Early Win for Gene Therapy in Rare Form of SCID

December 22, 2022

(MedPage Today) – A gene therapy for a rare type of severe combined immunodeficiency (SCID) was safe and sufficiently restored T-cell immunity in an early trial, results from a phase I/II study showed. At a single center, all 10 infants with Artemis-deficient SCID (ART-SCID) produced functioning T cells at a median of 12 weeks after the procedure, reported Morton Cowan, MD, of UCSF Benioff Children’s Hospital in San Francisco, and colleagues. Five of the six patients who were followed for at least 24 months had complete T-cell immune reconstitution at a median of 12 months, they noted in the New England Journal of Medicineopens in a new tab or window. (Read More)