February 21, 2024 | PRESS RELEASE

Auditory Insight Explains Hearing Loss Gene Therapy for Children

The firm analyzes preliminary results from three investigational clinical trials across the globe

NEW HAVEN, Conn., Feb. 21, 2024 /PRNewswire/ Auditory Insight, a leading consultancy for the hearing healthcare industry, analyzes preliminary results for gene therapy clinical trials addressing deafness and hearing loss in its Q1 2024 Research Note.

Download the Research Note: Gene Therapy for Hearing Loss: Interpreting Preliminary Clinical Trial Results.

In three sets of investigational clinical trials around the globe, gene therapy has transformed the hearing of children with a form of deafness from mutations in a gene called otoferlin. The children’s hearing improved from profoundly deaf—such that they were unable to hear a shouted voice in their ear—to a moderate hearing loss, requiring only hearing aids as ongoing treatment, according to Auditory Insight’s analysis of the preliminary data.

“When I first saw these results, I felt more hope than I had in a long time for a pharmacological hearing loss solution,” enthused Nancy M. Williams, president of Auditory Insight. “This was both a professional and personal milestone for me.” She has advised gene therapy companies on surgeon, audiologist, and patient needs for gene therapy and genetic testing.

Williams’ work at Auditory Insight has tremendous personal meaning since she has a lifelong genetic, progressive hearing loss.

Auditory Insight’s latest research note provides an independent, rigorous analysis of published audiograms and other data from investigational clinical trials for gene therapy addressing otoferlin mutations. Three companies lead these trials: Eli Lilly’s Akouos unit, Regeneron (who acquired Decibel Therapeutics), and China’s Refreshgene Therapeutics.

In a surgical procedure, the surgeon injects working copies of the gene into the patient’s inner ear, where these transgenes create missing otoferlin proteins, a necessary ingredient in the complicated recipe for hearing.

The research note highlights three important conclusions:

  1. Target Population. The gene therapy partially restored hearing for children aged less than one year to 11 years.
  2. Efficacy. The trials have restored patients’ hearing in treated ears from profoundly deaf to moderate hearing loss.
  3. Time to Results. Patients’ hearing showed improvement at four weeks and continued to notch up gains at the six-month mark.

“Clinicians leading the trials have stated that they will continue to follow up with patients to determine the full length of time needed for the gene to express itself in improved hearing,” Williams explains.

For the estimated 200,000 people worldwide with otoferlin mutations, these clinical trial results offer optimism for those who seek restored hearing. The trials also matter because they advance the learnings of scientists developing gene therapy for other forms of genetic hearing loss.

Williams is available to discuss this research and implications for gene therapy companies targeting hearing loss.

About Auditory Insight

Auditory Insight partners with senior leaders of hearing health pharmaceutical and device companies to create successful market development strategies. The firm also advises growth equity and private equity funds on creating portfolio value in hearing healthcare.

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