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DFNB77

Development stage: Preclinical
Lead developer: Rescue Hearing
Modality: CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration: Intracochlear

Overview

DFNB77 is a CRISPR/Cas9-based gene therapy being developed by Rescue Hearing to treat nonsyndromic hearing loss caused by mutations in the LOXHD1 gene. The therapy utilizes an adeno-associated viral (AAV) vector to deliver gene-editing components designed to correct or replace pathogenic variants in the LOXHD1 gene. LOXHD1 encodes a protein essential for the structural integrity and function of cochlear hair cell stereocilia. By restoring LOXHD1 activity, the therapy aims to promote hair cell survival and improve auditory function in patients, specifically targeting those within the cochlear implant population who suffer from progressive hearing loss.

Other names: LOXHD1 gene therapyLOXHD-1 gene therapyLOXHD 1 gene therapyRescue Hearing DFNB77

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