According to a story from BioSpace, the genetic medicines company Passage Bio recently announced plans for its sixth program for gene therapy development. The company plans to work with the Gene Therapy Program at the University of Pennsylvania. The target disease for this program will be Charcot-Marie-Tooth disease type 2A. Passage Bio is focused on the development of gene therapies for monogenic rare diseases that affect the central nervous system.
About Charcot-Marie-Tooth Disease
Charcot-Marie-Tooth disease is a hereditary disorder of the peripheral nervous system. It is most characterized by a progressive loss of touch sensation and muscle tissue in several different parts of the body. The cause of this disease is usually linked to a genetic mutation, but the mutation involved varies depending on the variant of Charcot-Marie-Tooth disease. There are multiple types of Charcot-Marie-Tooth disease, with all types aside from type 2 having a demyelinization effect. Type 2 causes damage to the neuronal axon instead. Symptoms include foot drop, muscle wasting (typically in the arms, legs, and hands), painful muscle spasms, loss of sensation in the limbs, scoliosis, trouble speaking, chewing, and swallowing, and tremors. Treatment typically includes therapy and surgery in order to maintain function. There is no cure. The disease can occur early in life or as late as the 30s and 40s. To learn more about Charcot-Marie-Tooth disease, click here.
The type 2A variant of this illness generally presents with less severe symptoms than type 1 and tends to affect the lower limbs more than other areas of the body.
New Hope in Gene Therapy
With the current lack of treatments available for this disease, patients will continue to see disease progression throughout their lifetimes and will ultimately become wheelchair dependent. It is clear that more effective therapies for Charcot-Marie-Tooth disease type 2A are urgently needed. Passage will look to delivery systems and AAV vectors developed at the University of Pennsylvania as the basis for its investigational gene therapy. A successful gene therapy would have the potential to slow or even halt the progression of symptoms in patients with the disease.
