AskBio Advances Commercial-Scale Manufacturing for Parkinson's Gene Therapy in Phase II Trial
核心洞察
AskBio has introduced its commercial manufacturing process to supply ametefgene parvec (搜索) (AB-1005) for the REGENERATE-PD Phase II trial following FDA IND amendment approval.
The investigational gene therapy delivers GDNF (搜索) protein to the brain via adeno-associated viral vector to potentially slow Parkinson's disease (搜索) progression and improve motor outcomes.
REGENERATE-PD is enrolling 127 participants aged 45-75 with moderate-stage Parkinson's disease (搜索) across sites in Germany, Poland, the United Kingdom, and the United States.
AskBio Inc., a Bayer AG subsidiary, has successfully transitioned to commercial-scale manufacturing for its investigational Parkinson's disease (搜索) gene therapy ametefgene parvec (搜索) (AB-1005), marking a significant milestone in the company's Phase II clinical development program. The advancement follows approval of a United States Food and Drug Administration (FDA) Investigational New Drug Application (IND) amendment, enabling the company to supply its REGENERATE-PD trial with material produced at Viralgen (搜索)'s commercial manufacturing facility.
Manufacturing Platform Enables Trial Progression
The transition to commercial manufacturing represents a critical operational achievement for AskBio's Parkinson's program. "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 (搜索) trials, including our REGENERATE-PD trial, which recently saw the randomization of our first participants in Germany."
Viralgen (搜索), AskBio's wholly owned subsidiary, utilizes an intensified, high-efficiency, next-generation large-scale suspension manufacturing process that provides consistently high purity product. The San Sebastián, Spain-based facility features three cGMP suites with 2,000-liter capacity each, certified by the AEMPS/EMA, and has produced more than 1,200 AAV batches to date.
GDNF Gene Therapy Mechanism and Clinical Rationale
Ametefgene parvec (搜索) is composed of an adeno-associated viral vector containing the human glial cell line-derived neurotrophic factor (GDNF (搜索)) transgene. The therapy allows for stable and continuous expression of GDNF in localized regions of the brain after direct neurosurgical injection with convection-enhanced delivery. In nonclinical studies, GDNF has been shown to promote the survival and morphological differentiation of dopaminergic neurons (搜索), which could aid in the preservation and restoration of dopaminergic neuronal circuitries normally lost in Parkinson's disease (搜索).
"Manufacturing efficiency of our AAV gene therapy-based drug product is mission critical for our Phase II Parkinson's disease (搜索) 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."
REGENERATE-PD Trial Design and Patient Population
REGENERATE-PD is a Phase II, randomized, double-blind, surgery controlled trial evaluating the efficacy and safety of intraputaminal ametefgene parvec (搜索) in adults aged 45-75 years with moderate-stage Parkinson's disease (搜索). The trial will enroll an estimated 127 participants across sites in Germany, Poland, the United Kingdom, and the United States. The study is currently enrolling participants in Poland, the United Kingdom, and the United States, with first participants already randomized in Germany.
Regulatory Recognition and Broader Development Program
The investigational therapy has garnered significant regulatory attention across multiple jurisdictions. In December 2025, AskBio received Pioneering Regenerative Medical Product designation (SAKIGAKE) in Japan for ametefgene parvec (搜索). In February 2025, the company was granted Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. Previously, AskBio received FDA Fast Track designation and the Innovation Passport from the United Kingdom Medicines and Healthcare products Regulatory Agency (UK MHRA).
Beyond Parkinson's disease (搜索), AskBio is exploring ametefgene parvec (搜索) in participants with the parkinsonian subtype of multiple system atrophy (搜索) (MSA-P) in a fully enrolled Phase I clinical trial in the United States. MSA-P affects approximately 400,000 people worldwide and is characterized by rapid progression within 5-10 years of symptom onset, with no currently approved treatments available.
Addressing Unmet Medical Need
Parkinson's disease (搜索) represents a substantial global health burden, with prevalence doubling over the past 25 years. More than 10 million people worldwide are estimated to be living with the condition, making it the world's second most prevalent neurodegenerative disease and the most frequent movement disorder. The progressive death of dopamine-producing nerve cells in the brain leads to continuous loss of motor function, with symptoms including tremors, muscle rigidity, and slowness of movement, as well as non-motor symptoms such as fatigue, cognitive issues, and depression.
Current treatment options lack holistic symptom management, creating an urgent need for new therapeutic approaches. Through its combination of investigational gene therapy and innovative neurosurgical delivery, AskBio aims to test the GDNF (搜索) hypothesis by delivering this neurotrophic factor to degenerating nigrostriatal neurons in a well-controlled clinical study to understand the effects of GDNF in a moderate Parkinson's disease (搜索) population.