A single treatment with the potential
for long-lasting results
RGX 202 is an investigational gene therapy utilizing a novel microdystrophin construct for the treatment of Duchenne Muscular Dystrophy (DMD).
DMD is a severe, progressive, degenerative muscle disease caused by mutations in the DMD gene, which encodes dystrophin, a protein involved in muscle cell structure and function. Without functional dystrophin protein, muscles throughout the body degenerate and become weak. Individuals with DMD experience progressive muscle weakness and eventually lose the ability to walk. Respiratory and heart muscles are also impacted, leading to the need for ventilator-assisted breathing along with the development of cardiomyopathy.
RGX-202 utilizes REGENXBIO’s propriety NAV AAV8 vector to deliver a gene to muscle cells that encodes for microdystrophin, a shortened and functional form of the dystrophin protein. Microdystrophin is targeted as the functional protein since the gene that encodes for full-length dystrophin is too large to fit into the AAV vector. The novel RGX-202 microdystrophin transgene includes coding regions that retain essential functional elements of naturally occurring dystrophin, including a unique C-Terminal (CT) domain for potential improved function. A well-characterized muscle-specific promoter (Spc5-12) is employed to direct expression of microdystrophin protein in skeletal and heart muscles. Additional RGX-202 features are designed to improve gene expression and reduce immunogenicity.
There is presently no cure for DMD. Currently approved treatments either do not address the underlying cause of DMD or are only applicable to a subset of patients with specific genetic mutations. One-time administration of RGX-202 aims to address the underlying cause of DMD, independent of genetic mutation, by potentially enabling production of microdystrophin in muscle cells to protect them from damage and ultimately preserve muscle function.
RGX-202 is in the preclinical phase of development. We are currently completing Investigational New Drug (IND) application enabling studies, and plan to submit an IND for a first-in-human clinical trial in mid-2021.