RichterAvailable and upcoming CRISPR treatments
The first approved CRISPR-based therapy is Casgevy (exagamglogene autotemcel), for sickle cell disease and transfusion-dependent beta thalassemia. Status below is as of September 2026.
Treatment status by condition
| Condition | Status | How it is given | Who it may suit |
|---|---|---|---|
| Sickle cell disease with recurrent pain crises | Approved in several countries | One-time infusion of your own edited stem cells, in hospital | Patients who meet the age and severity criteria set by their regulator |
| Transfusion-dependent beta thalassemia | Approved in several countries | One-time infusion of your own edited stem cells, in hospital | Patients who need regular red blood cell transfusions |
| Other inherited conditions | In clinical trials | Varies by trial | Patients enrolled through a trial site |
What decides eligibility
- A confirmed diagnosis
- An age within the approved range for your country
- A history of severe symptoms, such as recurrent pain crises or regular transfusions
- Fitness for high-dose chemotherapy before the infusion
- Access to a qualified treatment centre
Watch: how the treatment works
Text summary of the video
The video shows how a patient's blood stem cells are collected, edited in a laboratory, and returned after chemotherapy. It then follows the recovery period in hospital and the long-term follow-up. Read this summary if you cannot play the video.