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SPL23
- Development stage
- Preclinical
- Lead developer
- SpliSense
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Inhalation
01
Overview
SPL23 (also known as SPL23-2) is an inhaled antisense oligonucleotide (ASO) developed by SpliSense for the treatment of cystic fibrosis (CF) in patients carrying the W1282X nonsense mutation. The drug works by binding to the CFTR pre-mRNA and inducing the skipping of exon 23. This mechanism bypasses the premature termination codon introduced by the W1282X mutation and prevents nonsense-mediated mRNA decay (NMD), thereby allowing the production of a shortened but functional CFTR protein. This approach is particularly significant for CF patients who do not produce functional CFTR protein and are ineligible for current CFTR modulator therapies.
02
Targets
Cystic fibrosis transmembrane conductance regulator (CFTR) exon 23 3' splice siteCFTR (Cystic fibrosis transmembrane conductance regulator)
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