Catalent, a global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products, and Decibel Therapeutics, a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments for hearing and balance disorders, have signed an agreement for Catalent to provide Decibel with process and analytical development, and CGMP clinical manufacturing services, for Decibel’s lead investigational gene therapy product candidate, DB-OTO.
 
DB-OTO is a dual-vector adeno-associated virus (AAV) gene therapy that is designed to restore hearing to individuals with profound, congenital hearing loss caused by mutations in the otoferlin gene. Decibel is developing DB-OTO in collaboration with Regeneron Pharmaceuticals. Under the terms of the agreement, Catalent will provide material from its Maryland-based gene therapy facilities to support Decibel’s planned IND-enabling studies and Phase 1/2 clinical trial of DB-OTO.
 
“There are currently no approved therapies that treat congenital deafness caused by genetic deficiencies.  In our preclinical studies, we have delivered full-length, functional otoferlin protein under the control of a cell-selective promoter which restored hearing function in a mouse model of otoferlin deficiency, demonstrated cell selective expression in non-human primates, and preliminary safety and tolerability of delivery of DB-OTO,” said John Lee, executive vice president, pharmaceutical development and interim chief scientific officer, Decibel. “We look forward to working with Catalent on the development of DB-OTO as we progress towards clinical trials.”
 
Manja Boerman, president, cell and gene therapy, Catalent, said, “Partnering early with innovative companies allows us to develop and optimize robust, scalable manufacturing processes and the analytical methods to assess them. Our Maryland development centers in Gaithersburg and the University of Maryland BioPark in Baltimore focus on providing process optimization services to meet our customers’ needs for early-stage clinical gene therapies.”
 
Catalent has five gene therapy facilities in Maryland that provide services from clinical- to commercial-scale, and house multiple CGMP manufacturing suites, including fill/finish, testing laboratories, warehousing, supply chain capabilities, and other associated services.