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GUT-1
- Development stage
- Unknown
- Lead developer
- GUT
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intralesional, Parenteral
01
Overview
GUT-1 (also known as STNM01) is a synthetic double-stranded RNA oligonucleotide (small interfering RNA) that selectively targets and silences the expression of carbohydrate sulfotransferase 15 (CHST15). CHST15 is a type II transmembrane Golgi protein responsible for the biosynthesis of chondroitin sulfate E (CS-E), a highly sulfated matrix glycosaminoglycan implicated in mucosal inflammation, tissue remodeling, and submucosal fibrosis. Originally developed by Stelic Institute and currently being developed by GUT, GUT-1 is administered via endoscopic submucosal injection. It is in Phase II development for the treatment of moderate-to-severe ulcerative colitis and pancreatic ductal adenocarcinoma, and has been evaluated in Phase I trials for Crohn's disease.
- Other names
- CHST15 siRNACHST-15 siRNACHST 15 siRNACHST15-targeting RNA oligonucleotideCHST-15-targeting RNA oligonucleotideCHST 15-targeting RNA oligonucleotide
02
Targets
CHST15 (Carbohydrate sulfotransferase 15)
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