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Gene Therapy

Spinal Muscular Atrophy Sickle Cell Disease Duchenne Muscular Dystrophy CAR T-cell Therapy CRISPR Technology Retinal Diseases Wiskott-Aldrich Syndrome Huntington's Disease Hearing Restoration CAR-T Therapy Crispr Technology Transfusion-Dependent Beta Thalassemia Muscular Dystrophy Hearing Loss Pediatric Gene Therapy Gene Editing AADC Deficiency Hemophilia B Genetic Hearing Loss CRISPR Inherited Hearing Loss Manufacturing Processes Childhood Blindness Leukocyte Adhesion Deficiency (LAD-1) Cholesterol Management Personalized Medicine Metabolic Disorders Inherited Deafness Inherited Disorders Dystrophic Epidermolysis Bullosa ADA-SCID Hearing Loss Treatment Synovial Sarcoma Bespoke Treatments Hunter Syndrome Leukocyte Adhesion Deficiency 1 (LAD-1) Cerebral Adrenoleukodystrophy (CALD) SMA Treatments Inherited Eye Disorders Clinical Trials OTOF-related Hearing Loss Sarepta Therapeutics Cognitive Function Viral Inhibition Strategies Inherited Eye Conditions Beta Thalassemia Luxturna Severe Leukocyte Adhesion Deficiency Type 1 Inherited Blood Disorders

FDA Expands Casgevy Gene Therapy to Children Ages Two and Up

Earlier treatment could reduce lasting organ damage, requiring regulators and clinicians to monitor long-term safety, durability and access.

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CAR‑T Cells Targeting GPNMB Eradicate Glioblastoma in Preclinical Models

FDA Agrees Three‑Year Phase I/II Data Can Support uniQure’s AMT‑130 Accelerated BLA

Single Infusion Gene Editing Lowers LDL by Up to 62% in Early Trial

FDA Approves First Gene Therapy for Inherited Hearing Loss

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

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

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