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Gene Therapy

Spinal Muscular Atrophy Duchenne Muscular Dystrophy Sickle Cell Disease CRISPR Technology AADC Deficiency Gene Editing Muscular Dystrophy Wiskott-Aldrich Syndrome Retinal Diseases CAR-T Therapy CAR T-cell Therapy Transfusion-Dependent Beta Thalassemia Sarepta Therapeutics Huntington's Disease Beta Thalassemia Manufacturing Processes Inherited Blood Disorders Clinical Trials ADA-SCID Inherited Eye Disorders Hunter Syndrome Inherited Disorders Synovial Sarcoma Crispr Technology Cerebral Adrenoleukodystrophy (CALD) Childhood Blindness Hemophilia B Leukocyte Adhesion Deficiency (LAD-1) Leukocyte Adhesion Deficiency 1 (LAD-1) Metabolic Disorders Personalized Medicine Bespoke Treatments CRISPR Viral Inhibition Strategies Cognitive Function

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

The decision follows phase 3 data showing statistically meaningful one-year motor improvement in children and teenagers.

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

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

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

Regulators Halt Elevidys Gene Therapy Worldwide After Safety Failures

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