Health ❯ Medical Treatments

Gene Therapy

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

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

The readout lands under heightened FDA scrutiny following two liver‑failure deaths in non‑ambulatory patients.

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

CRISPR-Based Gene Therapy Shows Promise in Curing Hereditary Angioedema