Approved treatment
At least one medicine using this approach has been authorised by a national regulator for this use.
Explicação simples
Zynteglo adds a working copy of the beta-globin gene into a patient's own blood stem cells using a modified virus, so their body can make normal haemoglobin again. Most people treated in its trials no longer needed blood transfusions. It treats the same condition as Casgevy by a different route.
Aprofundar
Betibeglogene autotemcel delivers a functional βA-T87Q-globin gene to autologous CD34+ cells by lentiviral transduction, restoring haemoglobin production in transfusion-dependent beta thalassemia. FDA approved August 2022. Requires the same myeloablative conditioning as editing-based products.
Sources
- U.S. Food and Drug Administration
Zynteglo ↗