Regeneron Adds Hearing Loss Gene Therapy Program Via Decibel Therapeutics Acquisition

Regeneron Pharmaceuticals successfully completed its acquisition of Decibel Therapeutics, strengthening the company’s gene therapy and auditory programs.

“After several years of successful collaboration, the integration of Decibel’s programs, capabilities and accomplished team will further bolster Regeneron’s genetic medicines portfolio and advance our mission of helping patients around the globe,” says Leonard S. Schleifer, MD, PhD, board co-chair, president and CEO of Regeneron. “We will continue to strategically deploy capital by selectively pursuing deals that secure access to novel technologies and approaches that are complementary to our technologies, portfolio and strengths in research.”

The acquisition of Decibel builds on prior collaboration between the companies and includes three ongoing gene therapy programs targeting different forms of congenital, monogenic hearing loss. The most advanced clinical-stage candidate is DB-OTO, which is currently being studied in the global Phase 1/2 CHORDTM clinical trial, and is an investigational cell-selective, adeno-associated virus (AAV) gene therapy designed to provide durable, physiological hearing to individuals with profound, congenital hearing loss caused by mutations of the otoferlin gene. Preclinical programs include AAV.103 for people with GJB2-related hearing loss and AAV.104 for people with stereocilin (STRC)-related hearing loss.

“As we advance Regeneron’s commitment to genetic medicine, we welcome our new colleagues and their important expertise in the biology of auditory disorders,” says George D. Yancopoulos, MD, PhD, board co-chair, president and chief scientific officer of Regeneron. “In addition to DB-OTO, we have worked with Decibel to build a rich pipeline of programs targeting congenital hearing loss caused by single-gene mutations, including the more common GJB2 and STRC mutations. We are actively expanding our expertise in cutting-edge genetic medicine approaches, which currently includes gene silencing, gene editing and gene therapy technologies with the potential to address many serious and hard-to-treat diseases.”