Prevail moves toward clinic for gene therapy in Parkinson’s, nerve disorders

A company developing gene therapies for neurodegenerative diseases in March closed a round of financing that it hopes will bring it into clinical development.

New York-based Prevail Therapeutics said it had raised $50 million in a Series B round, with Surveyor Capital and AbbVie Ventures joining a list of investors that also includes OrbiMed, Pontifax, RA Capital Management, EcoR1 Capital, Omega Funds, BVF Partners, Boxer Capital, Adage Capital Management and Alexandria Venture Investments. The company, which launched in 2017, has raised $129 million to date, it said.

According to its website, the company’s focus is on developing adeno-associated viral vector-based gene therapies for diseases like Parkinson’s disease. Its hypothesis is that by restoring healthy lysosomal function to the cells of a patient’s nervous system, it can stop the progression of a disease. To that end, it has in-licensed Regenxbio’s NAV AAV9 vector for Parkinson’s and other, related diseases.

The only currently approved gene therapy, Spark Therapeutics’ Luxturna (voretigene neparvovec), for a rare form of blindness, is also based on an AAV vector, whereby specially engineered viruses transfect a cell and insert a corrected gene into them. Spark is also exploring gene therapies for neurodegenerative and central nervous system diseases, particularly CLN2 disease and Huntington’s disease, along with undisclosed diseases, all of which are in preclinical development.

Other efforts to develop viral vector-based gene therapies for Parkinson’s disease have taken place as well, both commercially and academically. One company developing such a therapy is Voyager Therapeutics, which has an AAV-based gene therapy, VY-AADC, in Phase II development, under a partnership with Neurocrine Biosciences. In the 42-patient trial, participants are randomized in a double-blind fashion to receive either the gene therapy or placebo via a surgical procedure.

On the academic front, Japan’s Jichi Medical University has a Phase I/II study of an AAV-based gene therapy that is enrolling six patients and is set to complete in January 2022.

Prevail Therapeutics raises $50M for gene therapy in Parkinson’s, other neurodegenerative disorders