Regenxbio discovered novel RGX-202, a one-time gene therapy candidate for the treatment of Duchenne muscular dystrophy.
RGX-202 is designed to deliver a novel microdystrophin transgene containing functional elements of the C-terminal (CT) domain found in naturally occurring dystrophin.
RGX-202 is designed to support gene delivery and targeted expression across skeletal and cardiac muscle using the NAV AAV8 vector used in many clinical trials and a well- characterised muscle-specific promoter (Spc5-12).
Muscular dystrophy of Duchenne (Duchenne) is a rare genetic disorder, caused by mutations in the gene responsible for making dystrophin, a protein of central importance for muscle cell structure and function.
Globally, Duchenne disease mainly affects about 1 in 3,500 out of 5,000 men. Individuals with Duchenne experience muscle weakness and lose the ability to walk. Respiratory and heart muscles are also affected, causing breathing difficulties and the need for ventilator support, and it also reults in development of cardiomyopathy. Currently there exist no cure for Duchenne.
U.S. Food and Drug Administration (FDA) grants fast track designation for RGX-202, for the treatment of Duchenne muscular dystrophy (Duchenne).
