Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
Gli1 ASO
- Development stage
- Preclinical
- Lead developer
- Ionis Pharmaceuticals
- 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
- Intratumoral, Intravenous, Subcutaneous
01
Overview
Gli1 ASO refers to a class of antisense oligonucleotides designed to target and silence the expression of the GLI1 (GLI family zinc finger 1) transcription factor. GLI1 is a critical downstream effector of the Hedgehog (Hh) signaling pathway, which is frequently dysregulated in various malignancies, including basal cell carcinoma, medulloblastoma, and pancreatic cancer. By binding to GLI1 mRNA through Watson-Crick base pairing, these ASOs typically induce RNase H-mediated degradation of the transcript, thereby reducing GLI1 protein levels and inhibiting the transcription of Hh-target genes involved in cell proliferation, survival, and epithelial-mesenchymal transition. This approach is particularly relevant in cancers that have developed resistance to upstream Hedgehog pathway inhibitors, such as SMO antagonists, as GLI1 acts at the terminal end of the signaling cascade.
- Other names
- GLI1 antisense oligonucleotideGLI-1 antisense oligonucleotideGLI 1 antisense oligonucleotideanti-GLI1 ASOanti-GLI-1 ASOanti-GLI 1 ASO
02
Targets
GLI1
Beyond the preview
Go deeper on Gli1 ASO.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on Gli1 ASO.