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Find Clinical Drug Pipeline Developments & Deals by SpliSense
The funding will support SpliSense to continue the clinical development of an inhaled antisense oligonucleotide drug SPL84 for treating patients suffering from cystic fibrosis.
SPL84 is an antisense oligonucleotide product that binds specifically to the mutated CFTR RNA in the targeted sequence. It is being evaluated for the treatment of cystic fibrosis.
SPL84, the Company's lead inhaled anti sense oligonucleotide (ASO) product for the treatment of patients with CF, carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the transmembrane conductance regulator (CFTR) gene.
SPL84, has shown to have completely restored CFTR activity in the CF gold standard pharmacological model, suggesting potential cure for these patients.
Data show that SPL23-ASO, targeting the W1282X nonsense mutation, enables production of a functional cystic fibrosis transmembrane conductance regulator protein.
Proceeds will be used to advance the Company’s pipeline, including its lead Anti Sense Oligonucleotide product, SPL84-23. SPL84-23 to treat the 3849+10kb C->T CFTR mutation, that has showed ability to completely restore CFTR channel function in patient-derived cell cultures.