Decibel Therapeutics announced authorization of its Clinical Trial Application, CTA, by the United Kingdom, U.K., Medicines and Healthcare products Regulatory Agency, MHRA, to initiate a Phase 1/2 clinical trial in pediatric patients of DB-OTO, its lead gene therapy product candidate. DB-OTO is being developed in collaboration with Regeneron Pharmaceuticals, Inc. and is a cell-selective adeno-associated virus gene therapy designed to provide durable hearing in individuals with profound, congenital hearing loss caused by mutations of the otoferlin gene. "This CTA marks the second regulatory clearance for DB-OTO and what we believe to be the first in Europe for any gene therapy targeting otoferlin. This is an important milestone as we execute our international clinical development strategy, which addresses pediatric patients of diverse ages, including infants two years of age and younger," said Laurence Reid, Ph.D., Chief Executive Officer at Decibel. "We believe DB-OTO has transformative potential for individuals with otoferlin-related hearing loss, and we intend to initiate the clinical trial in the first half of 2023. We expect to share initial data from the first cohort of patients in the first quarter of 2024."
Published first on TheFly
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