- The U.S. Food and Drug Administration (FDA) granted clearance to Decibel Therapeutics' ( NASDAQ: DBTX ) investigational new drug (IND) application for starting a phase 1/2 trial of gene therapy DB-OTO to treat hearing loss in pediatric patients.
- DB-OTO is aimed to provide durable hearing in individuals born with profound congenital hearing loss due to deficiency of a protein called otoferlin, according to the company.
- Decibel said DB-OTO is being developed in collaboration with Regeneron Pharmaceuticals ( NASDAQ: REGN ) and is an adeno-associated virus (AAV)-based gene therapy product candidate.
- The company added that a one-time administration of DB-OTO had shown a production of otoferlin protein and durable auditory brainstem responses to sound in a congenitally deaf rodent disease model.
- Decibel expects the first two participants in the U.S. portion of the phase 1/2 trial will be as young as seven years of age and afterwards will include children as young as two years of age, and infants below two years of age.
- The company noted that DB-OTO is the second product candidate after DB-020 in its pipeline to advance into clinical testing.
- DBTX +18.24% to $3.50 premarket Oct. 17
For further details see:
Decibel stock surges 18% as FDA clears hearing loss gene therapy to enter trial