Drug Development Pipeline
Restore CFTR Function
This program is developing suppressor tRNA delivery to treat nonsense mutations. Nonsense mutations (also known as “x” or “stop” mutations) in the CFTR gene cause the production of CFTR protein to stop prematurely. Delivery of suppressor tRNA would allow lung cells to override these premature stop signals and make full-length, functional CFTR protein.
An mRNA delivery program is also being studied. mRNA is a molecule that contains genetic instructions to make proteins. Delivery of CFTR-encoded mRNA would allow the lung cells to create normally functioning CFTR protein, regardless of an individual’s specific CFTR gene mutation.
Laboratory studies to develop and test this potential therapy are underway.
This program is sponsored by ReCode Therapeutics and partially funded by the Cystic Fibrosis Foundation.
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