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The Bespoke Gene Therapy Consortium (BGTC) launched in October to bring together 27 partners to ease the roadblocks to gene therapy development for ultra-rare diseases. The 5-year, US$76 million initiative will tackle precompetitive research challenges, establish a standardized development framework, take on manufacturing hurdles and set up a streamlined regulatory path for these agents. The partners include NIH institutes, the FDA, 10 industry players and not-for-profit organizations.