SPL84

SPL84 is an inhaled antisense oligonucleotide (ASO) drug in development for the treatment of cystic fibrosis (CF), specifically targeting patients who carry the 3849+10 kb C->T splicing mutation in the CFTR gene. This mutation leads to aberrant splicing and production of non-functional CFTR protein, which is central to the pathology of CF. SPL84 works by binding to the mutated region of CFTR pre-mRNA, modulating its splicing and promoting production of correctly spliced mRNA, thereby increasing levels of functional CFTR protein. The drug is administered via nebulization directly into the lungs for targeted delivery. Developed by SpliSense, SPL84 has received FDA Fast Track designation and has completed phase 1 studies demonstrating safety and tolerability; it is currently being evaluated in phase 2 clinical trials[1][3][4][5][6][8].

Other names

SPL84-23-1SPL-84-23-1SPL 84-23-1