Parkinson’s startup Prevail raises $75M for gene therapy programs

OrbiMed-backed Prevail Therapeutics has launched a $75 million first-round financing that will help fund development of its gene therapy for Parkinson’s disease (PD) and other programs.

Prevail was set up last year by OrbiMed's co-head of private equity Jonathan Silverstein, with seed funding from the investment firm as well as a nonprofit research foundation formed by Silverstein to tackle PD caused by mutations in the glucocerebrosidase (GBA1) gene after he was diagnosed with the illness in 2016.

OrbiMed has participated in the series A financing as well, along with Pontifax Fund, RA Capital Management, EcoR1 Capital, Omega Funds, BVF Partners, Boxer Capital, Adage Capital Management and Alexandria Venture Investments.

Prevail’s co-founder and CEO, Asa Abeliovich, M.D., Ph.D., who is on the faculty of Columbia University and whose lab conducted a chunk of the research underpinning the company’s research programs, said the first round “supports out plan to develop a pipeline of gene therapies with the potential to dramatically improve the lives of patients with a range of genetically defined neurodegenerative diseases.”

The common thread is to target neurodegenerative diseases caused by lysosomal dysfunction, a pathology seen in various forms of PD, including early-onset variants, as well as some types of Alzheimer's disease and frontotemporal dementia.

Prevail has also taken a license to an adeno-associated virus (AAV) gene therapy vector technology developed by Regenxbio called NAV AAV9. This will be used across its gene therapy candidates including lead program PR001, which is being developed for GBA1-linked PD, a variant thought to affect around 10% of all patients with the disease. The hope is that therapies developed for this subset of patients may also have a role more broadly in treating PD.

For now, Prevail isn’t taking about timelines to start trials of PR001 in humans, but a spokesman said the company is “focused on advancing the program as fast as they can.”