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Gene Therapy

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

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.

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

Toddler With Restored Hearing Becomes Oval Office Focus as Trump Hails Gene Therapy

FDA Approves First Gene Therapy for Inherited Hearing Loss

NHS Gene Therapy Restores Sight for 6-Year-Old

UK Brings Forward Newborn SMA Screening Pilot to October 2026

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