Small molecule bicyclic and tricyclic cftr correctors
AssigneeDiscoverybiomed, Inc.
InventorsErik Schwiebert, John Streiff, John Dixon, Hongwu Gao
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AssigneeDiscoverybiomed, Inc.
InventorsErik Schwiebert, John Streiff, John Dixon, Hongwu Gao
Estimating the term…
Novel CFTR corrector compounds that are effective in rescuing halide efflux, delF508-CFTR protein processing, and apical functional chloride ion transport in a cell are provided. Also provided are methods for treating protein folding disorders (e.g., cystic fibrosis). The methods include administering a CFTR corrector compound or pharmaceutically acceptable salt or prodrug thereof. Methods of rescuing halide efflux in a cell, correcting a processing defect of a delF508-CFTR protein in a cell, and correcting functional delF508-CFTR chloride channels in a cell are also provided.
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