17/09/2026
Gene Therapies in China: Products & Indications
AAV gene therapy — RPE65 inherited retinal dystrophy
AAV gene therapy — RDH12 inherited retinal disease
ZVS101e — Bietti crystalline retinal dystrophy
EXG102‑031 — Wet age-related macular degeneration
AAV gene therapy — Hemophilia A
Gene-edited autologous hematopoietic stem cell therapy — Transfusion-dependent beta-thalassemia
Vutrisiran (siRNA) — ATTR amyloidosis
Gene-modified therapy — SOD1-mutated ALS
Bopidagene injection (Xinjiuning®) — Moderate to severe Hemophilia B
Notes
Items 1–8: Available only in Boao Lecheng Pilot Zone.
Item 9: Nationwide NMPA approved.
MDT assessment required for eligibility.
CAR-T and stem cell therapies are not gene therapies and excluded.