Science ❯ Genetics

Gene Therapy

Clinical Trials CRISPR Technology Huntington's Disease Otoferlin Gene CCR5 Mutation Clinical Applications Clinical Research Hearing Loss Protein Engineering Gene Editing Neuronal Plasticity Sickle Cell Disease Applications in Medicine Stem Cell Research LAMP2 Gene Follistatin Deafness Personalized Medicine Gene Silencing Neurodegenerative Diseases Disease Mechanisms Skin Conditions Adeno-Associated Virus AADC Deficiency Hearing Impairment FOXP3 Gene CAR-T Therapy Gene Editing Techniques CRISPR Cochlear Implants AAV Technology Regulatory Pathways mRNA Technology Enzyme Deficiency SCN1A Gene Gene Regulation Genetic Engineering GeneTAC Technology KRAS Mutations CRISPR/Cas9 Cell Therapy

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

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

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

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

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