Decibel Therapeutics: Pivoting to Gene Therapy Treatment for Hearing Loss

Decibel Therapeutics, a Boston-based biotechnology company, announced last week that they have raised $82 million to pursue gene therapies for hearing loss. This is exciting for a few reasons:

  1. Genetics is one of the leading causes of hearing loss. According to the CDC, 50-60 percent of children born with hearing loss have it due to genetics. And genetic causes of hearing loss tend to be more severe, meaning they impact a person even more than milder forms of hearing loss.
  2. Other treatments in the works, such as Frequency’s FX-322, target other causes of hearing loss, but not genetics. So Decibel’s work has the potential to help an entirely different population of people.

Interestingly, Decibel was previously working on treatments that would help prevent hearing loss from happening in the first place. But according to Endpoint News, due to recent advances in genomic and regenerative technology, the company decided to shift to gene therapy for people who already live with hearing loss.

Currently, Decibel’s gene therapy program aims to test children with genetic deafness due to the otoferlin gene (OTOF). OTOF is one of the most frequent causes of sensorineural hearing loss. According to a May 2019 study published in PLos One, more than 160 mutations in OTOF have been identified so far. It tends to cause severe to profound hearing loss in children from birth. The idea is that the company’s technology will help to focus on the deficient hair cells in the ear and restore them.

Decibel’s long-term goal is to create cures for other genetic causes of hearing loss once the OTOF program is successful. That is currently in preclinical stages, and the company expects to start clinical testing in 2022, according to their press release.