Decibel Therapeutics Inc (NASDAQ:DBTX) announced the presentation of preclinical data on DB-OTO, its lead gene therapy product candidate.
- DB-OTO is a dual-vector adeno-associated candidate designed to restore hearing to individuals with profound, congenital hearing loss caused by mutations of the otoferlin gene.
- DB-OTO uses a cell-selective promoter to control otoferlin transgene expression in hair cells to enable the ear to transmit sound to the brain and provide a hearing.
- Administration of DB-OTO resulted in the restoration of functional otoferlin protein in mice and dose scaling and distribution in non-human primates (NHPs).
- New data further characterized the expression of otoferlin over time following DB-OTO administration in mice and NHPs, demonstrating that otoferlin expression increased over the first several weeks before plateauing.
- The Company also presented new data on its preclinical AAV.103 program to treat GJB2-related hearing loss and its preclinical AAV.104 program to treat stereocilin-related hearing loss through selective expression of stereocilin in outer hair cells.
- Decibel is developing DB-OTO, AAV.103, and AAV.104 in collaboration with Regeneron Pharmaceuticals Inc (NASDAQ:REGN), and Decibel retains global commercial rights in the three programs.
- Related: Regeneron Extends Hearing Loss Gene Therapy pact With Decibel.
- Price Action: DBTX shares traded 1.74% lower at $4.23 on the last check Thursday.
