Companies in #Gene Therapy (AAV)

Kriya Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing AAV-based gene therapies for common chronic diseases of high unmet need, including Type 1 diabetes, obesity, oncology, and rare diseases. Unlike most AAV gene therapy companies that focus on rare diseases with small patient populations, Kriya's strategic differentiation is targeting chronic conditions with much larger patient populations. Kriya operates its own in-house, large-scale GMP manufacturing facilities for gene therapy at its Research Triangle Park (Durham, North Carolina) headquarters — a notable strategic departure from the typical gene therapy CDMO-out-source model. The company was founded in 2019 by Shankar Ramaswamy, M.D. (former senior partner at McKinsey and former CEO of Axovant) and has raised over $600M in venture funding. Kriya was selected in June 2026 for the FDA PreCheck Pilot Program for its Durham, NC manufacturing facility.

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.