AskBio Inc., a gene therapy company operating independently under Bayer AG, has taken a key step forward in the development of its investigational Parkinson’s disease therapy by transitioning to a commercially ready manufacturing process. The company announced that its wholly owned subsidiary, Viralgen, will now supply commercial-scale drug product for the phase II REGENERATE-PD clinical trial of ametefgene parvec (AB-1005).
The move follows acceptance by the US Food and Drug Administration (FDA) of an amendment to AskBio’s Investigational New Drug (IND) application, allowing material produced at Viralgen’s large-scale manufacturing facility to be used in US clinical sites. The next-generation production platform is designed to improve efficiency and deliver a consistently high-purity adeno-associated virus (AAV) gene therapy product.
According to AskBio, the updated manufacturing approach supports continued global enrollment in the REGENERATE-PD study, which has already randomized participants in Germany and is actively recruiting in Poland, the United Kingdom, and the United States. Company leaders highlighted that reliable, scalable production is essential to supporting mid-stage clinical development and meeting regulatory expectations.
Ametefgene parvec is being investigated as a potential disease-modifying treatment for moderate-stage Parkinson’s disease, as well as for the parkinsonian subtype of multiple system atrophy (MSA-P). The therapy uses an AAV vector to deliver the gene for glial cell line–derived neurotrophic factor (GDNF), a protein shown in preclinical studies to support the survival and function of dopamine-producing neurons. The gene therapy is administered directly to targeted brain regions using a neurosurgical convection-enhanced delivery technique, with the goal of achieving long-term, localized expression of GDNF.
The REGENERATE-PD trial is a randomized, double-blind, surgery-controlled phase II study that plans to enroll approximately 127 adults aged 45–75 years with moderate Parkinson’s disease across Europe and the US. The study is designed to evaluate both the safety and potential efficacy of the approach in slowing disease progression and improving motor outcomes.
Beyond Parkinson’s disease, AskBio is also studying ametefgene parvec in a fully enrolled phase I trial involving patients in the US with MSA-P, a rare and rapidly progressive neurodegenerative disorder. This early-stage study is primarily assessing safety and tolerability, with preliminary efficacy measures included.
Regulatory agencies have shown interest in the program to date. Ametefgene parvec has received multiple expedited development designations for Parkinson’s disease, including FDA Fast Track and Regenerative Medicine Advanced Therapy (RMAT) status, the UK Medicines and Healthcare products Regulatory Agency’s Innovation Passport, and Japan’s SAKIGAKE designation. Despite these milestones, the therapy remains investigational and has not been approved by any regulatory authority.
Parkinson’s disease is the world’s second most common neurodegenerative disorder and continues to grow in prevalence, affecting more than 10 million people globally. Current treatments primarily manage symptoms and do not halt neuronal degeneration, highlighting the need for therapies that address underlying disease mechanisms. Similarly, MSA-P has no approved treatments and progresses rapidly after symptom onset.
Viralgen, founded in 2017, focuses exclusively on AAV-based gene therapy manufacturing and provides end-to-end support from development through commercial supply. AskBio, as part of Bayer’s broader life sciences portfolio, continues to position gene therapy as a potential option for both rare and prevalent neurological diseases as clinical testing progresses.
