Drug Development Pipeline
This program is working to develop a potential therapy for people with CF who have splicing mutations. Splicing is an essential process in which RNA is cut into pieces and then stitched back together in a specific way. Splicing mutations in the CFTR gene cause the RNA to be cut or stitched incorrectly, leading to a mutated CFTR protein. SPL84 is a short nucleotide, or a small piece of genetic material, that is designed to bind to RNA and change its properties in specific ways. In the case of a splicing mutation, the short nucleotide is designed to ensure that the RNA is cut and stitched correctly, allowing functional CFTR protein to be made.
A Phase 1 study to test the safety and tolerability of SPL84 is currently underway. Part 1 of the study will enroll healthy volunteers. Part 2 of the study will enroll adults with CF who have at least one copy of the 3849 +10 Kb C->T mutation.
This program is sponsored by SpliSense and partially funded by the Cystic Fibrosis Foundation.
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