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antimiR-23b
- Development stage
- Unknown
- Lead developer
- INCLIVA Biomedical Research Institute
- Modality
- 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, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intraarticular, Intravenous, Ophthalmic, Subcutaneous
01
Overview
antimiR-23b is an investigational chemically modified, single-stranded antisense oligonucleotide (antimiR) designed to inhibit microRNA-23b (miR-23b). miR-23b is a natural repressor of muscleblind-like (MBNL) proteins, which are downregulated in myotonic dystrophy. By inhibiting miR-23b, antimiR-23b promotes the upregulation of functional MBNL1 and MBNL2 proteins and reduces toxic DMPK transcripts, thereby correcting splicing defects and addressing the underlying molecular pathology of myotonic dystrophy type 1 (DM1) and type 2 (DM2). Developed by ARTHEx Biotech (a spin-off from the University of Valencia and INCLIVA), its lead clinical-stage formulation, ATX-01, is an oleic acid-conjugated antimiR-23b being evaluated in Phase 1/2 clinical trials for DM1.
- Other names
- antagomiR-23bantagomiR23bantagomiR 23bantimiR-23bantimiR23bantimiR 23bantimiR-23b-V2antimiR23b-V2antimiR 23b-V2
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