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Gene Therapy

Duchenne Muscular Dystrophy Genetic Diseases Clinical Trials CRISPR Technology Crispr Technology AAV Capsids Genetic Interventions Gene Editing Retinal Dystrophy Patient Safety

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.

Lilly to Buy Adverum in Gene‑Therapy Deal Worth Up to $12.47 a Share

FDA Names First 9 Drugs for ‘National Priority’ Fast-Track Reviews, Targeting 1–2-Month Decisions

AI-Guided Pythia Steers CRISPR Edits Toward Precise DNA Integration

Sarepta Defies FDA, Keeps Duchenne Gene Therapy on the Market

Early Temferon Trial Shows Engineered Macrophages Reprogram Glioblastoma Niche to Rescue CAR-T Cells

Scientists Uncover Genetic ‘Dimmer Switch’ That Tunes Embryonic Gene Expression

FDA Investigates Fatal Liver Failures Linked to Sarepta’s Elevidys Therapy

CRISPR Technique Reawakens Dormant Genes to Treat Blood Disorders

Sarepta Halts Elevidys Shipments for Non-Ambulatory Patients After Second Fatal Liver Failure

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

First Personalized CRISPR Therapy Corrects Rare Genetic Disorder in Infant

Researchers Release AI-Driven Tool for Custom CRISPR-Cas9 Enzyme Design

Gene Therapy Restores Sight in Children Born Blind Due to Rare Eye Disorder

Pfizer Ends Hemophilia Gene Therapy Partnership With Sangamo, Sending Stock Plummeting

NHS Approves Groundbreaking Gene-Editing Therapy for Beta Thalassemia