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SPL24

Development stage: Preclinical
Lead developer: SpliSense
Modality: Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration: Inhalation

Overview

SPL24 is an antisense oligonucleotide (ASO) being developed by SpliSense Therapeutics for the treatment of Cystic Fibrosis. It specifically targets the N1303K mutation in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. The N1303K mutation is a common "non-responder" mutation that does not typically benefit from current CFTR modulator therapies. SPL24 is designed to modulate the splicing or expression of the CFTR mRNA to restore functional protein production. It is currently in the preclinical stage of development and is intended for delivery via inhalation.

Targets

CFTR (Cystic fibrosis transmembrane conductance regulator)

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