P.J. Brooks, PhD, Acting Director of the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS), describes the Bespoke Gene Therapy Consortium (BGTC).

BGTC program is a private-public initiative that is designed to bring together researchers from different perspectives, interests, and expertise, to create gene therapies for rare diseases that otherwise would be not financial viable.   

As Dr. Brooks explains, very few rare diseases have FDA-approved treatments despite the fact that the majority of them are caused by known single-gene alterations and are, thus, candidates for gene therapy. Unfortunately, the development of gene therapies is complex, time consuming, and expensive, especially for rare diseases.  By bringing together private and public researchers, the hope is to streamline therapies that can not only be used for one rare disease but easily adaptable for other rare conditions. 

The National Institutes of Health (NIH) and private partners will contribute approximately $76 million over five years to support BGTC-funded projects. 

Private partners include Biogen Inc., Janssen Research & Development, LLC, Novartis Pharma AG, Pfizer Inc., REGENXBIO Inc., Spark Therapeutics, Takeda Pharmaceutical Company Limited, Taysha Gene Therapies, Thermo Fisher Scientific Inc., and Ultragenyx Pharmaceutical. Several non-profit partners also are involved, including the Alliance for Regenerative Medicine (ARM), the American Society of Gene and Cell Therapy, CureDuchenne, National Institute for Innovation in Manufacturing Biopharmaceuticals, and the National Organization for Rare Disorders (NORD). In addition to NCATS, participating NIH institutes include the Eunice Kennedy Shriver National Institute of Child Health and Human Development; National Eye Institute; National Heart, Lung, and Blood Institute; National Human Genome Research Institute; National Institute of Arthritis and Musculoskeletal and Skin Diseases; National Institute of Dental and Craniofacial Research; National Institute of Mental Health; National Institute of Neurological Disorders and Stroke; National Institute on Deafness and Other Communication Disorders; and the Brain Research Through Advancing Innovative Neurotechnologies (BRAIN) Initiative.

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