AskBio Inc 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 US Food and Drug Administration (FDA) Investigational New Drug Application (INDA) amendment. 

This enables the company to now advance its REGENERATE-PD phase-II clinical trial in the US 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, 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 randomisation of our first participants in Germany. The trial is currently enrolling participants in Poland, the United Kingdom, and the United States.” 

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.

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. All designations were for PD. 

“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.”

AskBio is also exploring ametefgene parvec in participants in the US 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.

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.