P.J. Brooks, PhD, Acting Director of the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS), describes NCATS’ push to increase efficiency and decrease costs in the development of rare disease treatments.

As Dr. Brooks explains, fewer than 10% of rare diseases have FDA-approved treatments despite the fact that roughly 80% of them are caused by known single-gene alterations and are, thus, candidates for gene therapy. However, the development of gene therapies is complex, time consuming, and expensive, especially for rare diseases. 

One solution to increase efficiency is to apply a “many-diseases-at-a-time” approach, which has been successful in the development of cancer treatments. To encourage this approach, NCATS is leading a number of initiatives including the Bespoke Gene Therapy Consortium (BGTC) and Platform Vector Gene Therapy (PaVe-GT) pilot project. These two efforts serve different but complementary goals. The PaVe-GT pilot project is testing methods to increase the efficiency of gene therapy trial startup by using a standardized process, with the same capsid and manufacturing methods, for four different rare diseases while the BGTC program is a private-public initiative that hopes to streamline the path from animal studies to human testing. For the latter program, researchers will conduct four to six clinical trials using a streamlined approach with each trial focused on a different rare disease. 

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