Treatments
Approved Gene-Editing Therapies
Every gene-editing medicine that a national regulator has authorised, worldwide.
How this list is built
This list is deliberately short, and its shortness is the point. Enormous scientific progress has so far produced a very small number of approved editing medicines. Everything else on this site that sounds promising is research.
| # | Entry | Category | Status |
|---|---|---|---|
| 1 | Casgevy (exagamglogene autotemcel) The first CRISPR-based medicine approved anywhere in the world: a one-time therapy made… | Sickle cell disease; beta thalassemia | Approved |
| 2 | Lyfgenia (lovotibeglogene autotemcel) Approved on the same day as Casgevy for sickle cell disease, but a gene-addition therapy… | Sickle cell disease | Approved |
| 3 | Zynteglo (betibeglogene autotemcel) An approved gene-addition therapy for transfusion-dependent beta thalassemia: the… | Beta thalassemia | Approved |
What this list does not tell youApproval is country-specific and does not mean a treatment is available, affordable or suitable for any individual. This list includes gene-addition therapies where they are approved for the same conditions, clearly labelled, because the distinction is the point.