Science ❯ Genetics

Gene Therapy

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

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