Passage Bio Establishes New Gene Therapy Development Program for Nerve Disorder

Passage Bio, a genetic medicines company developing adeno-associated virus (AAV)-delivered gene therapies for treating rare monogenic central nervous system (CNS) diseases, announced on Sept. 9, 2019 that it has licensed a sixth gene therapy development program under a research, collaboration, and license agreement between its Gene Therapy Program (GTP) and the University of Pennsylvania. The license is for the clinical development of a potential treatment for patients with Charcot-Marie-Tooth Neuropathy Type 2A (CMT2A).

The clinical trial is anticipated to be a global, open-label, multi-center, dose-escalation study to evaluate the safety, tolerability, and exploratory efficacy endpoints in subjects with CMT2A. CMT disease encompasses a heterogeneous group of inherited, progressive, chronic peripheral neuropathies. Many types affect the myelin sheath of nerves; CMT2 results from the degeneration of the axons of neurons. CMT2A, caused by dominantly inherited mutations in the MFN2 gene, is the most common type of CMT2. Patients with CMT2A usually begin to experience progressive weakness in childhood and most become wheelchair dependent, according to the company.

“CMT2A affects almost all of the severe dominant CMT2 cases, and patients suffering from this rare disease experience progressive muscle atrophy of legs and arms, with no FDA-approved curative or symptomatic medications available,” said Dr. Stephen Squinto, co-founder and interim CEO at Passage Bio, in a company press releaes. “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. Passage Bio will develop this experimental therapy, designed to restore the normal function of the MFN2 gene, which is mutated in patients with CMT2A, and we look forward to initiating a clinical trial in the near future.”

“Just one year after we formally launched our gene therapy program, we are witnessing two major players in the field working collaboratively to develop potential treatments for one of the more common types of CMT,” said Gilles Bouchard, chairman of the Charcot-Marie-Tooth Association, in the press release. “We are delighted to partner with Passage Bio and Penn in this effort and to contribute key elements of the Strategy to Accelerate Research (STAR) program, such as pre-clinical and clinical assets, access to top CMT experts, and engaging the CMT community.”

Source: Passage Bio