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Gene Therapy

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

FDA Agrees Three‑Year Phase I/II Data Can Support uniQure’s AMT‑130 Accelerated BLA

It opens a route for accelerated approval with negotiations underway over the design of a required post‑approval confirmatory trial.

Life Biosciences Doses First Patient With Eye Reprogramming Gene Therapy

Single Infusion Gene Editing Lowers LDL by Up to 62% in Early Trial

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

FDA Grants Accelerated Approval to Kresladi, First Gene Therapy for Severe Pediatric LAD-1

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

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