Gene Therapy, Startups, Venture

Orbimed partner in search of a cure for Parkinson’s launches new gene therapy player

Jonathan Silverstein is actively looking for a biotech miracle.

Jonathan Silverstein

The Orbimed partner formed a nonprofit group called the Silverstein Foundation after he was diagnosed with Parkinson’s, and now he’s launching a new biotech which he hopes can provide just that.

The new biotech is called Prevail Therapeutics. Based in New York, it will be run by Columbia University’s Asa Abeliovich, looking to develop gene therapies that will try to correct a disease that has proven to be one of the toughest targets in drug development. Orbimed and the foundation — which Silverstein is also funding — are backing the startup along with RegenXBio, one of the leaders in AAV tech used in the gene therapies now in clinical development.

The key focus at Prevail will be the GBA1 mutation carried by about 1 in 10 Parkinson’s patients. According to the biotech, the link between the GBA1 gene, glucocerebrosidase (GCase) protein production, and clearing out an accumulation of alpha-synuclein in the brain “may have implications for the broader Parkinson’s population, as well as in other neurodegenerative disease.”

The venture capitalist told CNBC that he’s seen biotechs like Biogen deliver miracles to patients, adding: “And … now I’ve got to create another miracle.”

Adds Silverstein:

We believe RegenXBio’s NAV Technology Platform provides us with a promising opportunity to develop potential life-changing therapies for patients suffering from Parkinson’s Disease and other neurodegenerative diseases. Dr. Abeliovich’s leadership and deep expertise in the molecular mechanisms of neurodegeneration uniquely position Prevail to make a significant mark in developing innovative biologic therapies for Parkinson’s Disease and other central nervous system diseases with huge unmet medical needs.


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