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Gene Therapy

Huntington's Disease CRISPR Technology Hearing Improvement Rare Diseases Spinal Muscular Atrophy Duchenne Muscular Dystrophy Blood Disorders Metachromatic Leukodystrophy Gene Regulation Pediatric Gene Therapy

CRISPR Enzyme Cas12a2 Selectively Destroys Cells Based on RNA in Lab Tests

Translation hinges on animal studies followed by practical delivery to specific tissues.

Roche Starts Placebo-Controlled Phase 3 Trial of Elevidys to Pursue EU Approval

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

FDA Proposes ‘Plausible Mechanism’ Pathway for Individualized Rare-Disease Therapies

FDA Approves One-Time Intrathecal Gene Therapy Itvisma for Older SMA Patients

Exa‑cel Posts Complete Primary-End Point Responses in Early Pediatric Trials, With One Fatal Busulfan Event

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

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

One-Time CRISPR Edit Halves LDL and Triglycerides in Early Human Trial

NEJM Study Finds 95% Long-Term Success for ADA-SCID Gene Therapy

Capsida Halts STXBP1 Gene Therapy Trial After First Pediatric Participant Dies

Kidney Organoids Reveal NFκB‑Linked AAV2 Toxicity, With Bardoxolone Methyl Mitigating Damage

Dual-Vector Gene Therapy Restores Sight in Usher Syndrome Patient; Trial Advances to Higher Doses

Experimental Fabry Gene Therapy Achieves Near-Break-Even Costs and Advances to Larger Study

AAV-OTOF Gene Therapy Restores Hearing in Teens and Adults

CRISPR Technique Reawakens Dormant Genes to Treat Blood Disorders

Study uncovers early-life window for in vivo gene therapy of blood stem cells

Penn Researchers Develop Gene Therapy Tools for Advanced Retinal Degenerations

Gene Therapy Restores Immune Function in Children with Rare Disorder