Sensorion advances hearing loss gene therapy programs

Sensorion continues to advance multiple gene therapy programs for hearing loss, including GJB2-GT (SENS-601), which targets mutations in the GJB2 gene, a leading cause of genetic congenital deafness, and is positioning the program as its lead gene therapy candidate with first‑in‑human studies planned in 2026. Sensorion has also selected SENS-601 (GJB2‑GT) as its lead program, submitting Clinical Trial Applications in Canada and France for pediatric GJB2‑mediated hearing loss, and discontinuing further development of its SENS‑501 (OTOF‑GT) hearing loss program to focus its clinical development portfolio.

Novartis has generated biomarker data in muscular dystrophy, particularly in Duchenne muscular dystrophy (DMD), using biomarkers such as muscle‑derived microRNAs and serum creatine kinase levels to track disease progression and therapeutic response. Novartis is applying these biomarkers in early‑phase clinical trials to assess the efficacy of investigational therapies aimed at muscle regeneration and disease modification in DMD, with recent data highlighting their utility in monitoring treatment‑induced changes in muscle health and function.