SOMERSET, NEW JERSEY & BOSTON, MASSACHUSSETTS — Catalent, the leading 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, today announced they have signed an agreement for Catalent to provide Decibel with process and analytical development, and CGMP clinical manufacturing services1, 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. We look forward to working with Catalent on the development of DB-OTO as we progress towards clinical trials.”
–John Lee, EVP, Pharmaceutical Development and Interim Chief Scientific Officer of Decibel
“Partnering early with innovative companies allows us to develop and optimize robust, scalable manufacturing processes and the analytical methods to assess them,” commented Manja Boerman, Ph.D., President, Catalent Cell & Gene Therapy. “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.
- CGMP refers to the Current Good Manufacturing Practice regulations enforced by the FDA. CGMPs provide for systems that assure proper design, monitoring, and control of manufacturing processes and facilities.
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 program, 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.
About Catalent Cell & Gene Therapy
With deep experience in viral vector scale-up and production, Catalent Cell & Gene Therapy is a full-service partner for adeno-associated virus (AAV) and lentiviral vectors, and CAR-T immunotherapies. When it acquired MaSTherCell, Catalent added expertise in autologous and allogeneic cell therapy development and manufacturing to position it as a premier technology, development and manufacturing partner for innovators across the entire field of advanced biotherapeutics. Catalent has a global cell and gene therapy network of dedicated, large-scale clinical and commercial manufacturing facilities, and fill-finish and packaging capabilities located in both the U.S. and Europe. An experienced partner, Catalent Cell & Gene Therapy has worked with industry leaders across 70+ clinical and commercial programs. For more information, visit biologics.catalent.com/cell-gene-therapy/
Catalent is the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products. With over 85 years serving the industry, Catalent has proven expertise in bringing more customer products to market faster, enhancing product performance and ensuring reliable global clinical and commercial product supply. Catalent employs approximately 15,000 people including around 2,400 scientists and technicians, at more than 45 facilities, and in fiscal year 2020 generated over $3 billion in annual revenue. Catalent is headquartered in Somerset, New Jersey.
Source: Decibel Therapeutics, Catalent