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SPL5B
- 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
01
Overview
SPL5B is an inhaled antisense oligonucleotide (ASO) being developed by SpliSense for the treatment of pulmonary diseases characterized by mucus hypersecretion and obstruction, such as cystic fibrosis (CF) and idiopathic pulmonary fibrosis (IPF). The drug is designed to target and modulate the expression of the *MUC5B* gene, which encodes one of the primary gel-forming mucins in the human respiratory tract. By reducing the overproduction of MUC5B protein, SPL5B aims to decrease mucus viscosity, improve airway clearance, and alleviate the symptoms of chronic lung disease. The program is currently in early-stage development and has received financial support from the Cystic Fibrosis Foundation to advance toward clinical trials.
02
Targets
MUC5B (Mucin-5B)
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