Category: News

Research Triangle Park, N.C. – SEPTEMBER 17, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced the completion of enrollment for REGENERATE MSA-101, its Phase 1 clinical trial of AB-1005, an investigational gene therapy being developed as a treatment for multiple system atrophy-parkinsonian type (MSA-P).1  The completion of enrollment marks a significant milestone in the development of AB-1005, an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene, and it brings this potential treatment option one step closer to reaching the many patients affected by MSA-P. MSA-P can initially be difficult to distinguish from Parkinson’s disease and is marked by slow movement, lack of coordination, imbalance, dizziness, and fainting, among other symptoms.2 Individuals experience increasing difficulty with movement and autonomic dysfunction.2 This is a result of progressive loss of nerve cells in the brain and spinal cord.2 Affecting approximately 400,000 people worldwide, MSA is a rare disease that in most cases seems to occur for unknown reasons.2,3,4 Symptoms tend to develop in people over 50 years of age, followed by rapid progression within 5–10 years.2  “There is currently no disease-modifying treatment to stop or slow the progression of MSA, which makes the completion of enrollment in REGENERATE MSA-101 an important step in AskBio’s efforts to advance this clinical program and potentially bring a safe treatment to patients” said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA at AskBio. “This latest milestone follows recent additional AB-1005 announcements highlighting meaningful progress in our Parkinson’s disease program, including Phase 1b favorable safety outcomes.”  AskBio is also exploring AB-1005 beyond MSA-P in participants living with Parkinson’s disease in REGENERATE-PD, the company’s randomized, double-blind, surgically controlled Phase 2 clinical trial evaluating the safety and efficacy of AB-1005 delivered to the putamen in the brain of adult participants aged 45–75 years with moderate-stage Parkinson’s.5  In January, AskBio announced that the first participants had been randomized in REGENERATE-PD.6 In February, AskBio received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for AB-1005 for Parkinson’s disease, following the presentation of Phase 1b 36-month data that demonstrated a favorable safety profile and continued positive trends in assessed clinical outcome measures with no product-related serious adverse events.7,8  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 the Phase 1 REGENERATE MSA-101 Trial  REGENERATE MSA-101 is a randomized, double-blind, placebo-controlled Phase 1 trial designed to determine the safety of AB-1005, an investigational gene therapy delivered to the putamen in patients with multiple system atrophy-parkinsonian type (MSA-P).1 The trial will include adults aged 35 to 75 years, with a clinical diagnosis of MSA-P (as defined by current consensus criteria), who are suitable surgical candidates.1 The trial enrolled 11 participants who were randomized on the day of surgery to receive either AB-1005 or a non-invasive control surgery without any

Research Triangle Park, N.C.– MAY 27, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced today the recent publication of the complete results of its Phase 1b trial of AB-1005, a glial cell line-derived neurotrophic factor (GDNF) investigational gene therapy for the treatment of Parkinson’s disease (PD), in Movement Disorders, an official peer-reviewed journal of the International Parkinson and Movement Disorder Society.1 “The exploration of the neurorestorative and neuroprotective potential of GDNF gene transfer is critical to advancing our understanding of AB-1005, which may one day be a groundbreaking treatment to slow the progression of Parkinson’s disease,” said Chad Christine, MD, Professor of Neurology at the University of California, San Francisco, and publication author. “These encouraging results support further evaluation of this therapy in a randomized trial.” The publication, which is available online, states that treatment with AB-1005 was well tolerated with no serious adverse events related to GDNF gene therapy in all 11 participants and is associated with numerical stability in clinical assessment readouts (mild cohort) and improvement (moderate cohort) in clinical assessments of motor function at 18 months post-gene transfer. In addition to these data, which were first presented in April 2024 at the American Academy of Neurology Annual Meeting, the publication notes that non-motor assessments remained numerically stable throughout the 18-month follow-up in the mild and moderate cohorts. Stability findings may be an indication of the potential for AB-1005 to positively affect disease progression.1,2 “These results support the continued study of AB-1005 as a potential gene therapy to slow the progression of Parkinson’s disease,” said Lila Collins, PhD, Associate Director of Portfolio Development & Review at the California Institute for Regenerative Medicine. “We are pleased to have supported this important trial and look forward to AskBio’s Phase 2 REGENERATE-PD results, which will provide additional insights into the durability of the clinical response and potential disease-modifying effect of AB-1005.” In February 2025, AB-1005 was granted Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration.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 fully established or evaluated. About Parkinson’s disease  Parkinson’s disease (PD) is a progressive neurodegenerative disease.4 It has a significant impact on a person’s daily life.4  In PD, the death of dopamine-producing nerve cells in the brain leads to the continuous loss of motor function.5 Symptoms include tremors, muscle rigidity, and slowness of movement.6 Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, cognitive issues, and depression.6 Symptoms typically intensify over time and make everyday tasks increasingly demanding.6 The prevalence of PD has doubled over the past 25 years.4 Today, more than 10 million people worldwide are estimated to be living with PD.7 This makes it the world’s second most prevalent neurodegenerative disease.8 It is also the most frequent movement disorder.4,9  At present there is no cure, and current treatment options lack the holistic management of symptoms,

Research Triangle Park, N.C. – MAY 19, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, presented—during a dedicated late-breaking science session at this year’s European Society of Cardiology Heart Failure meeting—the complete dataset from a Phase 1 trial investigating AB-1002 for the treatment of congestive heart failure (CHF). This year’s meeting is being held in Belgrade, Serbia, from May 17 to 20, 2025. This non-randomized, sequential dose escalation trial includes escalating dose cohorts to evaluate the safety and preliminary efficacy of investigational gene therapy AB-1002 in participants with NYHA Class III non-ischemic heart failure with reduced ejection fraction (HFrEF).1 It is estimated that 64 million people worldwide are living with heart failure, and despite standard of care, mortality and morbidity in heart failure remain very high.2,3   The late-breaking oral presentation highlights that single-dose administration of AB-1002 at 12 months post-dose resulted in the following outcomes:4 Meaningful changes from baseline were considered to be: NYHA FC, ≥1 point; LVEF, ≥5%; MLHFQ, ≥10 points; VO2 max, ≥1.5 mL/kg/min; 6MWT, 30 meters. “No treatment-emergent or serious adverse events were deemed related to AB-1002 in this trial, and we saw clinically meaningful improvement of several efficacy assessments in some participants with non-ischemic CHF,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “These results support our belief that the AB-1002 chimeric capsid may be highly cardiotropic when administered as a single intracoronary injection at relatively low doses, and we’re excited to further evaluate the safety and efficacy of AB-1002 in GenePHIT, our currently recruiting Phase 2 trial.” Innovative therapies are urgently needed to address the underlying mechanisms associated with CHF, including improving pump function in participants with HFrEF.5 AB-1002 is a rationally designed cardiotropic adeno-associated virus (AAV) vector delivering a transgene for continuous onsite expression of the inhibitor 1 (I-1c), inhibitor of protein phosphatase 1, a protein that has been linked to heart failure. This Phase 1 trial is ongoing, and participants will be followed for 36 months post-intervention. Preliminary results of this trial, which did not include the additional three participants, were presented at the American Heart Association Scientific Sessions in November of 2023.6 AB-1002 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been fully established or evaluated. About AB-1002 AB-1002 is a one-time gene therapy administered to the heart to help promote increased production of a modified version of the therapeutic inhibitor 1 (I-1c) protein designed to block the action of protein phosphatase 1, which is linked to CHF.6,7 This investigational gene therapy has not been approved by any regulatory authority, and its efficacy and safety have not yet been established or fully evaluated. About Congestive Heart Failure Heart failure occurs when the heart cannot pump blood efficiently enough to meet the body’s needs, including providing sufficient oxygen to the organs.8 Congestive heart failure results in the slowing of the blood flow out of the heart,

Research Triangle Park, N.C.– MAY 8, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, will deliver 15 presentations at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, which takes place May 13–17, 2025, in New Orleans, Louisiana, USA. The company’s oral and poster presentations will provide updates on key research and development activities in gene therapy and gene editing, as well as advancements in manufacturing capabilities. Featured oral presentations will include “AI Use for Promoter and Transgene Design.” This will be delivered by Mansuo Shannon, Chief Scientific Officer at AskBio, as part of the Novel Approaches to Overcome Limits of Therapeutic Transgene Delivery and Durability scientific session. The presentation will explore how AI/ML (Artificial Intelligence/Machine Learning) may enhance efficacy, safety, and specificity of gene therapies by potentially predicting the best possible genetic sequences for the intended therapeutic outcome. In addition, AskBio’s Srinethe Saravanakumar will present “Vector Assembly Factories in Recombinant Adeno-Associated Virus Type 2 Producing Cells” as part of the AAV Biology and Mechanism session. The presentation will provide an in-depth visualization of rAAV2 (recombinant adeno-associated virus type 2) capsid assembly using electron tomography. It will also explore the potential effects of subcellular retargeting/expansion of capsid assembly through selected AAP (assembly-activating proteins) in vector production. The company’s third oral, “A Rock in a Storm: Using Data as Your Foundation to Speed and De-risk AAV Manufacturing,” will be presented by Viralgen’s María Iglesias González as part of the Tools and Technology session. This will explore how a data-led approach has the potential to significantly reduce variability, prevent costly failures, and shorten timelines from R&D to commercial production by enabling better process understanding, predictive modeling, real-time monitoring, and quality control across the development lifecycle. “Our scientific contribution to ASGCT this year underscores our long-standing commitment to advancing gene therapy research and providing leadership within the scientific community,” said Gustavo Pesquin, Chief Executive Officer, AskBio. “We are sharing updates from our early-stage programs and highlighting how we are adopting innovative approaches in our development work with the use of digitalization and AI. Equally important, we are also showcasing the work being done to advance our manufacturing processes as we continue to strengthen our end-to-end platform capabilities.” AskBio continues to develop an ambitious portfolio of investigational AAV-based gene therapies to treat some of the world’s most serious diseases, including congestive heart failure, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. By targeting these therapy areas, AskBio aims to deliver breakthrough treatments that could benefit tens of millions of patients worldwide.1–6 AskBio’s presentations at ASGCT include (all times CDT): Orals Posters About AskBio  AskBio Inc., a wholly owned and independently operated subsidiary of Bayer AG, 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

Research Triangle Park, N.C. – March 7, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced the advancement of the Phase 1/Phase 2 LION-CS101 clinical trial of investigational gene therapy AB-1003 in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) with dosing of the first participant in the second cohort. The recommendation to advance to the second cohort followed the completion of a Data Safety Monitoring Board (DSMB) review of trial recruitment activity, and safety reporting from the first cohort that determined it was safe to proceed to cohort two. DSMBs are independent groups of experts appointed to periodically review information from clinical trials. These reviews typically include risk-benefit assessments and monitoring for serious or unexpected adverse safety events alongside any significant benefit of therapies.1 The LION-CS101 clinical trial is a double-blind, randomized, placebo-controlled, dose-escalation clinical trial to evaluate the safety of AB-1003 gene therapy in adult participants (18–65 years) who have genetic confirmation of LGMD2I/R9. The trial includes two sequential, dose-level cohorts. Adult participants diagnosed with LGMD2I/R9 will be given a single intravenous infusion of AB-1003 or placebo. The trial was initiated in 2023. It will include up to 14 participants at six sites throughout the United States.2 Participants in the first cohort remain in the study until completion. Enrollment in cohort two is ongoing. For more information about the LION-CS101 clinical trial, visit clinicaltrials.gov (NCT05230459) or askbio.com. “The burden of LGMD2I/R9 on patients and their families is profound,” said Nicholas Johnson, MD, Principal Investigator and Vice Chair of Research at the Department of Neurology, Virginia Commonwealth University School of Medicine. “Dosing the first participant in the second cohort of the trial is positive news for people living with LGMD2I/R9. This is a rare and debilitating type of muscular dystrophy, and this advancement brings the LION-CS101 trial another step closer to completion.” AskBio has received rare pediatric disease designation (RPDD), orphan-drug designation (ODD) and fast track designation (FTD) for AB-1003 for the potential treatment of LGMD2I/R9 from the United States Food and Drug Administration (FDA).3-4 These designations serve as clear recognition of the significant unmet medical need in LGMD2I/R9, for which there is no approved therapy.5 “The dosing of the first participant in cohort two marks an important milestone for the trial as enrollment continues for LION-CS101,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “We are encouraged by the DSMB’s recommendation to advance our study, following their thorough assessment of AB-1003 in cohort one and are excited to proceed with the second cohort.”   AB-1003 is an investigational recombinant adeno-associative virus (AAV)-based gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been fully established or evaluated. It is designed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9 as a one-time intravenous (IV) infusion.2-4 About Limb-Girdle Muscular Dystrophy Type 2I/R9 LGMD2I/R9 is a rare form of LGMD caused

Not intended for UK Media Berlin, Germany, and Research Triangle Park, N.C., USA, February 19, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that investigational gene therapy AB-1005 for the treatment of Parkinson’s disease (PD) has been granted Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration (FDA). “The FDA’s decision to grant RMAT designation to AB-1005 is exciting news for people living with Parkinson’s disease and their loved ones,” said Gustavo Pesquin, CEO, AskBio. “This milestone could potentially expedite the development of our important investigational gene therapy program, and it highlights our promising data and the potential of AB-1005 for patients and the medical community. We look forward to working closely with the FDA to accelerate our program.” The FDA determined that AB-1005, an investigational gene therapy intended to slow disease progression and improve motor outcomes in patients with PD, met the criteria for RMAT designation. This decision follows a review of information and data provided by AskBio, including clinical evidence from the open label, uncontrolled study Phase Ib trial of AB-1005. AskBio’s 36-month Phase Ib data showed that the administration of AB-1005 was well tolerated with no product-related serious adverse events.1 Further, the moderate PD cohort showed trends for improvement or stability on several PD-relevant clinical scales at 36 months compared to baseline, including Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and self-reported PD motor diaries, together with trends in reductions in Parkinson’s medications (levodopa-equivalent daily dose [LEDD]).1 Most participants in the mild PD cohort showed an overall stable clinical status with little change in MDS-UPDRS, the self-reported PD motor diary, or LEDD.1 RMAT is a designation granted by the FDA to regenerative therapies, including gene therapies, being developed to treat, modify, reverse, or cure serious or life-threatening diseases or conditions.2 Investigational products receiving this designation must have produced preliminary clinical evidence indicating that they may have the potential to address unmet medical needs for such diseases or conditions.2 RMAT provides recipients with enhanced access to the FDA, which could include intensive guidance on efficient drug development, rolling Biologics License Application (BLA) review, and other actions to expedite review.2 “The RMAT designation for AB-1005 underscores the high unmet medical need and the potential of this investigational gene therapy to make a difference for patients with Parkinson’s disease,” said Christian Rommel, Executive Vice President, Global Head of Research and Development and Member of the Pharmaceuticals Leadership Team at Bayer. “This is the latest example of what can be achieved through the joint commitment of AskBio and Bayer to deliver breakthrough innovation for patients.” The first participants in the AB-1005 Phase II REGENERATE-PD clinical trial have been randomized in the United States, and the trial is currently recruiting.3 Additional study sites in the United States, Germany, Poland, and the United Kingdom are expected to be opened for enrollment in first half of 2025.3 AB-1005 has not been approved by any

Berlin, Germany, and Research Triangle Park, N.C., USA, January 14, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the first participants have been randomized in its Phase II clinical trial in patients with Parkinson’s disease (PD). “The randomization of the first participants in REGENERATE-PD is positive news for people living with Parkinson’s disease and the physicians treating them,” said Dr Rajesh Pahwa, MD, Director, Parkinson’s Disease and Movement Disorder Center, University of Kansas Medical Center, USA, and REGENERATE-PD Principal Investigator. “There is a significant need for neurorestorative therapies in Parkinson’s and seeing the advancement of an important investigational gene therapy in a Phase II clinical trial gives hope to patients and the medical community.” In 2024, AskBio initiated recruitment in the United States arm of REGENERATE-PD.1 The objective of this randomized, double-blind, Phase II clinical trial is to evaluate the safety and efficacy of AB-1005 delivered to the putamen in adult participants aged 45–75 years with moderate-stage PD.2 REGENERATE-PD is estimated to enroll approximately 87 participants across clinical centers that are being opened in the United States, Germany, Poland, and the United Kingdom. AskBio also presented 36-month Phase Ib data at the International Congress of Parkinson’s Disease and Movement Disorders, which was held in Philadelphia, Pennsylvania, from September 27 to October 1, 2024.3 The data demonstrated that administration of AB-1005 was well tolerated with no attributed serious adverse events.3 The Moderate PD cohort showed trends for improvement or stability on several motor scales at 36 months, including Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and motor diaries, and trends in reductions in Parkinson’s medications (levodopa-equivalent daily dose [LEDD]).3 Most participants in the Mild PD cohort showed an overall stable clinical status with little change in MDS-UPDRS, the self-reported PD motor diary, or LEDD.3 “AskBio continues to mark significant milestones in the clinical development of the investigational gene therapy AB-1005 as we strive to bring a safe and effective gene therapy to patients with moderate stage Parkinson’s disease,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “With REGENERATE-PD participants now being randomized, we are excited by our progress with AB-1005 and look forward to sharing further updates at an appropriate scientific forum as the program advances through next year and beyond.” “The advancement of AskBio’s Parkinson’s program is a significant milestone that highlights our commitment to exploring the potential of gene therapy in an area of significant unmet medical need,” said Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division, and Global Head of Research and Development. “We believe that therapeutic modalities like gene therapy can change the treatment landscape for people living with the debilitating effects of Parkinson’s disease and look forward to seeing what we will learn in this next phase of research and development for AB-1005.” AskBio is also exploring AB-1005 beyond Parkinson’s disease and is currently enrolling participants in the United States with the