Gene therapy for blood disorders
Gene therapy adds a working copy of a faulty gene. China approved a one-time gene therapy for haemophilia B in April 2025, and gene therapy programmes for thalassaemia are available.
Who it may suit
- Adults with haemophilia B who meet antibody and liver criteria
- People with transfusion-dependent thalassaemia (programme criteria apply)
- Patients able to attend long-term follow-up
What happens
- Blood tests, including screening for antibodies to the virus carrier
- Haemophilia B: a single infusion, then weekly blood tests
- Thalassaemia: stem-cell collection, chemotherapy, then return of the modified cells
- Follow-up plan agreed with your home haematologist
Haemophilia B: about 2 to 4 weeks. Thalassaemia: about 2 to 3 months.
Similar Western gene therapies list at around US$2–3.5 million.
Things to know. Liver enzymes can rise and may need treatment. Thalassaemia programmes involve chemotherapy and its risks. Long-term effects are still being studied.
Why an introduction matters
Many of China’s leading specialists work in busy public hospitals without international patient offices. They rarely answer enquiries from abroad directly, and a file sent without an introduction often goes unread. Cases usually reach them through people they already know and trust. Our team on the ground makes that introduction, presents your case properly in Chinese, and follows up until you have an answer.
Some programmes, especially stem-cell and other cell treatments, are experimental. We introduce them only if you ask and your own specialist agrees, and we never describe them as cures.