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ACP-siRNA
- Development stage
- Preclinical
- Lead developer
- Avixgen
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Peptide-Drug Conjugates → Peptide Conjugates → Peptides, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
ACP-siRNA is an advanced cell-penetrating peptide (ACP)-siRNA conjugate being developed by DxVx and its subsidiary Avixgen for the treatment of central nervous system (CNS) disorders. The therapeutic candidate utilizes the proprietary ACP platform, a peptide-based drug delivery system designed to efficiently cross the blood-brain barrier (BBB) without toxicity. Once inside the brain, the siRNA component induces cleavage of target mRNA, thereby suppressing the expression of disease-related proteins. ACP-siRNA is currently in preclinical development, with studies demonstrating high BBB permeability and target gene silencing in animal models of neurodegenerative diseases such as Parkinson's disease.
- Other names
- ACP-siRNA conjugateAdvanced Cell-penetrating Peptide-siRNA conjugate
02
Targets
Innate immune RNA sensorsAGO2 (Argonaute RISC catalytic component 2)
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