Decibel Therapeutics Presents Promising Preclinical Findings on DB-OTO, Gene Therapy Product Candidate Designed to Provide Hearing to Individuals with Otoferlin Mutations
- Category: DNA RNA and Cells
- Published on Wednesday, 24 February 2021 14:26
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Podium presentations and posters shared at the 44th Annual Association for Research in Otolaryngology Conference
BOSTON, MA, USA I February 24, 2021 I Decibel Therapeutics (Nasdaq: DBTX), a clinical-stage company developing treatments to restore and improve hearing and balance, presented preclinical data supporting the company’s gene therapy product candidate, DB-OTO, a potential transformative treatment for individuals with hearing loss due to mutation of the otoferlin gene. These and other data were presented by the company at the 44th Annual MidWinter Meeting of the Association for Research in Otolaryngology (ARO), which was held virtually February 20–24, 2021.
Decibel’s DB-OTO product candidate is designed to use dual AAV vectors and a cell-selective promoter to express functional otoferlin in hair cells of the inner ear, with the goal of enabling the ear to transmit sound to the brain and enable hearing. DB-OTO is intended to be delivered to patients using the surgical approach employed by otologists and pediatric otolaryngologists during a standard cochlear implantation procedure. Exploratory studies highlighted in the presentations utilizing the delivery procedure in non-human primates showed successful distribution and expression across the cochlear length. In these studies, DB-OTO and the surgical administration were generally well tolerated. The company presented data which suggest hair cell selective expression of otoferlin may enable greater efficacy and durability and minimize potential toxicity. In addition to these data, Decibel presented preclinical findings demonstrating that DB-OTO restored normal hearing sensitivity in animal models of otoferlin deficiency. Decibel is developing DB-OTO in collaboration with Regeneron Pharmaceuticals and expects to submit an IND or CTA in 2022.
“This is an exciting time for hearing and balance therapeutics, as we build preclinical evidence to support advancing gene therapies for the inner ear. We believe the data we shared at ARO, including potential benefits of cell-selective expression and utilization of an established surgical procedure, if shown in humans, would provide a competitive advantage for DB-OTO and support our approach to developing highly targeted gene therapies for hearing and balance,” said John Lee, Executive Vice President, Pharmaceutical Development and Interim Chief Scientific Officer.
A selection of Decibel’s presentations from ARO are available on the company’s website:
- A podium presentation by Adam Palermo, Ph.D., Decibel’s Vice President, Head of Genomics and Computational Biology, provided an overview of preclinical DB-OTO data from mice and non-human primates. The presentation can be found here.
- A podium presentation by Gabriela Pregernig, Ph.D., Associate Director of Computational Biology at Decibel, highlighted potential mechanisms of ubiquitous promoter-driven AAV toxicity in the ear. The presentation can be found here.
- Peter Weber, MBA, M.D., Decibel’s Senior Medical Advisor, delivered a podium presentation that described the surgical approach for DB-OTO. He presented data in which dual vector AAV and Decibel’s cell-selective promoter drove highly selective expression of GFP in hair cells of non-human primates. Details can be found here.
- A poster by Lars Becker, Ph.D., Associate Director of Discovery at Decibel, demonstrated that a variety of capsids are able to transduce inner hair cells of the inner ear in non-human primates and that the presence of neutralizing antibodies was not observed to impact transduction. The presentation can be found here.
About Decibel Therapeutics
Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, one of the largest areas of unmet need in medicine. Decibel has built a proprietary platform that integrates single-cell genomics and bioinformatic analyses, precision gene therapy technologies and expertise in inner ear biology. Decibel is leveraging its platform to advance gene therapies designed to selectively replace genes for the treatment of congenital, monogenic hearing loss and to regenerate inner ear hair cells for the treatment of acquired hearing and balance disorders. Decibel’s pipeline, including its lead gene therapy product candidate, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of a world in which the privileges of hearing and balance are available to all.
SOURCE: Decibel Therapeutics