Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
HOXB-AS3 LNA gapmer
- Development stage
- Preclinical
- Lead developer
- The Ohio State University
- 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
- Intravenous, Intraperitoneal
01
Overview
HOXB-AS3 LNA gapmer is a research-stage antisense oligonucleotide (ASO) designed as a locked nucleic acid (LNA) gapmer to target the long non-coding RNA (lncRNA) HOXB-AS3. HOXB-AS3 is significantly overexpressed in NPM1-mutated acute myeloid leukemia (AML) and plays a critical role in regulating ribosomal RNA (rRNA) transcription by interacting with EBP1 and guiding it to the rDNA locus. By inducing the knockdown of HOXB-AS3, the gapmer suppresses rRNA synthesis and de novo protein production, leading to inhibited cell proliferation and reduced colony formation in leukemic cells. Preclinical studies in patient-derived xenograft models have demonstrated that treatment with this gapmer can prolong overall survival. It was primarily developed as a research tool by academic institutions, including The Ohio State University and Aalborg University.
- Other names
- HOXB-AS3 antisense oligonucleotideHOXB-AS-3 antisense oligonucleotideHOXB-AS 3 antisense oligonucleotideHOXB-AS3 gapmerHOXB-AS-3 gapmerHOXB-AS 3 gapmer
02
Targets
HOXB-AS3 (HOXB cluster antisense RNA 3)
Beyond the preview
Go deeper on HOXB-AS3 LNA gapmer.
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 HOXB-AS3 LNA gapmer.