Regeneron Rises on Gene Therapy Hearing Restoration Success
DB-OTO, an investigational gene therapy, showed dramatic and sustained hearing improvements in children with a rare form of genetic hearing loss.
Regeneron Pharmaceuticals (NASDAQ: REGN) saw its shares climb after announcing breakthrough results for its investigational gene therapy, DB-OTO, which demonstrated significant hearing improvement in children with profound genetic deafness. The positive data, stemming from the Phase 1/2 CHORD trial, was published in the prestigious , lending significant credibility to the findings and boosting investor confidence.
The trial focused on children with hearing loss caused by mutations of the otoferlin (OTOF) gene. The latest results showed that 11 of the 12 participants experienced clinically meaningful hearing gains. Some participants' hearing improved to normal or near-normal levels, allowing them to perceive conversational speech and sound for the first time. Following the news, , reflecting optimism about DB-OTO's potential as a first-of-its-kind treatment.
This promising therapy was brought into Regeneron's pipeline through its 2023 acquisition of Decibel Therapeutics, a strategic move that appears to be paying dividends. DB-OTO is designed to deliver a working copy of the OTOF gene via a modified adeno-associated virus (AAV) directly to the inner ear, aiming to restore the biological function required for hearing. According to the company's , the treatment was generally well-tolerated with no serious adverse events related to the gene therapy itself.
The potential market for DB-OTO, while targeting a rare condition, represents a significant unmet medical need. Current standards of care, such as cochlear implants, assist with hearing but do not restore the underlying biological function. A one-time gene therapy that provides durable hearing restoration could revolutionize the treatment paradigm. Jonathon Witton, a Regeneron executive, highlighted the company's goal to shift the standard of care from symptomatic management to a targeted therapeutic approach.
Looking ahead, Regeneron is navigating the complex manufacturing and regulatory pathways for this advanced therapy. The treatment has already received multiple key designations from the FDA, including Orphan Drug, Rare Pediatric Disease, and Fast Track status, which could expedite its journey to market. As the company prepares for potential commercialization, it is focusing on associated with gene therapies to ensure scalable and high-quality production. Investors and the medical community will be closely watching for further data from the CHORD trial as it progresses.