REGENXBIO Inc. announced positive safety and effectiveness data from the Phase 1/2/3 AFFINITY DUCHENNE® study of RGX-202, a gene therapy that uses an adeno-associated viral (AAV; a virus that does not cause disease and acts like a package for the healthy gene) vector to deliver a transgene (a manufactured version of a piece of DNA) to deliver a novel microdystrophin which is also in naturally-occurring dystrophin. RGX-202 is a one-time treatment designed to specifically address underlying cause of Duchenne muscular dystrophy, allowing a person with DMD to produce microdystrophin, and prevent damage to skeletal and heart muscles. In the current study:

• RGX-202 has been well tolerated in 12 patients across both dose levels 
• New biomarker data continues to support consistent robust expression, transduction and appropriate localization at the sarcolemma (muscle cell membrane) of the RGX-202 microdystrophin across all ages.
• the first five boys dosed in the Phase 1/2 portion of the AFFINITY DUCHENNE trial showed functional improvements that exceeded available external natural history controls measured at 9 months (dose level 2) and 12 months (dose level 1).

See the full press release and community letter below.

Want to know more about Duchenne Research?

Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: