On a video call in early September, Sarah Tabrizi first saw the data that she and other researchers studying Huntington’s ...

A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature ...

Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced the first commercial patient treatment with FDA-approved ZEVASKYN ...

Almost two thirds of patients with a rare form of blood cancer who took part in a clinical trial of a revolutionary new ...

Serious side effects, including neurotoxicity and intestinal inflammation, that appear weeks or months after patients receive ...

Almost two thirds of patients with T-cell acute lymphoblastic leukaemia involved in a clinical trial of the treatment are now ...

A groundbreaking new treatment using genome-edited immune cells, developed by scientists at UCL (University College London) and Great Ormond Street ...

Patients with a previously untreatable form of blood cancer are now living disease-free after a world-first gene therapy. Alyssa Tapley, 16, became the first patient with an aggressive form of ...

March Bio is continuing enrollment in the Phase 2 trial, with the Independent Data Monitoring Committee’s approval to advance to Simon Stage 2 of the Phase 2 trial. The Company anticipates presenting ...

A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell leukemia.

Serious side effects, including neurotoxicity and intestinal inflammation, that appear weeks or months after patients receive CAR T cell therapy for multiple myeloma share a common immune root cause, ...