Regeneron Pharmaceuticals has expanded its gene therapy programs by acquiring Decibel Therapeutics, a biotech company focused on developing treatments for hearing loss. The $1.1 billion deal provides Regeneron with three promising gene therapy candidates that use adeno-associated virus (AAV) vectors to restore hearing.
The most advanced asset is DB-OTO, an AAV-based gene therapy designed to provide long-term hearing to individuals with profound congenital hearing loss caused by otoferlin gene mutations. DB-OTO is currently being evaluated in a Phase 1/2 clinical trial known as CHORD. The gene therapy aims to deliver a functional copy of the otoferlin gene to inner ear hair cells, potentially enabling hearing restoration.
The acquisition also includes two earlier-stage gene therapies, AAV.103 and AAV.104, targeting other genetic forms of hearing loss – GJB2 and STRC respectively. Both utilize a similar AAV gene delivery approach to DB-OTO.
According to Regeneron, the addition of Decibel’s pipeline and capabilities will strengthen its genetic medicines portfolio. Gene therapy has become a major focus for Regeneron beyond its foundational expertise in antibodies. The company is exploring gene silencing, gene editing and gene therapy technologies across a range of therapeutic areas.
Hearing loss represents a new area for Regeneron, building on an existing collaboration with Decibel. Integration of Decibel’s team and experience in inner ear biology and AAV gene therapy for hearing disorders will be invaluable as Regeneron advances the acquired programs.
Gene therapy aims to address disease at its genetic root cause by introducing functional genes into cells. The goal is to durably restore protein expression and correct the downstream impacts of gene mutations. Gene therapy has shown promise for treating rare monogenic disorders like certain forms of inherited hearing loss.
Both Regeneron and Decibel have utilized AAV vectors to deliver gene therapy payloads. AAV is considered one of the most effective vehicles for gene delivery and has an established safety profile. The viruses can be engineered to target specific cell types following injection into the body.
For DB-OTO, the AAV vector carries a functional copy of the otoferlin gene. Inner ear hair cells are the targets for gene transduction. Otoferlin protein is critical for hearing signal transduction, but mutations in the encoding gene cause profound congenital deafness. Gene therapy aims to restore otoferlin expression and regain hearing function.
Regeneron’s push into gene therapy aligns with its mission of tackling serious diseases with novel technologies. Gene-based treatments have potential for one-time curative therapies. The acquisition of Decibel’s pipeline further diversifies Regeneron’s genetic medicine capabilities as it aims to help patients worldwide.