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Gene Therapy

Spinal Muscular Atrophy Sickle Cell Disease Duchenne Muscular Dystrophy CRISPR Technology AADC Deficiency Hemophilia B Transfusion-Dependent Beta Thalassemia Gene Editing CAR T-cell Therapy Muscular Dystrophy

FDA Approves First Gene Therapy for Inherited Deafness

Regeneron will supply the one-time treatment free in the U.S., easing a key barrier to access.

FDA Grants Accelerated Approval to Kresladi, First Gene Therapy for Severe Pediatric LAD-1

Brazil Treats First Infant in In‑Country Gene Therapy Trial for Severe Spinal Muscular Atrophy

Oldham Teen Returns to Thank Nurse After Pioneering CRISPR Thalassaemia Treatment

Abu Dhabi Hospital Delivers World’s First Dose of Itvisma Gene Therapy for SMA

FDA Approves One-Time Intrathecal Gene Therapy Itvisma for Older SMA Patients

FDA Approves Novartis’ Itvisma for SMA Patients Ages 2 and Up

World-First Gene Therapy for Hunter Syndrome Shows Early Gains in First Child

EMA Panel Backs Waskyra Gene Therapy for Wiskott‑Aldrich Syndrome

Lilly Licenses MeiraGTx Eye Gene Therapy in Deal Worth Up to $475 Million

First UK Patient Receives CAR T Therapy for Multiple Sclerosis in UCLH Trial

NEJM Study Finds 95% Long-Term Success for ADA-SCID Gene Therapy

Galicia Approves €100 Million CAR-T Purchase for 250 Multiple Myeloma Patients

Dual-Vector Gene Therapy Restores Sight in Usher Syndrome Patient; Trial Advances to Higher Doses

First Patient With Usher Syndrome Regains Sight Through Double-Vector Gene Therapy

Regulators Halt Elevidys Gene Therapy Worldwide After Safety Failures

Vigo Hospital Administers Spain’s First Public Gene Therapy for Hemophilia B

Sarepta Halts Elevidys Shipments for Non-Ambulatory Patients After Second Fatal Liver Failure

Gene Therapy Disables HSV-1’s Uracil-DNA Glycosylase and Protects against Encephalitis

First Personalized CRISPR Therapy Successfully Treats Infant’s Rare Genetic Disorder