BOSTON–(BUSINESS WIRE)–Decibel Therapeutics, a clinical-stage biotechnology company developing novel gene therapeutics for restoration of hearing loss and balance disorders, today announced that it will present new findings on its pipeline and platform at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), which will be held virtually from May 12–15.
Decibel’s platform utilizes leading single-cell genomics and bioinformatics capabilities to enable AAV-based gene therapy to restore functionality of cochlear and vestibular hair cells in multiple forms of hearing loss and balance disorders. The company has used its platform to identify a suite of genetic control elements enabling cell-specific expression in each of the key cell types of the inner ear. Decibel is further identifying key reprogramming factors to regenerate hair cells in the inner ear by gene therapy or small molecules.
Decibel will deliver three presentations:
- Adam Palermo, Ph.D., Senior Director and Head of Genomics and Computational Biology, will discuss the use of single-cell genomics and bioinformatics to develop cell-specific promoters for therapeutically important cell types in the ear.
- Jonathon Whitton, Au.D., Ph.D., Director of Clinical Development, will detail progress on DB-OTO, being developed in collaboration with Regeneron Pharmaceuticals, which is designed to restore hearing to congenitally deaf children with otoferlin deficiency by providing a normal copy of the otoferlin gene specifically to hair cells.
- Joseph Burns, Ph.D., Senior Director and Head of Biology, will describe DB-201, an AAV-based regenerative medicine in preclinical development for balance disorders that arise as a result of hair cell loss. DB-201 utilizes a supporting-cell-specific promoter to precisely regulate regeneration of vestibular hair cells.
“The research our team will share at ASGCT represents the progress we have made in developing our precision AAV platform to deliver genes that can restore hearing and balance function,” said John Lee, CSO of Decibel. “Our AAV platform, combined with our single-cell genomics and bioinformatics capabilities, is driving our progress to create transformative therapeutics for people who are living with severe hearing loss and balance disorders.”
The three presentations the Decibel team will share during the conference are the following:
Dual AAV Delivery of Otoferlin Durably Rescues Hearing in Congenitally Deaf Preclinical Models
Presenter: Jonathon Whitton, Au.D., Ph.D.
Session Title: Gene Therapy for the Special Senses
Date & Time: Tuesday, May 12, 11:15 a.m.
Tailored AAV-Based Transgene Expression in the Inner Ear with Cell Type-Specific Promoters
Presenter: Adam Palermo, Ph.D.
Session Title: Controlling AAV Gene Expression: Shifting Paradigms
Date & Time: Wednesday, May 13, 4:45 p.m.
A Transient Burst of Transgene Expression Promotes Regeneration of Mature Vestibular Hair Cells
Presenter: Joseph Burns, Ph.D.
Session Title: AAV Vectors Preclinical and Proof-of-Concept Studies in CNS Disorders
Date & Time: Friday, May 15, 10:45 a.m.
About Decibel Therapeutics, Inc.
Decibel Therapeutics, a clinical-stage biotechnology company, has established the world’s first comprehensive drug discovery, development and translational research platform for hearing loss and balance disorders. Decibel is advancing a portfolio of discovery-stage programs aimed at restoring hearing and balance function to further our vision of a world in which the benefits and joys of hearing are available to all. Decibel’s lead therapeutic candidate, DB-020, is being investigated for the prevention of ototoxicity associated with cisplatin chemotherapy. For more information about Decibel Therapeutics, please visit decibeltx.com or follow @DecibelTx.
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