University of Pennsylvania Inks Deal with Passage Bio for Gene Therapy Rights Involving Charcot-Marie-Tooth Neuropathy Type 2A (CMT2A)

Sep 13, 2019 | Charcot-Marie-Tooth Neuropathy Type 2A (CMT2A), Gene Therapy, Passage Bio, Penn, Rare Diseases

Study of DNA molecules by a doctor .

The University of Pennsylvania (Penn) and its Gene Therapy Program (GTP) has licensed its sixth therapy to brand new start-up biotech venture Passage Bio, a developer of gene therapies for rare monogenic CNS diseases. The deal includes a preclinical treatment for Charcot-Marie-Tooth Neuropathy Type 2A (CMT2A). Passage Bio had the funds ready as just days earlier they secured $110 million Series B financing—they have raised a total of $225.5 million.

Clinical Trials

Passage Bio seeks to take the Penn developed gene therapy intellectual property (IP) into clinical trials for patients with CMT2A shortly reports Patricia Inacio of Charcot-Marie-Tooth News. The gene technology breakthroughs are based on pioneering work done by Dr. James Wilson and Penn’s Wilson Lab

What is CMTA2?

Ms. Inacio reports that CMT2A is caused by mutations in the MFN2, a gene that directs a protein called mitofusin 2, which is involved in the fusion of mitochondria, determining their form. Although mutations in the MFN2 gene are known and several research initiatives to address are underway, no treatments are addressing CMT2A.

Emerging CMT2A-focused gene therapies deliver non-mutated versions of the mutated gene into patients’ cells. The primary vehicle for delivery is the adeno-associated virus (AAV). Over the past couple years there have been significant advancements in vector design enabling widespread gene delivery in the brain and spinal cord supporting research in neurological diseases.

Passage Bio

Founded in 2018, Passage Bio has already raised $225.5 million. Based in Philadelphia, they are deeply connected to innovative researching coming out of Penn. They position the company as a fully integrated genetic medicines company developing a portfolio of six life-transforming AAV-delivered therapeutics for the treatment of rare monogenic central nervous system diseases. Founded by industry veterans Stephen Squinto, Ph.D. and Tachi Yamada, MD in a partnership with gene therapy pioneer James Wilson, MD, Ph.D. Based in Penn, Dr. Wilson runs a lab focusing on gene therapy discovery research and next-generation gene transfer vectors and their application in the treatment of a variety of acquired and inherited diseases.

Call to Action: For those interested in gene therapies we recommend keeping an eye on Passage Bio and the Penn’s Wilson Lab


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