TSHA - Spinal Delivery Of Taysha's Gene Therapy Shows Promise In Rare Childhood Neurodegenerative Disease | Benzinga
An investigational gene therapy, dubbed scAAV9/JeT-GAN, for a rare neurodegenerative disease, giant axonal neuropathy (GAN), that begins in early childhood was well tolerated and showed signs of therapeutic benefit in a Phase 1 trial led by the National Institutes of Health (NIH).
The study was supported by NINDS and the National Institute of Arthritis and Musculoskeletal and Skin Diseases, Hannah’s Hope Fund, Taysha Gene Therapies Inc (NASDAQ:TSHA), Bamboo Therapeutics/Pfizer Inc (NYSE:PFE), Child Neurology Society, and the American Society of Gene and Cell Therapy.
Fourteen children with GAN, ages 6 to 14 years, were treated with gene transfer therapy and then followed for about six years to assess safety.
The gene therapy, which utilizes a modified virus to deliver functional copies of the defective ...