AskBio Receives Approval on Regenerative Medicine for Investigational Parkinson’s Disease Treatment
Thursday, February 20, 2025
AskBio has received Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration (FDA) for AB-1005, an investigational gene therapy for Parkinson’s disease (PD).
Parkinson’s disease is a progressive neurodegenerative disorder affecting motor and non-motor functions. It results from the degeneration of dopamine-producing neurons in the brain, leading to symptoms such as tremors, muscle rigidity, and slow movement.
No cure exists, and current treatments primarily focus on symptom management, highlighting the need for new therapies.
The Phase Ib trial evaluated the safety and potential clinical benefits of AB-1005 in 11 patients with mild to moderate PD. The therapy was administered through a one-time bilateral convection-enhanced delivery to the putamen.
REGENERATE-PD is a Phase II, randomised, double-blind, sham-controlled trial evaluating the efficacy and safety of AB-1005 in adults aged 45-75 with moderate PD.
AB-1005 is an investigational gene therapy using an adeno-associated viral vector (AAV2) to deliver the glial cell line-derived neurotrophic factor (GDNF) transgene to specific brain regions. This approach aims to promote dopamine neuron survival and function.
This designation recognises the potential of AB-1005 to address unmet medical needs in the treatment of PD and may accelerate its development and regulatory review.
AB-1005 aims to slow disease progression and improve motor function in PD patients.
Additionally, trends in reduced use of Parkinson’s medications were observed. In the mild PD cohort, most participants maintained a stable clinical condition.
RMAT designation is granted to regenerative therapies, including gene therapies, that show preliminary clinical evidence of potential effectiveness in treating serious conditions.
This status offers enhanced engagement with the FDA, including intensive guidance on drug development, rolling Biologics License Application (BLA) review, and expedited regulatory processes.
AskBio has initiated the Phase II REGENERATE-PD clinical trial to assess the efficacy and safety of AB-1005 in patients with moderate PD.
The study has begun randomising participants in the United States, with recruitment planned in Germany, Poland, and the United Kingdom in the first half of 2025.
Source: askbio.com
