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Gene Therapy

CRISPR-Cas9 Patient Outcomes FDA Approval Dystrophic Epidermolysis Bullosa CRISPR Technology Hearing Restoration AMT-130 Duchenne Muscular Dystrophy Base Editing

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

uniQure will seek an early 2026 follow-up meeting after staff signaled Phase I/II results are unlikely to provide primary evidence for a BLA.

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