ASO for Splicing Mutation Restores CFTR Protein in Patient Cell Study

ASO for Splicing Mutation Restores CFTR Protein in Patient Cell Study

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ASO for Splicing Mutation Restores CFTR Protein in Patient Cell Study

SPL84-23, an investigational antisense oligonucleotide (ASO), restored the levels and function of CFTR — the defective protein in cystic fibrosis (CF) — in lab-grown cells from patients carrying a common and disease-causing splicing mutation, a study reported. Based on these positive preclinical findings, SpliSense plans to launch a Phase 1/2 clinical trial next year of the potential therapy in people with CF caused by this mutation, called 3849+10kb C-to-T. The study, “Antisense oligonucleotide-based drug development for Cystic Fibrosis…

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