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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, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → 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)
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