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SPL5AC
- Development stage
- Preclinical
- Lead developer
- SpliSense
- Modality
- Small Molecules, 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
01
Overview
SPL5AC is an antisense oligonucleotide (ASO) drug developed by SpliSense to specifically reduce the expression and production of MUC5AC, a mucin protein implicated in muco-obstructive lung diseases. Overexpression of MUC5AC leads to increased mucus viscosity, airway obstruction, and recurrent infections in conditions such as cystic fibrosis (CF), chronic obstructive pulmonary disease (COPD), asthma, and non-cystic fibrosis bronchiectasis. By targeting MUC5AC mRNA, SPL5AC aims to decrease mucus accumulation in the airways, improve mucus clearance, and reduce infection and inflammation. The drug is being advanced toward first-in-human clinical trials for these indications[1][2][9].
- Other names
- MUC5AC-targeting ASOMUC-5AC-targeting ASOMUC 5AC-targeting ASOMUC5AC ASOMUC-5AC ASOMUC 5AC ASO
02
Targets
MUC5AC (Mucin-5AC)
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