Abstract
The last years shown enormous advancement in the therapeutic potential of RNA treatments, specifically antisense oligonucleotides (ASO), leading to increased numbers of ASO regulatory approvals. SpliSense is developing inhaled ASOs for the treatment of pulmonary diseases; among them are Cystic Fibrosis (CF) mutations with an unmet need. SpliSense’s most advanced program, SPL84, modulates the splicing to produce normal CFTR proteins in 3849 + 10 kb C->T patients. SPL84 was shown to restore CFTR activity in patient cells. Pulmonary drug delivery is challenging, due to a variety of biological, physical, chemical, and structural barriers, especially when targeting the nucleus where splicing occurs. The thick mucus layer covering CF airways is a greater obstacle for drug penetration.
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