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Berlin, Germany, and Research Triangle Park, NC, USA, April 16, 2024 – Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, on Sunday April 14 presented results from the 18-month Phase Ib clinical trial for AB-1005, an investigational gene therapy for treating patients with Parkinson’s disease (PD).1,2The data were presented at the American Academy of Neurology 2024 Annual Meeting in Denver, Colorado, USA. The study met its primary objective, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. Eleven patients were enrolled into two cohorts, Mild stage PD (6 patients) and Moderate stage PD (5 patients), based on timing from PD clinical diagnosis and the severity of PD symptoms at trial screening.1 As of November 3, 2023, 57 nonserious adverse events (AEs) and 6 serious adverse events (SAEs) were reported. Most AEs were transient and were expected perioperative events (<1 month from treatment). These included headache, tremor, dyskinesia, arthralgia, musculoskeletal chest pain, fatigue, COVID-19, and magnetic resonance imaging (MRI) abnormalities. The 6 SAEs reported in 3 patients (n = 1 in the Mild Cohort and n = 2 in the Moderate Cohort) were all assessed as unrelated to the treatment by the Investigator and the Sponsor. Bilateral infusions of AB-1005 within the putamen (up to 1.8 mL) were well tolerated, with no SAEs associated with the investigational gene therapy or contrast agent. Neurosurgical delivery of AB-1005 resulted in putamen coverage of 63% ± 2%, exceeding the goal of greater than 50% coverage with AB-1005. Scheduled 6-month postoperative MRIs revealed findings of asymptomatic unilateral T1 hypointensity adjacent to 3 of the putaminal infusion trajectories. Clinical follow-up for up to 5 years post administration is ongoing.2 “These early findings are encouraging and show AB-1005 to be well tolerated in this study in patients with mild to moderate Parkinson’s disease,” said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA, AskBio. “Further, they highlight areas of potential future exploration in our upcoming Phase II REGENERATE PD trial, which will look more closely at the potential efficacy of AB-1005 in the treatment of Parkinson’s disease.” Patients also completed 18-month neurological assessments and self-reported questionnaires at regular intervals to evaluate the severity of motor and non-motor symptoms associated with PD.1 Mild Cohort The Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) is an internationally recognized tool used to assess the severity of PD symptoms, including motor symptoms. The Mild Cohort (n = 6) demonstrated relative stability from baseline to 18 months for both MDS-UPDRS Part II patient-reported Activities of Daily Living scores and Part III clinician-rated Motor Examination scores in “ON” and “OFF” medication states. Patient-reported PD Motor Diaries provide a tool for assessing patient motor state over an extended 3-day period and then normalized to a 16-hour waking day. The Mild Cohort (n = 5) showed a -1.3 hour reduction in “Good ON” time, a 0.2 hour increase in “ON” time with troublesome

Not intended for UK Media Berlin, Germany and Research Triangle Park, NC, USA, February 13, 2024 – Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the first patient has been randomized in GenePHIT (Gene PHosphatase Inhibition Therapy), a Phase II trial of AB-1002 (also known as NAN-101) for the treatment of congestive heart failure (CHF). GenePHIT is an adaptive, double-blind, placebo-controlled, randomized, multicenter trial to evaluate the safety and efficacy of a single intracoronary infusion of AB-1002 in adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms who have been medically stable for at least four weeks. This milestone in the development of AB-1002 for the treatment of CHF potentially brings this investigational therapy one step closer to treating patients with high unmet medical need.[2] GenePHIT will include between 90 and 150 adults with left ventricular ejection fraction between 15 and 35 percent, who continue to suffer from heart failure symptoms despite guideline recommended therapy. The primary efficacy endpoint at 52 weeks is a modified win ratio of several clinically meaningful assessments.1 “The randomization of the first patient as a part of the Phase II GenePHIT trial is an important moment for the heart failure community,” said Timothy D. Henry, MD, MSCAI, Principal Investigator and Steering Committee Member. “GenePHIT will evaluate the safety and efficacy of AB-1002 in the largest number of patients to date and improve our understanding of gene therapy overall for the treatment of congestive heart failure. The initiation of this trial brings us a step closer to potentially changing the course of this deadly and devastating disease.” “Being able to announce this important GenePHIT trial update during Heart Failure Awareness Week adds special significance to this milestone,” said Roger J. Hajjar, MD, Scientific Chair CHF, AskBio. “The enrollment of this first patient in the Phase II trial represents the culmination of many years of dedicated research and development in all aspects of cardiac gene therapy for congestive heart failure. Although there is still much to learn about this early-stage investigational gene therapy, we hope today’s announcement, which highlights AskBio’s ability to advance AB-1002 gene therapy for the treatment of congestive heart failure, is encouraging news for everyone hoping to see new treatment options.” “Heart failure is a devastating disease with increasing unmet medical need, especially in a progressively aging population,” said Christian Rommel, PhD, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of Research and Development. “The potential impact of gene therapy to address this disease at its root cause is immense, and we are thrilled about this step in our path to deliver truly innovative treatment options for patients.” AB-1002 is an investigational gene therapy that has not received marketing authorization, and its efficacy and safety have not been established or fully evaluated. AB-1002 is manufactured by Viralgen Vector Core, S.L., a wholly owned and independently operated subsidiary of

Berlin, Germany/Research Triangle Park, North Carolina, USA, January 4, 2024 – Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced today the completion of the 18-month data collection in the Phase Ib clinical trial for AB-1005 (AAV2-GDNF), an investigational gene therapy for treating patients with Parkinson’s disease (PD).1,2 The study met its primary objective, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. Eleven patients were enrolled into two cohorts, Mild stage PD (6 patients) and Moderate stage PD (5 patients), based upon the timing from a PD diagnosis and the severity of their PD symptoms at screening.1 Neurosurgical delivery of AB-1005 was well tolerated by all patients with target putamen coverage of 63% ± 2%, exceeding the goal of greater than 50% coverage with AB-1005. No serious adverse events have been attributed to AB-1005, with continued clinical follow-up for up to 5 years post administration ongoing.2 “We are encouraged by these early data, which show AB-1005 to be well tolerated in this study in patients with mild to moderate Parkinson’s disease,” said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA, AskBio. “Although there is still much to learn about this early-stage investigational gene therapy, these first findings will inform our work in this space and have the potential to contribute to the clinical advancement of AB-1005 for the treatment of Parkinson’s disease.” Patients also completed 18-month neurological assessments and self-reported questionnaires at regular intervals to evaluate the severity of motor and non-motor symptoms associated with PD. Additionally, brain imaging was performed to longitudinally assess safety and potential changes in dopamine handling or abnormal metabolic patterns associated with PD.1 AskBio is planning to present the 18-month study data, including secondary endpoints, at a scientific meeting in Q2 2024. Planning is underway for a Phase II trial that is expected to begin screening patients in the first half of 2024. The trial design has been harmonized with feedback from U.S. and European health authorities. “People living with Parkinson’s disease deserve options to address their unmet medical need,” said Christian Rommel, PhD, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of Research and Development. “The positive outcome of the AB-1005 Phase Ib clinical trial is an important step forward in our goal to deliver much-needed treatments in areas where innovation has the potential to make a tremendous impact.” About AB-1005 AB-1005 is an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene, which allows for stable and continuous expression of GDNF in localized regions of the brain after direct neurosurgical injection with MRI-monitored convection enhanced delivery.3,4 GDNF is a homodimer that is a distantly related member of the transforming growth factor-β superfamily. In midbrain neuronal cell cultures, recombinant human GDNF promoted the survival and morphological differentiation of dopaminergic neurons and increased their high-affinity dopamine uptake.

Research Triangle Park, N.C. – November 17, 2023 – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the first patient has been randomized at the Ohio State University Wexner Medical Center in the Phase 1 REGENERATE MSA-101 clinical trial of AB-1005, a gene therapy being developed as a treatment for multiple system atrophy-parkinsonian type (MSA-P).[1] This marks a significant milestone in the development of AB-1005 gene therapy, an adeno-associated viral vector encoding for glial cell line-derived neurotrophic factor (AAV2-GDNF) that is delivered to the putamen, and brings this therapeutic one step closer to potentially reaching patients. AB-1005 is also currently being investigated for the treatment of mild to moderate Parkinson’s disease with the enrollment of the Phase 1b study having now been completed.[2]   “It means a lot to the MSA community to know that the first patient has been enrolled in the Phase 1 REGENERATE MSA-101 trial,” said Philip M. Fortier, MA, President and Executive Director, Defeat MSA Alliance. “There is no cure for MSA, and there are currently no treatments to stop or slow the progression of the disease. This makes it especially hard for patients, given the rapid decline many will experience. Today’s milestone hopefully brings us one step closer to potentially changing the outcome for MSA patients.” MSA-P, which can initially be difficult to distinguish from Parkinson’s disease, is marked by slow movement, lack of coordination, imbalance, and dizziness, among other symptoms, as individuals experience increasing difficulty with movement.[3] This is the result of a progressive loss of nerve cells in the brain and spinal cord. Affecting an estimated 100,000–500,000 worldwide, MSA is a rare disease that in most cases seems to occur at random.3,[4],[5] Symptoms tend to appear in people during their 50s, followed by a rapid progression within 5–10 years.3 “In those with MSA-P, the loss of dopamine producing neurons leads to markedly disabling symptoms, such as profound motor impairment throughout the hands, legs, and trunk. Previous experience with a similar approach in a population of Parkinson’s disease patients has been very encouraging and supported the consideration of this approach in MSA-P. The intent is for GDNF levels in the brain to help preserve dopamine neurotransmission, which is noticeably reduced in MSA-P,” said Nicolas M. Phielipp, MD, University of California Irvine, REGENERATE MSA-101 Principal Investigator. “We’re including a genetic sequence in the AAV2 vector that codes for the GDNF protein and are delivering this to the putamen. In this way, we’re targeting local brain cells and adjacent brain tissue that can benefit from the protein’s growth properties. This trial marks the first step toward understanding the potential that GDNF gene therapy might have for patients with MSA-P.” “Enrolling the first patient in our REGENERATE MSA-101 trial is an important step in AskBio’s ongoing work to advance GDNF gene therapy” said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA, AskBio. “The clinical advancement of AB-1005 for the treatment of MSA-P

Research Triangle Park, N.C., October 13, 2023. Asklepios BioPharmaceutical, Inc. (AskBio or the company), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced its updated Executive Leadership Team (ELT) structure, which includes four newly created roles designed to improve the company’s ability to efficiently bring its clinical-stage gene therapies to patients. The addition of these new roles aligns AskBio’s leadership structure with its increased scope and capabilities as an end-to-end gene therapy company active from the earliest stages of discovery through to research; development; and, with the expertise and support of Bayer, eventually delivery. “I am pleased to share that we have added four critical roles to our ELT—Chief Business Officer, Chief Strategy Officer, Chief Product Supply Officer, and Head of People and Culture,” said Gustavo Pesquin, CEO, AskBio. “This move ensures that we have the leadership required to build on our more than 20 years of groundbreaking discovery experience and become a high-performing delivery organization. These new leadership positions increase our focus on all that it takes to meet the needs of the tens of millions of people around the world living with debilitating diseases that we hope to one day treat. Our expanded team will usher in a new era of productivity and can dramatically increase our ability to transform lives.” Tracy Dowling has been named Chief Business Officer and General Counsel. Dowling joined AskBio in 2021 from Spark Therapeutics and now oversees Business Development, Alliance Management, Legal, Compliance, Communications, and a newly created Chief of Staff role. She will further develop key partnerships that will enhance AskBio’s technological and clinical portfolio and advance the field. This will be done by creating in-licensing, out-licensing, and other collaboration opportunities, which will grow the company’s capabilities and share its technologies with others. Ralph Herbst serves as Chief Strategy Officer. Herbst joined the AskBio ELT from Bayer. He leads Corporate Development and Portfolio Management, and he will play a key role in optimizing the AskBio-Bayer partnership. Herbst brings a strong track record of building and expanding cell and gene therapy pipelines and capabilities, which includes his work with Bayer’s Cell & Gene Therapy Platform and his critical involvement in Bayer’s acquisitions of BlueRock Therapeutics LP in 2019 and AskBio in 2020. Jason Krentz takes on the role of Chief Product Supply Officer. Overseeing programs that transition clinical development to commercial operations, he leads Product Supply operations, Facilities, and Information Technology. Krentz joined AskBio in 2022 from Tmunity Therapeutics, where, as Chief Technology Officer, he led the company’s Chemistry, Manufacturing, and Controls (CMC) operations. He brings over 20 years of technical operations and manufacturing experience to AskBio. Melissa Murrell has been appointed Senior Vice President, Head of People and Culture, and oversees all areas of Human Resources with a focus on culture and employee empowerment. Murrell joined AskBio in 2021 from Spark Therapeutics and has played key roles in the company’s efforts to create the structure and systems required to support its rapid growth

Gene therapy developed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9. Research Triangle Park, N.C.– August 3, 2023 – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the first patient has been dosed in the Phase 1 / Phase 2 LION-CS101 clinical trial of patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).[1] AB-1003 (also known as LION-101) is a novel investigational FKRP gene replacement therapy. “Hearing that the first patient has been dosed in this study evaluating AB-1003 is an exciting moment for the limb-girdle muscular dystrophy community and individuals living with this debilitating disease,” said Kelly Brazzo, Co-Founder & CEO, CureLGMD2i. “Given the current lack of disease modifying treatments for LGMD, many in the community know of the potential of gene therapy. The initiation of this trial offers hope that patients with this condition may, in the future, have a significantly improved quality of life.” LGMD2I/R9 is a form of LGMD caused by changes in the FKRP gene and is associated with weakness and wasting of arm and leg muscles.[2]Those with LGMD2I/R9 notice symptoms in late childhood, around 11 years of age, which may include loss of mobility and impaired heart and lung function.2,[3] As symptoms worsen, individuals generally require wheelchairs.2 LGMD2I/R9 is a rare disease, estimated to affect more than 5,000 people in the US and EU.[4] “While the inherited nature of limb-girdle muscular dystrophy means those with the FKRP gene mutation can’t produce a normal FKRP protein for physiological muscle function, AB-1003 is designed to introduce the normal FKRP gene into the muscle and express a normal protein, and it has shown promise in restoring normal FKRP protein function in muscle in preclinical studies performed in mouse models of LGMD,” said Nicholas Johnson, MD, Principal Investigator and Vice Chair of Research at the Department of Neurology, Virginia Commonwealth University School of Medicine. “This trial is the first step toward evaluating the safety of AB-1003 and assessing the potential that AB-1003 has to improve the lives of patients with this serious, inherited ultra rare condition.” “The first limb-girdle muscular dystrophy 2I/R9 patient dosed in the LION-CS101 clinical trial is another example of AskBio’s success in taking gene therapy candidates, such as AB-1003, from the bench into clinical development,” said Jude Samulski, PhD, Co-Founder and Chief Scientific Officer of AskBio. “With our knowledge and expertise, we are working to harness the power of gene therapies as potential treatments for many of the world’s most devastating diseases. Our investigational therapy for limb-girdle muscular dystrophy is one of these and an important part of our portfolio, which also includes clinical stage investigational therapies for congestive heart failure, Huntington’s disease, multiple system atrophy, Parkinson’s disease, and Pompe disease.” AB-1003, which is manufactured by AskBio’s wholly owned and independently operated subsidiary Viralgen, was granted fast track designation by the FDA and orphan drug designation by the European Commission.[5],[6] Recruitment for the LION-CS101 clinical

— LGMD is a Rare Form of Muscular Dystrophy with No Approved Therapy — Research Triangle Park, NC – February 16, 2023 – Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG,  announced today that the European Commission (EC) has granted orphan drug designation for AB-1003 (also known as LION-101)* for the treatment of limb-girdle muscular dystrophy (LGMD). AB-1003 is a novel investigational recombinant adeno-associated virus (AAV) based gene therapy currently being developed as a one-time intravenous (IV) infusion for the treatment of patients with LGMD type 2I/R9 (LGMD2I/R9), a disease subtype affecting 4.5 people per million worldwide, including more than 5,000 people in the EU and US. The EC decision, dated February 15, 2023, follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) and was received through AskBio’s EU-based subsidiary BrainVectis. AB-1003 is being investigated in the US in a Phase 1/2 multicenter study that will evaluate the safety, tolerability and efficacy of a single IV infusion of gene therapy in adult subjects with genotypically confirmed LGMD2I/R9.  “The EC orphan drug designation for AB-1003 is an important recognition of the unmet medical need in LGMD, which has no approved therapy,” said Sheila Mikhail, Co-Founder & CEO, AskBio. “The burden of this rare form of muscular dystrophy on patients and their families is significant, and this decision supports our efforts to potentially bring a new therapeutic option to people in the EU living with the 2I/R9 type of this devastating disease.” The EC grants orphan drug designation for medicinal products intended to treat a life-threatening or chronically debilitating disease that affects no more than five people in 10,000 in the EU, provided there is no other satisfactory treatment option or the medicine can be of significant benefit to those affected by a specific condition. This designation will provide special incentives in the EU, including eligibility for protocol assistance and possible exemptions or reductions in certain regulatory fees. In addition, if the medicine is approved for marketing, this designation will provide 10 years of marketing exclusivity. About Limb-Girdle Muscular Dystrophy (LGMD) and Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) Limb-girdle muscular dystrophy (LGMD) is a term for a group of diseases that cause progressive weakness and wasting of the muscles in the arms and legs.1 The muscles most affected are those closest to the body (proximal muscles), specifically the muscles of the shoulders, upper arms, pelvic area and thighs.1 The severity, age of onset, and features of LGMD vary among the many subtypes of the condition and are often inconsistent, even within the same family.1 Signs and symptoms may first appear at any age and generally worsen with time, although in some cases they remain mild.1 Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) is a form of LGMD and is caused by mutations in the FKRP gene.2 In LGMD2I/R9, signs and symptoms often develop in late childhood and may include difficulty running and walking.2 The symptoms gradually worsen over time toward significant disability, and affected people generally rely

— 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

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

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