Science ❯ Genetics

Gene Therapy

Clinical Trials CRISPR Technology Huntington's Disease Clinical Applications Hearing Loss Clinical Research Otoferlin Gene CCR5 Mutation AADC Deficiency Gene Regulation

Design Therapeutics Reports Early DT-216P2 Data in Friedreich’s Ataxia

The company signaled a potential registrational path with an update planned for the fourth quarter.

FDA Approves First Gene Therapy for Inherited Hearing Loss

Oslo Patient Is 10th Documented HIV Remission After CCR5 Stem-Cell Transplant

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

Parents Press UK to Fund Access to US Sanfilippo Trial for Their Two-Year-Old

Early Trials of Gene Regulation Drug Cut Dravet Seizures by Up to 91%, NEJM Reports

Lilly Strikes Up to $1.12 Billion Pact With Seamless Therapeutics for Hearing‑Loss Gene Editing

Aurora Therapeutics Launches to Scale Bespoke Gene Editing for PKU

FDA Meeting Minutes Undercut uniQure’s Plan to File Huntington’s Gene Therapy for Approval

Regeneron, Tessera Strike $150 Million Gene-Editing Pact for Alpha‑1 Antitrypsin Deficiency

MHRA Unveils Overhaul to Speed Rare-Disease Drug Approvals

Lowering a Brain Protein Reverses Age-Related Memory Deficits in Mice

Umbilical-Cord-Derived Cell Therapy Reduces Disease Activity in Children With Rare Skin Disorder

Single Gene Therapy Injection Reverses Deafness Across Age Groups

FDA Approves Zevaskyn Gene Therapy for Healing Large Epidermolysis Bullosa Wounds

FDA Halts Rocket’s RP-A501 Gene Therapy for Danon Disease After Patient’s Death

Breakthrough Gene Therapies Offer New Hope for Sickle Cell Disease

Zebrafish Reveal New Pathways for Spinal Cord Injury Recovery

Gene Therapy Restores Hearing in Children with Hereditary Deafness