Tech Transfer eNews Blog

Penn start-up advances gene therapy to treat rare CNS diseases

By Jesse Schwartz
Published: September 11th, 2019

A University of Pennsylvania start-up is advancing a series of gene therapy candidates to treat rare central nervous system (CNS) diseases.

Passage Bio recently licensed its sixth gene therapy, a preclinical treatment for Charcot-Marie-Tooth Neuropathy Type 2A (CMT2A), from Penn and its Gene Therapy Program (GTP). According to the start-up, the gene therapy is designed to restore the normal function of the MFN2 gene, which is mutated in cases of CMT2A.

Under the licensing agreement, GTP will conduct IND-enabling preclinical work on the therapy, while Passage Bio will conduct all clinical development, regulatory strategy, and commercialization activities.

The start-up has completed a $110 million Series B financing and in total has raised $225.5 million. It aims to launch a clinical trial for the CMT2A gene therapy in 2021.

“The Gene Therapy Program at Penn has developed AAV vectors and delivery methods to target the nerve cells that are affected in CMT2A, raising the possibility of slowing or preventing progression of the disease by tackling the underlying genetic cause,” says Stephen Squinto, co-founder and interim CEO of Passage Bio. “We look forward to initiating a clinical trial in the near future.”

Source: Genetic Engineering & Biotechnology News

Posted under: Tech Transfer e-News

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