Research Triangle Park, N.C.– November 14, 2022 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly-owned and independently-operated subsidiary of Bayer AG, today announced that Sheila Mikhail, the company’s Co-Founder and CEO, was named one of the “Fiercest Women in Life Sciences 2022” by industry-leading publication Fierce Pharma. Mikhail is one of 20 female leaders in life sciences to receive this honor. Recipients were selected following a rigorous review process that identified women who are leading the way and making a lasting impact on the industry. The inspirational women being recognized are leaving an indelible footprint across biotech, pharma and medtech. Mikhail, who is at the forefront of life science innovation and business leadership, has left an unparalleled mark on the industry as a serial entrepreneur, company builder and one of its few Latina CEOs. “Sheila is an exceptional businesswoman. Her command of the science of gene therapy is only rivaled by her incredible business acumen,” said Jude Samulski, Ph.D., AskBio Co-Founder and Chief Scientific Officer. “What makes her contribution to our industry most remarkable is her ability to create environments for cutting-edge science to thrive. Sheila’s work has directly resulted in breakthrough gene therapies reaching patients and in the development of others that we hope will do the same in the future. This honor is well deserved.” Mikhail co-founded AskBio over 20 years ago with the unwavering belief that gene therapies could cure disease and change the world. During her tenure at the company, she also led several spinoffs where she realized various strategic partnerships and acquisitions. These included Pfizer’s acquisition of Bamboo Therapeutics in 2016 and the acquisition of Chatham Therapeutics by Baxter (now Takeda) in 2014. More recently, Mikhail negotiated the $4 billion acquisition of AskBio and its cGMP manufacturing subsidiaries by Bayer AG in 2020, a reflection of the tremendous value she and co-founder Dr. Samulski have and continue to build with the company. “I’m honored to be recognized by Fierce for my contribution to our industry,” said Mikhail. “I am fortunate to have been part of the gene therapy field over a period where it matured from concepts to real life examples of transformative benefits for patients. In addition to bringing treatments to patients, my priorities have also included ensuring equitable access and creating opportunities to advance women and underrepresented minorities in biotech. As a head of an organization represented by over 50 percent women, with women holding key leadership positions, I am proud that together at AskBio we have created an inclusive environment where women feel they can thrive.” Today, AskBio is a global leader in gene therapy and works to bring innovative technology to the market and potentially life-changing therapeutics into the clinic. As CEO, Mikhail has helped grow the company to over 700 employees operating in five countries. She is also Co-Founder and Board Member of Columbus Children’s Foundation (CCF), which develops gene therapies for children with ultra-rare diseases and treats them free of charge. Mikhail was named the Overall US Entrepreneur of the Year
Category: News
AskBio Co-Founder Jude Samulski Receives Inaugural European Society of Gene and Cell Therapy Founders Award
— Award recognizes 40th anniversary of groundbreaking first cloning of AAV plasmid — Research Triangle Park, N.C.– OCTOBER 11, 2022 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced that R. Jude Samulski, Ph.D., AskBio President, Co-Founder and Chief Scientific Officer, received the inaugural European Society of Gene and Cell Therapy (ESGCT) Founders Award in Edinburgh, Scotland, during the recent ESGCT 29th Congress. The Founders Award honors Dr. Samulski’s contribution to the development of engineered recombinant adeno-associated virus (rAAV) vectors, which began 40 years ago with the cloning and rescue of infectious AAV from a recombinant plasmid.1 This work laid the foundation for subsequent developments of rAAV vectors capable of expressing foreign sequences, thereby enabling the use of AAV-based vectors for the delivery of various therapeutic genes into mammalian cells. The AAV inverted terminal repeat (ITR) sequences are an essential component of every AAV therapeutic drug. This and other breakthroughs made by Dr. Samulski have since contributed to three rAAV-based gene therapies becoming available for patients and many more in various stages of development. “ESGCT created the Founders Award this year to mark the Society’s 30th anniversary,” said Professor Hildegard Büning, ESGCT Board President. “The award highlights benchmark milestones in gene and cell therapy. The Founders Award 2022 recognizes the first cloning of an AAV plasmid, which Dr. Samulski and his team accomplished in 1982. That tremendous breakthrough helped make gene therapy a reality and remains at the core of the field today, making this milestone a natural choice for this inaugural honor.” Dr. Samulski has worked with AAV for 40 years, and for 25 years he was director of the University of North Carolina Gene Therapy Center. Dr. Samulski is a former member of the Recombinant DNA Advisory Committee (RAC), a committee tasked with assisting the FDA with gene therapy clinical trial approvals in the U.S. He also frequently serves as a gene therapy consultant to the FDA. In 2008, Dr. Samulski was recognized by the American Society of Gene & Cell Therapy (ASGCT) with the inaugural Outstanding Achievement Award. He was also invited by Pope Francis to the Vatican in recognition of his work in the treatment of Canavan disease. Dr. Samulski has played an integral role in advancing gene therapies into human clinical trials for hemophilia, Duchenne muscular dystrophy, giant axonal neuropathy, Pompe disease, congestive heart failure and others. He is the inventor of nearly 500 patented technologies related to AAV technology. “I’m overwhelmed with this acknowledgment and humbled by the thought that ESGCT has considered my early research to represent such a prestigious milestone for the society,” said Dr. Samulski. “I’m grateful to receive the ESGCT Founders Award during these exciting times for gene therapy and honored to be the first scientist to receive this recognition.” “Jude developed the foundational technology for making AAV-based gene therapy vectors and self-complementary AAV vectors,” said Sheila Mikhail, CEO and Co-Founder of AskBio. “His groundbreaking work 40 years ago remains central to our industry’s advances in
AskBio Contribution to Advancing Gene Therapy Highlighted at ESGCT 29th Congress
— Company presence includes 2 oral and 12 poster presentations — Research Triangle Park, N.C.– OCTOBER 10, 2022 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced that 14 company abstracts will be presented at the European Society for Gene and Cell Therapy (ESGCT) 29th Congress, which is being held in Edinburgh, Scotland, from October 11 to 14, 2022. The full company presence highlights the breadth of the AskBio contribution to advancing gene therapy and includes an oral presentation on the role of empty capsids in overall AAV immunogenicity and toxicity risks, 12-month data from ongoing Parkinson’s and congestive heart failure studies and data on synthetic DNA material from wholly owned subsidiary TAAV Biomanufacturing Solutions, SL. Additional activities include company experts participating in educational sessions and parallel symposia, as well as a symposium sponsored by wholly owned subsidiary Viralgen. Topics include data-driven vector design for precise control of gene expression and manufacturing to support gene therapy. “AskBio is relentless in its effort to serve as a catalyst for breakthrough innovation in gene therapy,” said Sheila Mikhail, CEO and Co-Founder of AskBio. “ESGCT gives us an opportunity to show how we are doing that in several important ways: driving the science related to capsids and the use of synthetic DNA material, advancing our pre-clinical and clinical stage pipeline and creating opportunities for industry colleagues to exchange knowledge.” Data highlights include: Program Abstract Title Presentation Details/Date Preclinical/R&D Using a systems biology approach to unravel the immunogenicity of AAV8 empty capsids in healthy volunteers Oral presentation (OR06) / October 11 (17:00 – 19:15 BST) Congestive Heart Failure Analysis of vector performance in patient heart biopsy for congestive heart failure provides support for rationally designed capsids Poster P671 / October 12 Preclinical/R&D In vivo evaluation of novel synthetic promoters for CNS gene therapy Poster P037 / October 12 Preclinical/R&D Transcriptomics of suspension 293 cells during AAV vector production Poster P345 / October 12 Huntington’s Disease Pathway gene therapy for Huntington’s disease: A Phase 1/2 dose-finding study to evaluate BV-101 striatal administration in adults with early manifest Huntington’s disease Invited oral presentation (INV33) / October 13 (8:30-10:45 BST) Preclinical/R&D miRNA coexpression during vector production to increase vector yield and transduction efficiency Poster P064 / October 13 Congestive Heart Failure A first-in-human Phase 1 clinical gene therapy trial for the treatment of non-ischemic heart failure using a novel rationally designed cardiotropic adeno-associated vector targeting calcium cycling Poster P670 / October 13 Parkinson’s Disease Safety and clinical findings 12-months following bilateral putaminal convection enhanced delivery of AAV2-GDNF in early and moderate stages of Parkinson’s disease Poster P170 / October 13 Alzheimer’s Disease Non-invasive AAV-CYP46A1 gene therapy mitigates disease progression in Alzheimer’s disease mice Poster P220 / October 13 Huntington’s Disease Astrocytes are key players in cholesterol gene therapy for Huntington’s disease Poster P182 / October 13 Preclinical/R&D Results from using AskBio capsid screening (ABCs) platform Poster P002 / October 13 TAAV Advanced characterization of enzymatically amplified doggyboneTM DNA for rAAV manufacturing Poster P300
AskBio Named One of Triangle’s Best Places to Work
Triangle Business Journal Selection Based on Employee-Provided Rankings in Six Categories Research Triangle Park, NC – August 31, 2022 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, has been named, by the Triangle Business Journal (TBJ), as one of the Triangle’s Best Places to Work. This award honors companies for creating employee-valued policies and work environments. “At AskBio, we are passionate about leading innovative science and delivering outcomes that transform people’s lives,” said Sheila Mikhail, JD, MBA, CEO and Co-Founder of AskBio. “As gene therapy pioneers, we play an important role in society. To be successful, our workplace must be as exceptional as our mission. Being added to this list is a special honor and one that highlights our focus on this significant aspect of our current and future success.” The 50 companies named to the 2022 Triangle’s Best Places to Work list represent a cross section of industries in North Carolina’s Raleigh-Durham-Chapel Hill area. Selections were the result of employee feedback on how well each company performs in six business-critical categories: Communication and Resources, Individual Needs, Manager Effectiveness, Personal Engagement, Team Dynamics, and Trust in Leadership. “We believe this recognition shows that AskBio is succeeding in its ongoing effort to provide the most attractive environment for employees,” added Philip Dana, Chief Human Resources Officer, AskBio. “Our company is committed to investing in the support, development and success of our nearly 800 team members across the US and Europe. We are implementing leading HR technologies, launching employee resource groups, working to deliver compensation and benefit programs similar to those of larger biotech companies and much more.” About AskBio Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson’s disease, and congestive heart failure. AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 750 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more at www.askbio.com or follow us on LinkedIn. About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its
BrainVectis, a subsidiary of AskBio, receives clearance to conduct Phase I/II clinical trial in France for its novel gene therapy for early-stage Huntington’s Disease
National Agency for Safety of Medicines and Health Products (ANSM) and French Ethics Committee approve protocol for trial expected to begin in Q4 2022 Research Triangle Park, NC and Paris, France – August 23, 2022 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, has received clearance to conduct a Phase I/II trial for its novel Huntington’s Disease (HD) gene therapy, BV-101, in France through its subsidiary BrainVectis. This authorization, provided by the National Agency for Safety of Medicines and Health Products (ANSM), the country’s governing drug authority, along with the approval of the trial protocol by the Ethics Committee in charge, enables the company to begin recruiting participants. BV-101 is a novel, exclusively designed adeno-associated virus (AAV) gene therapy vector that simultaneously addresses the metabolic dysfunction of diseased neurons as well as contributes to the clearance of the mutant huntingtin protein. BV-101 is administered through MRI-guided neurosurgical techniques directed to target tissues in the basal structures of the brain. In preclinical studies in mice, BV-101 demonstrated the ability to repair the essential cholesterol pathway, provide neuroprotection, and restore physical performance by delivering CYP46A1, a crucial enzyme in the brain which is reduced in people with Huntington’s Disease. BV-101 was granted orphan drug designation in the European Union in 2019 by the European Medicines Agency. “Unlike other attempts to treat Huntington’s Disease, BV-101 aims to restore cholesterol metabolism, reduce mutant huntingtin and to improve neuronal function. Importantly, BV-101 does not affect the levels of normal huntingtin protein in cells,” said Nathalie Cartier-Lacave, MD, founder of BrainVectis and now Vice President, Sector Lead Neurobiology, at AskBio. “If this proves successful, we have the potential to change the course of a devastating disease that causes severe functional and cognitive decline.” Currently, there are no approved disease modifying therapies for HD, a rare inherited neurodegenerative disease that, based on information from the Committee for Orphan Medicinal Products (COMP), affects approximately 62,000 people in the European Union. The disease is caused by anomalous repeating mutations in the huntingtin gene leading to abnormal protein aggregates in nerve cells. This results in a range of progressive symptoms, leading to complete physical and mental deterioration, with symptoms usually beginning in adults ages 30 to 50, but which can also occur at an earlier age. “The approval of this trial in France marks a major milestone to potentially treat one of the world’s most devastating genetic diseases,” added Sheila Mikhail, JD, MBA, CEO and Co-Founder of AskBio. “If successful, this novel approach for treating Huntington’s Disease may impact how we treat many other neurodegenerative diseases in the future.” About the BV-101 Clinical TrialThe BV-101 clinical trial will be an open-label, dose-escalation study to assess the safety, tolerability, and preliminary efficacy of administration of BV-101 in adult subjects with early-stage Huntington’s Disease (HD). The trial will include 12-18 participants and is expected to begin in Paris in Q4, 2022. The trial will be led by principal investigator, Alexandra Durr, MD, PhD, Professor Genetics, Reference Centre for Rare diseases-Neurogenetics. For more information about the BV-101 Huntington’s Disease
AskBio Bolsters its Global Leadership Team with Two New Executives
Doug Schantz joins as Senior Vice President of Clinical Operations and Phil McNamara to serve as Vice President of Communications Research Triangle Park, N.C. –August 11, 2022 – Asklepios BioPharmaceutical, Inc. (AskBio), an independently operated subsidiary of Bayer AG, is adding two industry veterans to advance clinical operations and lead corporate communications. Doug Schantz will serve as the Senior Vice President of Clinical Operations and will ensure that all clinical trials are executed expeditiously, in full regulatory compliance and to the highest quality standard. Phil McNamara, serving as Vice President of Communications, will be responsible for all internal and external communications and media relations. “Doug and Phil bring a wealth of experience to the AskBio team and are reflective of the quality of individual talent we have at the company,” said Phil Dana, Chief Human Resources Officer. “Both represent key hires for AskBio as we continue to advance our pipeline and establish our position as a global leader in genetic medicine.” Doug Schantz is a seasoned clinical operations expert in the biotechnology and pharmaceutical industry. He comes to AskBio from Alexion Pharmaceuticals where he most recently served as Vice President and Head of Clinical Operations. At Alexion, he was responsible for program leadership across all phases of clinical development in several therapeutic areas, including hematology, metabolic diseases, neuroscience, and nephrology. Prior experience was with AstraZeneca as the Executive Director of Development Operations, as well as successfully serving a 17-year stint at Pfizer and eight years with Eli Lilly. Phil McNamara joins AskBio from Novartis where he led Global Communications for its Cardio-Renal-Metabolic Franchise. He successfully built and led a team that supported various facets of the business and helped to enable a favorable market standing with patient and professional communities. Prior to Novartis, Mr. McNamara held global and US communication roles at Sanofi and Bristol Myers Squibb, respectively, and collectively brings over 20 years of diverse strategic communications experience to AskBio. Canwen Jiang, MD, PhD, AskBio’s Chief Development Officer and Chief Medical Officer adds, “Both Doug and Phil are vital members of our senior management team who will equally strengthen our presence within the industry and contribute to our success in all our clinical endeavors.” About AskBio Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, is a fully integrated gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson’s disease, and congestive heart failure. AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 750 patents
AskBio CEO and Co-Founder Sheila Mikhail receives 2022 Entrepreneurial Award from University of Chicago Booth School of Business
Prestigious recognition for outstanding professional achievement in the formation of a successful enterprise awarded to Booth School of Business Alumni Research Triangle Park, N.C. – June 2, 2022 – Asklepios BioPharmaceutical, Inc. (AskBio), an independently operated subsidiary of Bayer AG, today announced that CEO and Co-Founder Sheila Mikhail was recognized by the University of Chicago Booth School of Business for outstanding professional achievement among its alumni. As a 1993 finance MBA graduate with honors, Ms. Mikhail has been recognized with the 2022 Entrepreneurial Award. “This award is tremendously meaningful as the Booth School of Business is one of the nation’s top ranked business schools,” said AskBio Co-Founder and CEO Sheila Mikhail. “The award is a reflection of the accomplishments that could not be possible without the support of a world-class team at AskBio as we continue to advance our research, clinical candidates and manufacturing capabilities.” Ms. Mikhail earned a JD with honors from Northwestern University, a finance MBA with honors from the University of Chicago, and a BS with highest honors from the University of Illinois at Urbana-Champaign. She co-founded AskBio in 2001 with Dr. R. Jude Samulski, who is considered one of the leading pioneers for AAV gene therapy technology. Since co-founding AskBio, and under the leadership of Ms. Mikhail, the company, including its manufacturing facilities, has grown to employ over 700 biotech professionals operating in five countries. Eighteen months after closing a series A funding round of $235 million in April 2019, Ms. Mikhail orchestrated a $4 billion acquisition by Bayer AG in October 2020. Under Ms. Mikhail’s leadership, AskBio is flourishing with an expansive gene therapy technology toolbox, three state-of-the-art manufacturing facilities in San Sebastian Spain and a robust therapeutic pipeline, all at various clinical stages, to treat Pompe Disease, Limbe Girdle Muscular Dystrophy, Multiple System Atrophy, Congestive Heart Failure, Huntington’s Disease, and Parkinson’s Disease. “In my view, Sheila is one of the most accomplished Biotech executives in the industry,” commented Dr. Samulski, AskBio’s Co-Founder and Chief Scientific Officer. “The recognition by University of Chicago Booth School of Business is a testament of her unparalleled business acumen combined with her significant command of genetic science” The Distinguished Alumni awards are only available to graduates of Chicago Booth. The Entrepreneurial Award is given to a person who has demonstrated professional achievement of the highest caliber in the management and formation of a successful enterprise and is recognition by colleagues and peers for outstanding leadership and administrative abilities. About AskBio Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, is a fully integrated gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson’s disease, and congestive heart failure. AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research
AskBio President of Therapeutics Dr. Katherine High receives Award from American Society of Gene and Cell Therapy
Jerry Mendell Award recognizes Dr. High for her outstanding work to bring cell and gene therapies to patients Research Triangle Park, N.C. – May 19, 2022 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced that Dr. Katherine High, President of Therapeutics, received the Jerry Mendell award for Translational Science from the American Society of Gene and Cell Therapy (ASGCT). Recently inducted as a member of the National Academy of Sciences (NAS), Dr. High has received this award in recognition of her significant contributions to the field of genetic medicine. “I am honored to receive this recognition from ASGCT,” said Dr. Katherine High. “I have dedicated my career to building the translational science that enables us to move scientific discoveries to new medicines for patients. I look forward to furthering this work at AskBio and with our many partners to find the answers for devastating diseases that currently lack disease-modifying treatments.” Dr. High joined AskBio in 2021 and is internationally recognized for her pioneering bench-to-bedside research and clinical success for the clinical translation of genetic therapies for multiple inherited disorders. Most notable, while serving as the Inaugural Director of the Center for Cellular and Molecular Therapeutics at Children’s Hospital of Philadelphia (CHOP), Dr. High led a multidisciplinary team of scientists and physicians to spin out Spark Therapeutics, where she led the team that achieved the first U.S. Food and Drug Administration approval of a gene therapy for a genetic disease. Today, at AskBio, Dr. High oversees a robust pipeline of gene therapeutics to treat various neuromuscular, central nervous system, cardiovascular and metabolic disorders, all at various stages of clinical development. “As a true pioneer in the field, author of more than 200 scientific papers with multiple patents in gene therapy, Dr. High is one of the most accomplished persons in gene therapy today,” added Sheila Mikhail, co-founder, and Chief Executive Officer of AskBio. “I am privileged to have her on our team and, on behalf of all of us at AskBio, we congratulate her for this esteemed award and her continued accomplishments in genetic medicine.” The Jerry Mendell Award for Translational Science award is named for Jerry Mendell, MD, the first person in collaboration with Askbio co-founder Dr. R. Jude Samulski, to study viral mediated gene therapy for muscular dystrophy in humans and recognizes the extensive work required to bring gene and cell therapies to clinical trial. Mendell was also the principal investigator who brought Zolgensmaâ, a gene therapy treatment for Spinal Muscular Atrophy that uses the recombinant adeno-associated virus (rAAV) vector technologies created by AskBio founder and Chief Scientific Officer, Dr. R. Jude Samulski. It is also the same technology that is used for Luxturnaâ, the FDA approved gene therapy brought to market by the team led by Dr. High while at Spark Therapeutics. Dr. High was acknowledged on May 17, 2022, as part of the ASGCT’s 25th annual meeting, the premier event bringing together over 4,800 noted professionals and experts in gene and cell therapy in the United States and
AskBio to Present 11 Abstracts at Upcoming American Society of Gene and Cell Therapy’s 25th Annual Meeting
Presentations convey essential technological and scientific knowledge regarding AAV gene therapy and advancements across key disease areas CEO Sheila Mikhail and President of Therapeutics, Katherine A. High will be featured speakers Research Triangle Park, N.C. – May 16, 2022 –Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced that the Company will present 11 abstracts at the upcoming American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting being held May 16 – 19, 2022 at the Walter E. Washington Convention Center in Washington, D.C. The ASGCT Annual Meeting is the premier event for professionals in gene and cell therapy where noted industry professionals gather to learn from the latest advances in scientific and clinical research and cell and gene technology. Abstracts being presented by AskBio team members include new data and insights regarding Adeno Associated Virus (AAV) gene therapy, T-cell immune response to empty capsid technologies, inducible promoters and gene expression, and doggybone DNA (dbDNA) as well as data for AskBio’s key clinical development programs, including Pompe Disease, Parkinson’s Disease and Congestive Heart Failure. AskBio will make seven oral presentations and four poster presentations. CEO Sheila Mikhail and President of Therapeutics, Katherine A. High will be featured speakers during the event. “Having 11 abstracts accepted for presentations reflects the significant progress made by our teams across a broad front,” commented Kathy High, President, Therapeutics for AskBio. “I am very proud of the groundbreaking work by our research and clinical teams as we continue to advance our therapeutic pipeline and AAV gene therapy research and manufacturing.” Jude Samulski, Chief Science Officer and Co-Founder for AskBio said, “These presentations underscore our commitment to advancing the science of gene therapy to tackle many of the biggest challenges in the space today, including manufacturing, dosing, immune response and treatment efficacy. We hope that, together with our many colleagues in cell and gene therapy space, we can make a profound difference in the lives of patients around the world who are waiting for transformative gene therapies.” AskBio’s presentations at ASGCT include: Monday, May 16 Oral PresentationAbstract 37: Functional Assessment of T-cell Responses to AAV8 Empty Capsids in Healthy VolunteersSession: Immune Responses to AAV Vectors 10:30 am – 11:45 am, Room: 102 Oral PresentationAbstract 28: A First-in-Human Phase 1 Clinical Gene Therapy Trial for the Treatment of Heart Failure Using a Novel Re-Engineered Adeno-Associated Vector Session: Cardiovascular and Pulmonary Diseases 11:45 am – 12 PM, Room 206 Featured Speaker Launching Innovation Into Gene Therapy Companies Session: Part 2: Translating Science Into Medicine: Moving from Bench to Startup (Organized by the Bioindustry & Translational Science Committees) Sheila Mikhail, JD, CEO and Co-Founder, AskBio 1:30–2:18 PM Room: Salon G Tuesday, May 17 Oral Presentation: Abstract 434: Characterization of Alternative Reading Frame Proteins Generated from AAV Cassettes Session: Discoveries in Fundamental AAV Biology 4:00–4:15 PM, Ballroom A Poster Presentation: Abstract 796: Safety and preliminary efficacy of neurosurgical AAV2-GDNF delivery for Parkinson’s disease Session: Gene and Cell Therapy Trials in Progress 5:30PM, Hall D Poster Presentation: Abstract 711:
AskBio and Touchlight Restructure Joint Venture
Asklepios BioPharmaceutical, Inc. (AskBio) and Touchlight have announced a revised structure to their former joint venture, Touchlight AAV.
