RGNX

REGENXBIO develops gene therapy treatments using adeno-associated virus (AAV) vectors, with a focus on rare genetic diseases affecting the eye, ear, and muscle. The company is advancing multiple product candidates through clinical trials in partnership with larger pharmaceutical companies like AbbVie and Nippon Shinyaku, though some programs face regulatory hurdles including clinical holds and a Complete Response Letter from the FDA. REGENXBIO is in mid-to-late stage development, with several ongoing clinical trials but no approved products currently on the market.

Pipeline

Catalyst Calendar