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ISTH0047
- Development stage
- Preclinical
- Lead developer
- Isarna Therapeutics
- 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
- Subcutaneous
01
Overview
ISTH0047 is a second-generation, phosphorothioate-locked nucleic acid (LNA)-modified antisense oligonucleotide (ASO) gapmer developed by Isarna Therapeutics. It specifically targets transforming growth factor-beta 2 (TGF-beta 2) mRNA, inhibiting its expression and downstream signaling pathways (such as SMAD2 phosphorylation). ISTH0047 is being investigated preclinically for oncology indications, including glioblastoma (glioma), renal cell carcinoma (RCC), breast cancer, and lung metastasis, where it has demonstrated the ability to reduce tumor cell migration, invasion, and metastasis, as well as modulate the tumor microenvironment.
02
Targets
TGFB2 (Transforming growth factor beta 2)
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