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Gene Therapy

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7 ARTICLES

2w ago

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

Regulators have ordered additional imaging and longer follow‑up to determine whether the asymptomatic spinal nodules found in trial participants are linked to the AAV gene therapy which has paused near‑term approval plans.

7 ARTICLES

2mo ago

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

6 ARTICLES

10mo ago

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

8 ARTICLES

10mo ago

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

5 ARTICLES

last yr.

AI-Guided Pythia Steers CRISPR Edits Toward Precise DNA Integration

7 ARTICLES

last yr.

Sarepta Defies FDA, Keeps Duchenne Gene Therapy on the Market

3 ARTICLES

last yr.

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

4 ARTICLES

last yr.

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

6 ARTICLES

last yr.

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

Gene Therapy

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

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

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