Decibel Therapeutics Shares Encouraging Preclinical Data From Hearing Loss Gene Therapy Programs

Decibel Therapeutics Inc 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 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.