Page Title
Drug Development Pipeline
SPL84
Status
Phase TwoTherapeutic Approach
Genetic Therapy
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.
Status
A Phase 2 clinical trial is underway to test the effectiveness of SPL84 in adults with CF who have at least one copy of the splicing mutation 3849+10Kb C-to-T.
A recent Phase 1a study in healthy volunteers showed that the drug was safe and well tolerated.
Sponsor
This program is sponsored by SpliSense and partially funded by the Cystic Fibrosis Foundation.

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