Gene Therapy
Approaches that add, regulate or edit genetic material to address a molecular cause of disease.
How the treatment works
Gene therapy may supply a functional gene, regulate gene expression or edit a specific DNA sequence. These are different approaches. Delivering a working gene with a viral vector is not the same as repairing the patient’s existing mutation. Eligibility usually depends on an exact genetic diagnosis, organ function and the criteria for the particular product or study.
Clinical use in China
China has approved gene therapies in commercial clinical use. BBM-H901, approved in 2025 for eligible adults with moderate-to-severe haemophilia B, uses an AAV vector to deliver a functional factor IX gene. A September 2026 first-hand report described a commercial prescription in Tianjin, documenting a pathway from development into patient care.
Shanghai’s hearing-loss research
A notable Shanghai programme addresses hereditary deafness. Professor Yilai Shu’s team at Fudan University’s Eye & ENT Hospital led a multicentre study of OTOF-related congenital deafness published in Nature in April 2026, with follow-up extending to 2.5 years. Further work published in June investigated sequential administration to the other ear.
These studies combine genetic diagnosis, dual-AAV delivery and specialised otological procedures. They remain clinical research and should not be presented as a routine treatment that anyone with hearing loss can book.
Product-specific costs
Gene-therapy costs vary substantially by product. Public reporting in September 2026 described a reduced price of about RMB 1.39 million for the haemophilia B product. Payment arrangements, eligibility and what is included must be confirmed for the individual product and treating institution.