Creating a Playbook for Bespoke Gene Therapies

While there is a steady stream of new gene therapies expected to be approved in the next decade, there are hundreds of diseases that could benefit from gene therapies but are not pursued by drug developers because they affect too small a population to be considered commercially viable. In an effort to change the economics of gene therapy for ultra-rare diseases, the Foundation for the National Institutes of Health is establishing the Bespoke Gene Therapy Consortium under its Accelerating Medicines Partnership program. The proposed five-year, $102.5 million program involves the National Institutes of Health’s National Center for Advancing Translational Sciences, the U.S. Food and Drug Administration’s Center for Biologics Evaluation and Research, and a group of commercial gene therapy developers. We spoke to P.J. Brooks, deputy director of the Office for Rare Diseases Research at NCATS and one of the architects of the program, about the need it is trying to address, why it is looking beyond translational science to issues including manufacturing and regulation, and how it hopes to accelerate the development of gene therapies for rare diseases. This episode is part of our ongoing Platforms of Hope series that explores advances in gene therapy and gene editing.