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ApoB11:2'-OMe ASO
- Development stage
- Preclinical
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, 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, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intraperitoneal
01
Overview
ApoB11:2'-OMe ASO is a synthetic antisense oligonucleotide (ASO) conjugated to the ApoB11 peptide, designed to enhance blood-brain barrier penetration and central nervous system (CNS) delivery. It utilizes 2'-O-methyl chemical modifications for improved stability and reduced toxicity. The primary mechanism involves sequence-specific binding to the mRNA encoding α-synuclein (SNCA), leading to its downregulation through RNase H1-mediated degradation. In preclinical mouse models of synucleinopathies, including dementia with Lewy bodies, systemic intraperitoneal administration resulted in significant CNS accumulation, prolonged half-life, and robust suppression (~50%) of α-synuclein mRNA and protein in target brain regions, with favorable safety profiles observed at therapeutic doses. This supports ApoB11:2'-OMe ASO’s potential as a disease-modifying therapy for neurodegenerative disorders characterized by pathogenic α-synuclein accumulation[1][2].
- Other names
- ApoB11:2'-OMe ASOApoB11:ASO-α-Syn
02
Targets
Alpha-synuclein messenger RNA
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