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Gene Therapy

Huntington's Disease Hearing Improvement CRISPR Technology Cerebral Adrenoleukodystrophy RNA Drugs Hematopoietic Stem Cells Heart Failure RNA-based Medicines Respiratory Gene Therapy In Vivo Gene Therapy Inherited Retinal Degenerations Metachromatic Leukodystrophy Lentiviral Vectors Severe Leukocyte Adhesion Deficiency Gene Regulation Clinical Trials Personalized Medicine RNA Interference Blood Disorders MET Inhibitors Fabry Disease Genetic Deafness Vision Loss Treatment Pediatric Gene Therapy Duchenne Muscular Dystrophy Retinal Diseases Sickle Cell Disease Usher Syndrome Type 1B Adeno-Associated Virus (AAV) Immunotherapy Friedreich's Ataxia Neurological Disorders Hearing Loss ADA-SCID Spinal Muscular Atrophy Cardiovascular Disease Hearing Impairment Rare Diseases Respiratory Medicine

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

The decision follows phase 3 data showing statistically meaningful one-year motor improvement in children and teenagers.

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

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

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