RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, per interim data.

expressive language and fine motor skill acquisition than would be expected if untreated. The subject from the initial study of RGX-111 that led to FDA approval of the phase 1/2 trial also showed ...

Its product candidates include the NAV Technology Platform, which consists of exclusive rights to novel adeno-associated viral vectors, and therapeutic programs such as RGX-314, RGX-202 ...

"RGX-121 and RGX-111 represent potentially transformative new medicines for patient populations in great need of new options," said Curran M. Simpson, President and CEO of REGENXBIO. "This ...