Health ❯ Medical Treatments

Gene Therapy

Clinical Trials CRISPR Technology Duchenne Muscular Dystrophy CAR T-cell Therapy Genetic Disorders Sickle Cell Disease Huntington's Disease Spinal Muscular Atrophy Experimental Treatments Zolgensma

NHS Gene Therapy Restores Sight for 6-Year-Old

Early treatment shows the strongest gains in a new GOSH/UCL study of 15 children.

Zolgensma Given to SMA Infant in Hyderabad After Minister Helps Close Funding Gap

UK Brings Forward SMA Newborn Screening to October 2026 With Partial Rollout

Sarepta Reports 3-Year EMBARK Data Showing Elevidys Slows Duchenne Progression

Vertex Projects Casgevy Sales Above $100 Million in 2025 as CRISPR Therapeutics Waits for Payoff

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

FDA Adds Boxed Warning to Elevidys, Narrows Use to Ambulatory Patients 4 and Older

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

Gene Therapy Shows 75% Slower Huntington’s Decline in Early Trial

Bayer Starts Pivotal Phase 3 Trial of BlueRock Cell Therapy for Parkinson’s

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

FDA Lifts Hold on Elevidys Shipments to Ambulatory Duchenne Patients

AAV-OTOF Gene Therapy Restores Hearing in Teens and Adults

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

Personalized CRISPR Therapy Sends Baby KJ Home from CHOP

Gene Therapy Restores Immune Function in Children with Rare Disorder

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

Gene Therapy Reverses Heart Failure in Groundbreaking Animal Study

Gene Therapy Restores Hearing in Children with Hereditary Deafness