Gene Therapy

New Cholesterol Therapy Cuts Bad Cholesterol by Nearly 50%, Potential Statin Alternative

Researchers from the University of Barcelona and University of Oregon develop a novel DNA-based cholesterol therapy that reduced LDL cholesterol by 47% in animal models, without statin-related side effects.

FDA Approves World's First Gene Therapy for Genetic Hearing Loss in Record 61 Days

The FDA has approved Regeneron’s Otarmeni, the world’s first dual AAV vector-based gene therapy for severe-to-profound sensorineural hearing loss caused by OTOF gene mutations, with 80% of patients showing hearing improvement in clinical trials.