Drug Development Pipeline
This program is studying messenger RNA (mRNA) delivery to treat rare CFTR mutations, including 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 mRNA would allow lung cells to make full-length, functional CFTR protein.
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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