Gene therapy proved to be safe in patients with Parkinson’s disease

Parkinson's disease

Askbio’s AB-1005 (also known as AAV2-GDNF), an investigational gene therapy for the treatment of Parkinson’s disease, was well tolerated with no serious adverse events in a Phase Ib clinical trial.

The data were presented at the American Academy of Neurology 2024 Annual Meeting in Denver, Colorado, USA.

Based on the safety and efficacy results, a Phase II trial (REGENERATE PD) has been developed and is expected to begin enrolling in the US, EU, and UK later this year.

The study met its primary objective, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. Bilateral infusions of AB-1005 within the putamen (up to 1.8 mL) were well tolerated, with no SAEs associated with the investigational gene therapy or contrast agent.

Neurosurgical delivery of AB-1005 resulted in putamen coverage of 63% ± 2%, exceeding the goal of greater than 50% coverage with AB-1005.

Transformative impact for Parkinson’s patients

“These early findings are encouraging and show AB-1005 to be well tolerated in this study in patients with mild to moderate Parkinson’s disease,” said Krystof Bankiewicz, Scientific Chair, Parkinson’s and MSA, AskBio. “Further, they highlight areas of potential future exploration in our upcoming Phase II REGENERATE PD trial, which will look more closely at the potential efficacy of AB-1005 in the treatment of Parkinson’s disease.”

Most patients in the Mild Cohort achieved overall clinical stability with little change in MDS-UPDRS and PD Motor Diary outcomes. Participants in the Moderate Cohort achieved clinical motor improvement at 18 months.

Asklepios BioPharmaceutical (AskBio) is a wholly owned and independently operated subsidiary of Bayer.

Christian Rommel, Head of Research and Development at Bayer’s Pharmaceuticals Division, added: “While much remains to be done, we recognise with increasing confidence the potential of AB-1005 to provide a transformative impact for patients in the future.”

Diana Spencer, Senior Digital Content Editor, DDW

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