Therapeutics for Rare and Neglected Diseases - Science

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Work has continued on the six TRND pilot projects, which were chosen to establish processes in advance of solicitation with diversity of project stage, type of disease, and collaborators. The six pilot projects include: 1. Niemann-Pick C Disease, a rare disease 2. Hereditary Inclusion Body Myopathy, a rare disease 3. Giardiasis, a neglected disease 4. Schistosomiasis, a neglected disease 5. Sickle Cell Disease, a rare disease 6. Chronic Lymphocytic Leukemia, a rare disease In FY11, the first two TRND project solicitations were successfully launched. One hundred twenty-five proposals were received in response to these solicitations; these proposals came from industry, academia, government laboratories, and not-for-profit institutions. The proposals were reviewed by an external panel of experts. Ten projects were selected for collaboration, representing a diverse group of project types: 1. Duchenne Muscular Dystrophy, a rare disease 2 different projects 2. Fragile X Syndrome, a rare disease 3. Cryptococcal meningitis, a neglected disease 4. CBF leukemia, a rare disease 5. Neonatal Herpes Simplex Virus, a rare disease 6. Pulmonary Alveolar Proteinosis, a rare disease 7. Fibrodysplasia Ossificans Progressiva, a rare disease 8. Schistosomiasis, a neglected disease 9. Creatine Transporter Defect, a rare disease A third solicitation was also launched during FY12, and proposals will be selected for collaboration during FY13. Within two years of initiation, two projects (Aes-103 for Sickle Cell Disease and Auranofin for Chronic Lymphocytic Leukemia) yielded successful IND applications to the FDA, and first in-human clinical trials are ongoing in both. Two additional projects are on schedule for IND submission in FY13. One project has been discontinued for failure to meet milestones. All other projects have had collaborative and milestone agreements put in place, and are achieving interim milestones and progressing according to schedules. 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 Optimization 2. Pharmacokinetics / Pharmacodynamics 3. Toxicology 4. Dosing 5. Formulation 6. Regulatory Support 7. Project Management In addition to meeting its project-specific goals, TRND has developed novel technologies and collaborative paradigms that improve the efficiency of the translational process. For example, two projects are developing novel platform technologies that can be used to develop therapeutics to treat a variety of other human disorders. 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 and finalizing a TRND Research & Development Request for Proposals through which future research will be funded. 5. Designing and evaluating the solicitation and associated support system to bring new projects into the TRND pipeline.