Work has continued on the five TRND pilot projects, which 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 In FY11, the first TRND project solicitation was successfully launched. Sixty-three proposals were received in response to the solicitation;these proposals came from industry, academia, government laboratories, and not-for-profit institutions. The proposals were reviewed by an external panel of experts. Four projects were selected for collaboration, representing a diverse group of project types: 1. Duchenne Muscular Dystrophy, a rare disease 2. Fragile X Syndrome, a rare disease 3. Cryptococcal meningitis, a neglected disease 4. CBF leukemia, a rare disease A second solicitation was also launched during FY11, and proposals will be selected for collaboration during FY12. 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 FY11, two of the TRND pilot projects advanced to the Investigational New Drug (IND) application phase. In addition to scientific advancement on the individual TRND pilot projects, technology/paradigm development in rare diseases informatics, toxicology, and novel efficacy models has continued. 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.
| Kodippili, Kasun; Hakim, Chady H; Pan, Xiufang et al. (2018) Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine Model. Hum Gene Ther 29:299-311 |
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| Terry, Sharon; Austin, Christopher; Inglese, James et al. (2010) Assay, preclinical, and clinical brick walls and opportunities for system change through GRANDRx. Assay Drug Dev Technol 8:128-34 |
