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Gene Therapy

Huntington's Disease Clinical Trials CRISPR Technology Rare Diseases Hearing Improvement Spinal Muscular Atrophy Duchenne Muscular Dystrophy Blood Disorders Respiratory Medicine 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 Cardiomyopathy Gene Regulation Personalized Medicine RNA Interference Hearing Impairment MET Inhibitors Fabry Disease Genetic Deafness Vision Loss Treatment Pediatric Gene Therapy Retinal Diseases Sickle Cell Disease Usher Syndrome Type 1B Adeno-Associated Virus (AAV) Immunotherapy Friedreich's Ataxia Neurological Disorders Heart Failure Treatments Hearing Loss ADA-SCID Cardiovascular Disease Skin Diseases Cancer Treatment Cholesterol Management Lebersche Kongenitale Amaurose Pediatric Immune Disorders Cardiovascular Diseases Cellular Reprogramming Ethics in Medicine

uniQure Shares Plummet After AMT-130 Misses Pre-Specified Statistical Goal

Citing possible control-group attrition bias, uniQure is relying on a stronger 36-month dataset in its FDA filing.

FDA Places Clinical Hold on REGENXBIO's RGX‑121 After Spine MRI Findings

Journal and University Open Investigations After Child’s Death Linked to Brain-Targeted Gene Editing

Researcher Investigated for Concealing Child’s Death After Experimental Gene‑Editing Trial

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

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