SPL16

SPL16 is an antisense oligonucleotide (ASO) being developed by SpliSense Therapeutics for the treatment of cystic fibrosis. It specifically targets the CFTR 2789+5G->A mutation (and other 2789-series splicing mutations) within the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. The drug is designed to bind to the pre-mRNA and modulate the splicing process, correcting the defect caused by the mutation and restoring the production of functional CFTR protein. SPL16 is intended for administration via inhalation, allowing for direct delivery to the lung epithelial cells where the protein deficiency causes the most severe clinical symptoms. As of late 2022, the program was in the IND-enabling phase of development.