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Gene Therapy

CRISPR-Cas9 Patient Outcomes Duchenne Muscular Dystrophy CRISPR Technology In Vivo CAR-T Therapy Base Editing FDA Approval Dystrophic Epidermolysis Bullosa Hearing Restoration AMT-130 CAR T Cell Applications Disease Treatment Hereditary Angioedema Facioscapulohumeral Muscular Dystrophy Glaucoma

FDA Panel Rejects Deramiocel Cardiac Claim After Lancet Publishes HOPE‑3 Results

A 9–3 advisory vote and critical FDA reviews increase doubt about final approval ahead of the August 22 PDUFA date.

First Patient Dosed in Human Trial for Cell 'Rejuvenation' Therapy

Intellia’s Gene-Editing Drug Cuts HAE Attacks 87% in Phase 3 as FDA Filing Begins

CAR-T Therapy Puts Three Autoimmune Diseases Into Sustained Remission in One Patient

FDA Meeting Minutes Undercut uniQure’s Plan to File Huntington’s Gene Therapy for Approval

Capricor’s HOPE-3 Trial Reports Cardiac and Muscle Gains in Duchenne Muscular Dystrophy

Single-Infusion CRISPR Therapy Lowers LDL and Triglycerides in First Human Trial

Intellia Pauses Phase 3 Trials of CRISPR Therapy After Severe Liver Event

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

USC Researchers Advance Inner-Ear OCT for Noninvasive Hearing Diagnostics

FDA Approves Zevaskyn Gene Therapy for Healing Large Epidermolysis Bullosa Wounds