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

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 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: