(Overall for Pilot and Feasibility Program) The Pilot and Feasibility Program, directed by Dr. Verkman, is focused on the identification of CF targets, the development of screening assays and screening paradigms, and the identification of drug candidates. Two Pilot Project slots will be allocated for years 16?20. Each Pilot Project runs for a maximum of 2 years, with second year funding requiring satisfactory progress and justification for continued funding. Pilot project selection involves project recruitment, letters of intent, initial review by an Internal Advisory Committee, and generally external review by our External Advisory Committee and ad hoc reviewers. Recent accomplishments of the Pilot Program include manuscripts, patents, new grants, and potential out-licensing of CF drug candidates. The proposed pilots for funding in year 16 focus on a potential new mutation-agonist CF target, SLC26A9, and therapeutic development for a rare CF-causing CFTR mutation, C.3700A>G.
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