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Research Triangle Park, N.C.– JUNE 25, 2024 – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced initiation of recruitment to REGENERATE-PD, a Phase 2 clinical study assessing efficacy and safety of AB-1005, an investigational adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy for the treatment of moderate-stage Parkinson’s disease. “By enhancing levels of a naturally occurring growth factor, glial cell line-derived neurotrophic factor (GDNF) gene therapy is intended to promote the survival and functioning of vulnerable brain cells that degenerate in Parkinson’s disease”, said Alan Whone, MD, PhD, Consultant Senior Lecturer in Movement Disorder, Bristol Medical School, and Honorary Consultant Neurologist at North Bristol Trust, UK.  “The advancement of AB-1005 is a significant milestone in the development of a gene therapy for Parkinson’s disease and has the potential to bring an effective treatment one step closer to patients”.  Dr Whone will act as European Lead Principal Investigator (PI) on REGENERATE-PD once the program becomes active in the UK. According to the Parkinson’s Foundation, more than 10 million people worldwide suffer from Parkinson’s disease.1 “Following the encouraging results from the Phase 1b study and the presentation of 18-month data at the American Association of Neurology (AAN) meeting in April, we are excited to be progressing AB-1005 to this larger, Phase 2 study,” said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA, AskBio. “This latest advancement highlights our confidence in the potential of AB-1005 to provide a transformative impact for patients with Parkinson’s disease.” In January, AskBio announced that the Phase 1b trial of AB-1005 met its primary endpoint, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. The investigational gene therapy for the treatment of Parkinson’s disease was well tolerated with no serious adverse events that were considered related to AB-1005 in all 11 patients at 18 months.2 AskBio is also exploring GDNF therapy beyond Parkinson’s disease and is currently enrolling patients in the US with the parkinsonian subtype of multiple system atrophy (MSA-P) in a Phase 1 trial to assess the preliminary safety, tolerability, and efficacy of GDNF therapy for this rapidly progressing condition.3 AB-1005 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated. About Parkinson’s disease  Parkinson’s disease is a progressive neurodegenerative disorder caused by the death of nerve cells in the brain, leading to decreased dopamine levels.4 At diagnosis, it is estimated that patients have already lost 50-80% of their dopaminergic neurons.5 The loss of these neurons leads to a progressive loss of motor function and symptoms such as tremors, muscle rigidity, and slowness of movement.6 Even with medication, the symptoms of Parkinson’s disease can fluctuate during the course of the day. According to the Parkinson’s Foundation, more than 10 million people worldwide suffer from Parkinson’s disease, with approximately one million living in the United States.1 There is no

Research Triangle Park, N.C.– MAY 2, 2024 – Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, will deliver nine presentations offering insights into its adeno-associated virus (AAV) research and development, a key area of gene therapy focus for the company, at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, which takes place May 7–11, 2024, in Baltimore, Maryland, USA. Presentations will focus on AAV immune-mediated responses as well as the results from the ongoing Phase 1 clinical trial of AB-1002 gene therapy in patients with advanced heart failure. Luke Roberts, MBBS, PhD, Medical Director for Clinical Development at AskBio, will deliver an oral presentation sharing new clinical data from the company’s ongoing Phase 1 trial of AB-1002 in patients with advanced heart failure. This follows AskBio’s recent news that AB-1002 was granted FDA Fast Track Designation for the treatment of congestive heart failure (CHF). AB-1002 (also known as NAN-101) is an investigational gene therapy that has not yet received marketing authorization, and its efficacy and safety have not been established or fully evaluated. AskBio previously communicated that the delivery of AB-1002 was well tolerated and resulted in positive preliminary efficacy outcomes in some patients with non-ischemic CHF and may validate that the AAV2i8 vector capsid used is highly cardiotropic when administered as a single intracoronary infusion at relatively low doses. Preliminary data from the Phase 1 trial of AB-1002 were presented at the 2023 American Heart Association Scientific Sessions in November, and AskBio began enrolling patients in its Phase 2 GenePHIT study of AB-1002 in adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms in January 2024. AskBio’s ASGCT presence will also include key presentations showcasing the company’s continued commitment to optimizing AAV as a gene therapy, with a focus on preventing or reducing AAV immune response-related adverse events, which remains a vital area of investigation across the gene therapy treatment landscape. Attendees can look forward to an ASGCT spotlight speaker presentation on current immune modulation strategies given by Shari Gordon, PhD, Senior Director of Immunology at AskBio, on Day 3. On Day 4, Shari Gordon will deliver on behalf of Audry Fernandez, PhD, Principal Scientist & Group Lead, Immunology R&D at AskBio, an oral presentation on pre-clinical research into AAV-specific immune responses, and Liujiang Song, PhD, Principal Scientist for R&D Capsid and Biology at AskBio, will offer insights into AAV biology and vector intracellular fate during an oral presentation on AAV episome configuration using third generation long-read sequencing technologies and advanced bioinformatics. “Our presence at ASGCT this year highlights our continued commitment to sharing AAV developments with the gene therapy community. Covering clinical and pre-clinical research, our presentations show our robust progress and ongoing ambition to bring to patients transformative therapies that were once unthinkable,” said Gustavo Pesquin, Chief Executive Officer, AskBio. “With our clinical and early-stage programs advancing, these are exciting times at AskBio.” With an ambitious

Berlin, Germany, and Research Triangle Park, NC, USA, April 18, 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 U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the AB-1002 program. AB-1002 is an investigational one-time gene therapy that is administered to the heart with the intention of helping to promote the production of a constitutively active form of protein inhibitor 1 (I-1c) designed to block the action of protein phosphatase 1. Inhibiting the function of this protein, which is linked to congestive heart failure (CHF), could potentially lead to a therapeutic effect on the heart.[1],[2] “The FDA Fast Track Designation for AB-1002 is an important accomplishment for the clinical development of this program and highlights our goal of potentially bringing effective treatments to patients with advanced congestive heart failure,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “We look forward to completing our Phase II GenePHIT clinical trial, which is currently enrolling patients with severe heart failure, and are committed to exploring the full potential of AB-1002 for the treatment of this devastating disease.” The FDA Fast Track Program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and fill unmet medical needs.[3] The purpose of the Program is to get important new therapeutics to patients earlier.3 Therapeutics that receive this designation benefit from eligibility for more frequent meetings with the FDA to discuss the development plan and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review. “The Fast Track Designation for AB-1002 emphasizes the need to rapidly advance new therapeutic modalities such as gene therapy for people living with congestive heart failure,” said Christian Rommel, PhD, Head of Research and Development at Bayer’s Pharmaceuticals Division. “This designation underpins the potential of AB-1002 to address currently high unmet medical need, and we are excited about the opportunity to accelerate its development.” 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. AskBio is currently enrolling patients in the Phase II GenePHIT (Gene PHosphatase Inhibition Therapy) trial of AB-1002 (also known as NAN-101) for the treatment of CHF.[4]  About GenePHIT GenePHIT is a Phase II adaptive, double-blinded, placebo-controlled, randomized, multi-center trial to evaluate the safety and efficacy of the one-time administration of AB-1002, via antegrade intracoronary artery infusion, in males and females age >18 years with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms.4 Subjects are randomized into one of three treatment groups in a 1:1:1 fashion to either low dose, high dose, or placebo. Primary outcome measures include cardiovascular related death and change from baseline in NYHA classification, left ventricular ejection fraction (LVEF), peak oxygen uptake (pVO2), and Six Minute Walk Test (6MWT).4 For more information, please visit clinicaltrials.gov

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