AskBio Inc., a gene therapy subsidiary of pharmaceutical giant Bayer AG, has taken a significant step forward in advancing treatment options for Parkinson’s disease. According to PharmaBiz.com, the company announced that it has successfully implemented its proprietary commercial-scale manufacturing process to supply ametefgene parvec (AB-1005), an investigational gene therapy designed to treat both Parkinson’s disease and a related condition called multiple system atrophy-parkinsonian type (MSA-P). This manufacturing milestone follows approval of an FDA Investigational New Drug Application amendment and represents a critical progression in bringing innovative therapies to patients.
Manufacturing Innovation Drives Clinical Progress
The manufacturing process leverages Viralgen, AskBio’s wholly owned subsidiary and contract development and manufacturing organization specializing in adeno-associated virus (AAV)-based gene therapies. Viralgen’s newly implemented system utilizes an intensified, high-efficiency, next-generation large-scale suspension manufacturing process that ensures consistently high purity products. This technological advancement enables the company to supply sufficient quantities of the drug for the ongoing REGENERATE-PD phase II clinical trial across multiple international sites.
“Manufacturing efficiency is mission critical for our phase II Parkinson’s disease trial,” explained Christian Rommel, PhD, global head of research and development for Bayer’s pharmaceuticals division. The company’s ability to produce commercial-grade material reflects both technological innovation and commitment to rigorous regulatory standards.
Trial Expansion and Regulatory Recognition
The REGENERATE-PD trial represents a randomized, double-blind, surgery-controlled study evaluating ametefgene parvec’s efficacy and safety in treating adults aged 45–75 years with moderate-stage Parkinson’s disease. The trial, which recently saw its first participant randomized in Germany, is actively enrolling across four countries: Germany, Poland, the United Kingdom, and the United States. With an estimated 127 participants, this trial builds on promising results from earlier-stage studies.
Ametefgene parvec has earned multiple regulatory designations acknowledging its potential significance. In December 2025, it received Japan’s Pioneering Regenerative Medical Product designation (SAKIGAKE), while the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation in February 2025. The therapy also holds FDA Fast Track designation and the UK Medicines and Healthcare products Regulatory Agency’s innovative medicine designation.
How the Therapy Works
Ametefgene parvec operates through a sophisticated mechanism: it comprises an adeno-associated viral vector carrying the human glial cell line-derived neurotrophic factor (GDNF) transgene. Delivered directly into targeted brain regions via neurosurgery with convection-enhanced delivery, this approach enables stable, continuous GDNF expression. Preclinical research demonstrates that GDNF promotes survival and morphological differentiation of dopaminergic neurons—the cells progressively lost in Parkinson’s disease—potentially preserving and restoring the neural circuitry damaged by disease progression.
Addressing Critical Medical Need
Parkinson’s disease, affecting over 10 million people worldwide, represents the second most prevalent neurodegenerative disease. Its prevalence has doubled in 25 years, and current treatments fail to comprehensively manage symptoms while disease progression continues. Similarly, MSA-P affects approximately 400,000 people globally with no approved treatments, creating urgent demand for innovative therapeutic approaches.
This manufacturing advancement demonstrates AskBio’s commitment to translating gene therapy innovation into accessible treatments while maintaining the highest quality and regulatory standards.
