Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia.

Franco LocatelliPeter LangDonna WallRoland MeiselSelim CorbaciogluAmanda M LiJosu de la FuenteAmi J ShahBen CarpenterJanet L KwiatkowskiMarkus MaparaRobert I LiemMaria Domenica CappelliniMattia AlgeriAntonis KattamisSujit ShethStephan GruppRupert HandgretingerPuja KohliDaoyuan ShiLeorah RossYael BobruffChristopher SimardLanju ZhangPhuong Khanh MorrowWilliam E HobbsHaydar Frangoulnull null

Published in: The New England journal of medicine (2024)

Treatment with exa-cel, preceded by myeloablation, resulted in transfusion independence in 91% of patients with transfusion-dependent β-thalassemia. (Supported by Vertex Pharmaceuticals and CRISPR Therapeutics; CLIMB THAL-111 ClinicalTrials.gov number, NCT03655678.).

Keyphrases

sickle cell disease
cardiac surgery
crispr cas
acute kidney injury
small molecule
genome wide
genome editing
gene expression
smoking cessation