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Jack's Tomorrow is a U.S.-based nonprofit rare disease organization focused on funding and directing research to develop treatments for PURA Syndrome, a neurogenetic disorder caused by mutations in the PURA gene. Rather than operating as a traditional drug developer, the organization supports translational research, disease models, and therapeutic-enabling studies with academic and nonprofit research partners. Its work has included funding induced pluripotent stem cell models, mouse and other translational tools, and gene overexpression studies intended to de-risk future gene therapy approaches for PURA Syndrome. The group appears to function as a patient-led research catalyst and advocacy organization, helping build the scientific infrastructure needed for eventual therapeutic development.
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