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Gene Therapy

Synovial Sarcoma Transfusion-Dependent Beta Thalassemia Sickle Cell Disease Cerebral Adrenoleukodystrophy (CALD) CAR T-cell Therapy AADC Deficiency Childhood Blindness

First Death Reported in Patient Using Sarepta's Duchenne Gene Therapy

Sarepta Therapeutics investigates acute liver failure case, leading to a 23% stock drop and plans to update prescribing information.

Gene Therapy Restores Sight in Children Born Blind Due to Rare Eye Disorder

NHS Approves First Gene-Editing Therapy for Severe Sickle Cell Disease

Groundbreaking Lupus Treatment Trial Offers Hope for Drug-Free Remission

NHS Approves Groundbreaking Gene-Editing Therapy for Beta Thalassemia

FDA Approves Adaptimmune's Groundbreaking T-Cell Therapy for Rare Cancer