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Gene Therapy

Huntington's Disease Hearing Improvement CRISPR Technology Rare Diseases Spinal Muscular Atrophy 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 Hearing Impairment MET Inhibitors Blood Disorders 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 Cardiovascular Disease Skin Diseases Fabry Disease Respiratory Medicine Cerebral Adrenoleukodystrophy RNA Drugs Hematopoietic Stem Cells

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

Sponsors would gain a route to approval based on plausible biology, with obligations to verify outcomes after launch.

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

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