Rare Daily Staff

Passage Bio said that it has licensed an experimental gene therapy program for patients with Charcot-Marie-Tooth Neuropathy Type 2A, under its research, collaboration, and license agreement with the University of Pennsylvania and its Gene Therapy Program.

Charcot-Marie-Tooth (CMT) disease is a group of disorders that affect the peripheral nerves that run from outside the brain and the spine. Defects in at least 30 genes cause different forms of this disease. Common symptoms may include foot drop, foot deformity, loss of lower leg muscle, numbness in the foot or leg, and weakness of the hips, legs, or feet. CMT type 2A, 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.

Passage Bio will develop the experimental therapy, designed to restore the normal function of the MFN2 gene, which is mutated in patients with CMT2A. The CMT gene therapy license comes less than a week after Passage Bio announced completion of a $110 million series B financing round.

“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 Stephen Squinto, co-founder and interim CEO at Passage Bio. “The Gene Therapy Program (GTP) 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.”

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. The Charcot-Marie-Tooth Association is partnering with Passage Bio on the trial by contributing certain preclinical and clinical assets to the trial, providing access to CMT experts, and engaging the CMT community.

“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.

Under Passage Bio’s agreement with UPenn, GTP, and the Orphan Disease Center at Penn, Passage Bio licensed five gene therapy programs with the option of licensing seven more. The CMT gene therapy program license is the sixth program licensed through the agreement. As part of their deal, the GTP conducts the IND-enabling preclinical work and Passage Bio conducts all clinical development, regulatory strategy and commercialization activities.

Photo: Stephen Squinto, co-founder and interim chief executive officer at Passage Bio

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