Seven months after Prevail Therapeutics launched the New York-based company has secured $75 million in Series A financing to support the development of gene therapies for Parkinson’s disease and other neurodegenerative diseases.

The proceeds from the Series A round will be used to advance the company’s pipeline of AAV-based (adeno-associated virus) therapeutics targeting lysosomal dysfunction to treat patients with genetically-defined neurodegenerative diseases. Prevail’s lead therapy PR001, will be developed for a genetic subset of Parkinson’s disease and other mechanistically-related disorders, the company said.

Parkinson’s disease is a neurodegenerative disorder. It affects dopaminergic neurons in a part of the brain. Disease symptoms typically develop slowly and could begin with slight tremors in the hands, slowness of movement or limb rigidity. Symptoms include tremor, slowed movement, rigid muscles, impaired posture and balance, loss of automatic movements and speech changes. Several risk factors for Parkinson’s have been identified, including glucocerebrosidase (GBA1) gene mutation. The GBA1 mutation is the most common of Parkinson’s disease mutations. It accounts for about 10 percent of all Parkinson’s disease cases in the United States. The disease affects more than 10 million people worldwide. There is no cure for Parkinson’s disease.

Prevail Chief Executive Officer Asa Abeliovich reiterated that the $75 million financing round will be instrumental in developing gene therapies that have the potential to improve the lives of patients with a range of genetically-defined neurodegenerative diseases.

"Our lead program will target patients with Parkinson’s disease, and we plan to address additional disease areas where lysosomal dysfunction is an underlying cause," Abeliovich said in a statement.

Investors in the Series A financing round include OrbiMed, Pontifax Fund, RA Capital Management, EcoR1 Capital, Omega Funds, BVF Partners L.P., Boxer Capital, LLC, Adage Capital Management L.P., and Alexandria Venture Investments. OrbiMed and the Silverstein Foundation for Parkinson’s with GBA provided the seed funding to launch Prevail last year. As BioSpace previously reported Jonathan Silverstein, a partner and co-head of OrbiMed, was diagnosed with Parkinson’s disease at the age of 50.

"Prevail’s mission to develop gene therapies that address the underlying genetic causes of neurodegenerative disease is aligned with our commitment to identify treatments that can halt the progression of Parkinson’s and other diseases. The company has assembled an excellent team to accomplish this mission, including executive management, scientific advisors, and board members, and I look forward to working with them to make a difference for patients," Silverstein said in a statement.

When the company launched last year it did so with an exclusive licensing agreement with REGENEXBIO. Prevail will develop and commercialize gene therapy products using REGENXBIO’s NAV AAV9 vector for the treatment of Parkinson’s disease and other related neurodegenerative diseases.

The GBA1 mutation is a popular target for Parkinson’s disease research. Several companies have developmental programs in place, including Sanofi US and Allergan, Inc..

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