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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Prevail to initiate gene therapy clinical trial for Parkinson’s disease

The FDA has accepted Prevail Therapeutics’ Investigational New Drug application (IND) to initiate a Phase 1/2 gene therapy clinical trial for Parkinson’s disease (PD).

The trial will use Prevail’s lead program PR001, an AAV9-based gene therapy, to treat PD patients with glucocerebrosidase (GBA) mutation, a variant thought to affect around 10% of all patients with PD.

Earlier this year, the company had raised $129 million to advance its pipeline of AAV-based gene therapies to target lysosomal dysfunction and treat patients with neurodegenerative diseases.

Headquartered in New York, Prevail was launched in 2017 by Dr Asa Abeliovich (Columbia University), OrbiMed’s co-head of private equity Jonathan Silverstein along with Regenxbio and The Silverstein Foundation for Parkinson’s with GBA, a non-profit research foundation formed by Silverstein after he was diagnosed with Parkinson’s in 2016. Prevail holds a license to use the AAV9 vector which was originally developed by Regenxbio.

Prevail plans to initiate dosing of patients later this year and the trial will evaluate the safety and tolerability of PR001.

Source: Potential One-time Gene Therapy for Parkinson’s Linked to GBA Mutations to Enter Clinical trial; Press Release

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