Companies in #Gene Therapy (AAV)
Found 2 companies operating in this sector.
REGENXBIO is a clinical-stage biotechnology company developing gene therapies using its proprietary AAV (adeno-associated virus) NAV technology platform. The company is advancing a pipeline of one-time gene therapies for rare and serious diseases including Duchenne muscular dystrophy (RGX-202), Hunter syndrome (RGX-121), and wet age-related macular degeneration (RGX-314, partnered with AbbVie).
uniQure N.V. is a gene therapy company developing a single administration of AAV-based therapies to treat serious genetic diseases, with a portfolio spanning hemophilia, Huntington's disease, ALS, Fabry disease, and refractory epilepsy. The company developed the first FDA-approved hemophilia B gene therapy (Hemgenix, partnered with CSL Behring) and is advancing AMT-130, a one-time gene therapy for Huntington's disease, as its lead wholly-owned program.