The programme is aimed at treating a certain congenital deafness form caused by otoferlin (OTOF) gene mutations. SENS-501’s intra-cochlear administration was tolerated well, allowing the second ...
The Audiogene trial is designed to evaluate the safety, tolerability and efficacy of intra-cochlear injection of SENS-501 for the treatment of OTOF gene-mediated hearing loss in paediatric ...
The preliminary results from the Audiogene study, conducted in children aged six to 31 months without cochlear hearing implants, also showed that the SENS-501 (formerly OTOF-GT) gene therapy was ...
In addition to OTOF, Regeneron is committed to investigating several other targets for genetic forms of hearing loss, including GJB2. Regeneron (NASDAQ: REGN) is a leading biotechnology company ...
The main focus of the deal is Decibel's DB-OTO, an adeno-associated virus (AAV) gene therapy designed to correct mutations in the otoferlin or OTOF gene, which codes for a large protein expressed ...
The Audiogene trial is designed to evaluate the safety, tolerability and efficacy of intra-cochlear injection of SENS-501 for the treatment of OTOF gene-mediated hearing loss in paediatric patients ...
However, otoferlin-related hearing loss is ultra-rare. This specific condition is caused by variants in the OTOF gene, which leads to a lack of a functional otoferlin protein that is critical for ...