Patients with relapsed or refractory hematologic cancers have a poor prognosis. Chimeric antigen receptor (CAR) T-cell therapy as a bridge to allogeneic hematopoietic stem-cell transplantation ...

Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis by means of ex vivo clustered regularly interspaced short palindromic repeats ...

This podcast episode examines CAR T-cell therapy’s early successes, broader promise, and emerging risks, as the FDA considers ...

Infants were eligible if they were born between 32 weeks 0 days’ and 35 weeks 6 days’ gestation, had been admitted to a neonatal unit, and had intravenous access that had been established for ...

In the year after the elimination of a waiver requirement to prescribe buprenorphine, the number of prescribers increased above the anticipated value, but the number of persons who received the ...

We conducted an open-label, single-group, phase 3 study of exa-cel in patients 12 to 35 years of age with transfusion-dependent β-thalassemia and a β 0 /β 0, β 0 /β 0-like, or non–β 0 /β ...

The autologous cellular therapy exagamglogene autotemcel is generated by editing an erythroid-specific enhancer of BCL11A. Could another site be edited unintentionally? This study gauged the ...

Disclosure forms provided by the authors are available with the full text of this editorial at NEJM.org. From the Global Health Division, Bill and Melinda Gates Foundation (J.M.M.), and the Fred ...

For permission requests, please contact NEJM Reprints at [email protected] We and others identified retinoblastoma features that suggested that the tumors originate in long- and medium-wavelength ...

A 57-year-old woman presented with a 3-day history of shortness of breath and dizziness. Laboratory studies showed severe anemia with hemolysis, along with cellular abnormalities in peripheral blood.

Recent approvals of exa-cel for treatment of sickle cell disease and transfusion-dependent β-thalassemia mark the dawn of the era of gene editing in medicine. But ensuring access will be challenging.