (DTC) DNA test that can give you some more information about your health. According to the companys website, the health reports available with the test include genetic information that can clue you in to your genetic risk for conditions like type 2 diabetes, select variants of BRCA1/BRCA2 (the gene associated with breast, ovarian, and pancreatic cancer), celiac disease, uterine fibroids, and more. The brands test can tell you about your carrier status (meaning if you carry genes linked to an inherited disease that could affect your children) for some diseases like cystic fibrosis and sickle cell anemia.

However, these DTC tests dont often come with specific consultation to walk you through whats present in your genome and how that translates into actual risk. Thats why its important to work with a genetics expert or genetic counselor if you can, says Robert C. Green MD, a medical geneticist who leads the Preventative Genomics Clinic at the Harvard-affiliated Brigham and Womens Hospital, and is the director of the Genomes2People Research Program.You [can] have a geneticist or genetic counselor who basically talks to you about what [the test results] mean and what should you do about it. What should you worry about and what should you not worry about, says Dr. Green. For example, if you tested positive for the gene for a certain hereditary cancer, a genetic counselor can help you with the next steps, like if you should seek more testing or work with a specialist.

Dr. Green adds that DTC tests arent the most comprehensive testing option. Thats because most of them use whats called chip-based DNA technology, which essentially scan your genome for known common mutations or markers along your genome, he says. [This technology] can be very good for ancestry for [finding relatives] and for certain specific markers, such as the Ashkenazi Jewish BRCA1 mutation that 23andMe looks for. It does not look at every letter in your genes, and its not typically set up to find rare or novel mutations that can affect your health. (Theyre not always super accurate, eithera 2019 study found that these chips have a very high false-positive rate for rare genetic mutations.) For health reasons sequencingwhich looks at every letter in a segment of your genome or across the whole genomeis more expensive, but much, much more comprehensive, he says.

DNA testing is definitely not cheap (it can run anywhere from $200 up to $2,000 for the more in-depth testing, and isnt always covered by insurance) and its certainly not the only way to find out more information about your health.

If you dont know much about your family health history, Dr. Palaniappan encourages paying attention to key health markers including blood pressure, cholesterol, glucose, and heart rate, and getting those checked regularly. These measurable risk factors can be effectively treated to reduce your risk of heart disease, stroke and diabetes, says Dr. Palaniappan. Everyone can reduce the risk of disease by eating a healthy diet, getting enough exercise, and not smoking. Cancer screening tests such as mammograms and colorectal cancer screening can detect precancer and treatable cancers early, she says.

While getting the DNA test felt like a great first step to knowing more about my health, its also good to know that the everyday things that I sometimes dont even think about (like walking my dog) might have a bigger impact on my health than I thought before.What you do each and every daywhat you eat, how much you exercise, and your other health behaviors, can ultimately affect your risk of developing disease, says Dr. Palaniappan. If anything, Ive learned that not knowing your family health history doesnt have to be a huge blank spot, but if I ever do want to know more, there are optionswhich is empowering for sure.

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Im 28 and I Dont Know My Family HistoryHeres How That Affects My Health - Well+Good

The changes brought by the COVID-19 pandemic have had an impact across society, and courts and IP offices are no exception. As part of our series of articles looking at what to expect in 2021, Kilburn & Strode attorneys discuss some of the legal developments on the horizon.

Its not just business meetings, webinars and Christmas quizzes that are suited to Zoom; court and IP office hearings are increasingly conducted virtually too.

At the EPO, oral proceedings in examination and oppositions will be heldonlineunless there are exceptional circumstances until September 2021 (at least). Board of Appeal hearings are heading the same way. While some practitioners have been sceptical about these changes, at Kilburn & Strode we see video conferencing as a hugely powerful and accessible medium for getting to a good decision quickly.

This is good news for SMEs, who may now be able to afford to fight cases they couldnt otherwise, says associate Rosie Carrie. But, she adds, the long-term implications are hard to predict: Will parties be more likely to be confrontational when not seeing each other in person? Will they pursue arguments more strongly? Will the opportunity to resolve issues in an informal setting be missed?

At the EUIPO, there may be changes too. Areformin 2019 overlooked by many people at the time means that the CJEU now accepts appeals from the EU General Court only where they raise an issue that is significant with respect to the unity, consistency or development of EU law. In practice, this means the General Court is now the final arbiter for most cases involving EU trade marks and registered Community designs. That may lead to the EUIPO Boards of Appeal holding more oral hearings between parties, something that until now has been extremely rare.

Patent priorities

With normal routines disrupted and the opportunity to reflect, the pandemic has led many people to consider their values and priorities getting fit, learning new hobbies or their social and political values. Policy debates about the balance between competition and innovation, between access and reward; or between control and free speech have always been part of IP. In this new environment, will we see them become even more prominent?

In patents, for example, partnerNick Bassilsays he increasingly sees morality issues being raised regarding patents for stem cells, gene therapy etc, arising from the ordre public (public policy) and morality exception in Article 53(a) of the EPC: This will become more and more relevant with genetic medicine. More generally, debates about access to medicine, the role of patents, pricing mechanisms and sourcing of drugs are gaining attention as the pandemic increases awareness (if not understanding) of the role of medicine (see our separate article on political and social trends).

On your marks

In trade marks, meanwhile, debates are continuing about the proper scope of protection and whether making broad and/or multiple related trade mark applications should be discouraged (including on the grounds of bad faith). These issues were not fully resolved in last yearsSkyKickdecision from the CJEU, but should be addressed further in the MONOPOLY case pending before the EU General Court.

Trade mark partnerIain Stewartsays: These debates reflect wider concerns about the cluttering of trade mark registers. One view is that unlike in the US where there are strict use requirements, in Europe there are too many marks that are registered but not used. It means clearance searches in the EU are becoming a nightmare. Sometimes the results run to dozens of pages, explains Iain. As well as being a practical challenge for those seeking to apply for trade marks, particularly internationally, cluttering raises questions about the accessibility of the trade mark system and its ability to serve all users, especially SMEs.

UPC or not UPC?

The end of 2020 saw Germany take important steps towards ratifying the Unified Patent Court (UPC) Agreement, raising the possibility that it could theoretically come into effect, though significant legal challenges remain (meaning ratification by Germany and implementation in 2021 is unlikely).Gwilym Robertssays: The UPC was always a lofty ideal and the simplification it offers remains attractive. But with the continuing legal challenges it faces one wonders if its time to look at alternative multilateral arrangements bringing the same benefits.Whatever comes out Kilburn & Strode will continue to be a part of it as we continue to expand our European presence.

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How the law will change in 2021 - Lexology

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced multi-year collaborations with Cleveland Clinic and UT Southwestern Gene Therapy Program (UTSW) to advance next-generation mini-gene payloads for AAV gene therapies for the treatment of genetic epilepsies and additional CNS disorders. Taysha will have an exclusive option on new payloads, constructs and intellectual property associated with, and arising from, the research conducted under this agreement.

A team of researchers from Cleveland Clinic Lerner Research Institute will create mini-gene payloads designed to address some of the long-standing limitations in AAV gene therapy. UTSW will create and evaluate vector constructs in in vivo and in vitro efficacy models of genetic epilepsies and additional CNS disorders.

By pushing the boundaries of AAV vector engineering, we may be able to overcome some of the challenges inherent with gene therapy and potentially expand the range of treatable genetic CNS diseases with gene therapies. We appreciate the support from Taysha and UTSW in this work, said Dennis Lal, Ph.D., Assistant Staff at Cleveland Clinic Genomic Medicine Institute and Neurological Institute. We believe that our proprietary approach to overcoming current limitations of packaging capacity and our access to data on thousands of protein structures associated with a whole host of monogenic CNS disorders has the potential to enable a deep pipeline of functioning mini-genes.

Cleveland Clinic and UTSW are two of the worlds preeminent leaders in gene therapy innovation, and this collaboration is designed to leverage our capabilities and synergies with these institutions to pioneer novel approaches to address vector capacity, which is a common limitation when treating genetic disorders associated with large proteins, said Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. We look forward to a productive collaboration with the goal of developing treatments with promising benefits to patients with debilitating genetic epilepsies.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our collaboration with the Cleveland Clinic and UTSW, the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential benefits of rare pediatric disease designation and orphan drug designation to our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

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Taysha Gene Therapies Announces Collaborations to Advance Next-Generation Mini-Gene Payloads for AAV Gene Therapies for the Treatment of Genetic...

Recently demonstrated single-dose intravenous administration of a PATrOL-enabled compound resolves the causal genetic defect in myotonic dystrophy type 1 (DM1) in transgenic animals; Company on course to move one program into clinical development in CY2022

Plans to host an R&D day in the first half of CY2021 to provide updates on platform innovations, the DM1 and Huntington's disease (HD) programs and pipeline expansion in high value indications

Expects to complete consolidation of intellectual property in the space through acquisition of gene modulating technology from Vera Therapeutics in Q1 CY2021

PITTSBURGH, Feb. 11, 2021 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution using a new class of synthetic medicines, today reported its financial results for the three-month period ended December 31, 2020.

"In 2020 we validated that our PATrOL platform technology can deliver compounds that are broadly biodistributed, mutant allele-specific and well tolerated, including in non-human primates (NHPs). Thereafter we finalized screening compound libraries and moved into in vivo efficacy and tolerability studies that demonstrated that administration of a PATrOL-enabled compound resolves the causal genetic insult in an established transgenic animal model of myotonic dystrophy type 1 (DM1), a severe genetic disease with no effective therapies. This momentum is being carried forward into 2021 as we set the stage to enter the clinic in CY2022," said Dietrich A. Stephan, Ph.D., chief executive officer of NeuBase. "Predicated on our progress, we recently announced an agreement to acquire additional gene modulating technology to consolidate the intellectual property to protect and enhance value creation with this unique therapeutic modality."

"We look forward to providing more data and insights during an investor R&D day in the first half of CY2021, including updates on platform innovations, continued progress in Huntington's disease (HD) and DM1 and new pipeline programs. This event will provide an opportunity for us to introduce the expanded team, including Dr. Curt Bradshaw, Ph.D. chief scientific officer, who has overseen several development programs into the clinic and complements a world-class team of technical experts and drug developers."

First Quarter of Fiscal Year 2021 and Recent Operating Highlights

Financial Results for the Fiscal Quarter Ended December 31, 2020

About NeuBase TherapeuticsNeuBase is accelerating the genetic revolution using a new class of synthetic medicines which have been shown to be able to increase, decrease and change gene function, as appropriate, to resolve causal genetic defects in living systems. NeuBase's designer PATrOL therapies are centered around its proprietary drug scaffold to address genetic diseases at the source by combining the highly targeted approach of traditional genetic therapies with the broad organ distribution capabilities of small molecules. With an initial focus on silencing disease-causing mutations in debilitating neurological, neuromuscular and oncologic disorders, NeuBase is committed to redefining medicine for the millions of patients with both common and rare conditions. To learn more, visit http://www.neubasetherapeutics.com.

Use of Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements are distinguished by use of words such as "will," "would," "anticipate," "expect," "believe," "designed," "plan," or "intend," the negative of these terms, and similar references to future periods. These forward-looking statements include, among others, those related to the potential significance and implications of the Company's positive in vitro and in vivo preclinical data for its PATrOL-enabled anti-gene therapies for the treatment of myotonic dystrophy type 1 (DM1), the plan to provide updates on the Company's development pipeline, including the myotonic dystrophy type 1 (DM1) and Huntington's disease (HD) programs, at an R&D day in the first half of CY2021 and the Company's anticipated capital requirements over approximately the next twelve months. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those risk factors contained in our filings with the U.S. Securities and Exchange Commission, may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Company's current expectations, they are subject to various risks and uncertainties, and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Company's plans to develop and commercialize its product candidates; the timing of initiation of the Company's planned clinical trials; the risks that prior data will not be replicated in future studies; the timing of any planned investigational new drug application or new drug application; the Company's plans to research, develop and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of the Company's product candidates; the Company's commercialization, marketing and manufacturing capabilities and strategy; global health conditions, including the impact of COVID-19; the Company's ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors contained in our filings with the U.S. Securities and Exchange Commission. Except as otherwise required by law, the Company 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.

NeuBase Investor Contact:Dan FerryManaging DirectorLifeSci Advisors, LLCdaniel@lifesciadvisors.com OP: (617) 430-7576

NeuBase Media Contact:Cait Williamson, Ph.D.LifeSci Communicationscait@lifescicomms.com OP: (646) 751-4366

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NeuBase Therapeutics Reports Financial Results for the First Quarter of Fiscal Year 2021 - GlobeNewswire