Therapeutics for Rare and Neglected Diseases - Science

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Since TRND began in May 2009, work has begun on five pilot projects. The pilot projects were chosen to establish processes in advance of solicitation, with diversity of project stage, type of disease and collaborators. The five pilot projects include: 1. Schistosomiasis, a neglected disease 2. Hereditary Inclusion Body Myopathy, a rare disease 3. Niemann-Pick C Disease, a rare disease 4. Sickle Cell Disease, a rare disease 5. Chronic Lymphocytic Leukemia, a rare disease The development of therapeutics for these rare or neglected indications has involved and will continue to involve resources in the following areas, performed and/or provided by TRND: 1. Medicinal chemistry 2. Pharmacology 3. Toxicology 4. Pharmacokinetics/pharmacodynamics drug metabolism 5. Efficacy 6. Compound formulation and scale-up 7. Regulatory science and interactions with regulatory agencies including FDA 8. Early stage human clinical trials, as needed In addition to scientific advancement on the individual TRND pilot projects, technology/paradigm development in rare diseases informatics, toxicology, and novel efficacy models was begun. Operational work also continued in the following areas: 1. Determining governance of TRND and holding meetings of the Trans-NIH Staff Advisory Group (TAG). 2. Participating in numerous meetings with companies, academic scientists, and individuals from disease communities interested in learning about TRND. 3. Exploring potential partnerships with interested stakeholders in RNDs to seek opportunities to leverage TRND activities. 4. Crafting a TRND Research & Development Request for Proposals through which future research will be funded. 5. Designing a solicitation and associated support system to bring new projects into the TRND pipeline.