Category: Uncategorized

Not intended for UK Media Berlin, Germany, and Durham, N.C., USA, April 20, 2026 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that it has introduced its proprietary, commercially ready manufacturing process to supply ametefgene parvec (AB-1005), an investigational gene therapy for the treatment of Parkinson’s disease (PD) and multiple system atrophy-parkinsonian type (MSA-P), following submission of a United States Food and Drug Administration (FDA) Investigational New Drug Application (IND) amendment. This enables AskBio to now advance its REGENERATE-PD Phase II clinical trial in the United States with the material produced at the commercial manufacturing facility of AskBio’s wholly owned subsidiary, Viralgen, with other trial sites to follow, using an intensified, high-efficiency, next-generation, large-scale suspension manufacturing process that provides a consistently high-purity product. “Today’s news marks another important advancement in our Parkinson’s program,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio. “We are initiating supply with our manufacturing platform, to deliver our investigational gene therapy to participants in our ametefgene parvec trials, including our REGENERATE-PD trial, which recently saw the randomization of our first participants in Germany. The trial is currently enrolling participants in Poland, the United Kingdom, and the United States.” “Manufacturing efficiency of our AAV gene therapy-based drug product is mission critical for our Phase II Parkinson’s disease trial,” said Christian Rommel, PhD, Global Head of Research and Development for Bayer’s Pharmaceuticals Division. “It underscores the innovation of our production technology and reflects our continued commitment to meet high regulatory standards.” In December 2025, AskBio was granted Pioneering Regenerative Medical Product designation (SAKIGAKE) in Japan for ametefgene parvec, and in February 2025 the company was granted Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA.1,2 Prior to that, AskBio received FDA Fast Track designation and the Innovation Passport, the United Kingdom Medicines and Healthcare products Regulatory Agency (UK MHRA) innovative medicine designation.3 All designations were for PD. AskBio is also exploring ametefgene parvec in participants in the United States with the parkinsonian subtype of MSA-P in a fully enrolled Phase I clinical trial to assess the preliminary safety, tolerability, and efficacy for this rapidly progressing neurodegenerative condition.4 Ametefgene parvec 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 (PD) is a progressive neurodegenerative disease.5 It has a significant impact on a person’s daily life.5 In PD, the progressive death of dopamine producing nerve cells in the brain leads to the continuous loss of motor function.6 Symptoms include tremors, muscle rigidity, and slowness of movement.7 Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, cognitive issues, and depression.7 Symptoms typically intensify over time and make everyday tasks increasingly demanding.7 The prevalence of PD has doubled over the past 25 years.5 Today, more than 10 million people worldwide are estimated to be living with PD.8 This makes it the world’s

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