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ASO6
- Development stage
- Preclinical
- Lead developer
- Ophidion
- 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, Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
ASO6 is an antisense oligonucleotide (ASO) therapeutic developed by Ophidion for the treatment of Huntington's disease. It is designed to reduce the expression of the Huntingtin (HTT) gene and the production of the mutant Huntingtin protein. The drug utilizes Ophidion's proprietary OCCT (Ophidion CNS-targeted) nanoparticle delivery platform, which employs a receptor-mediated transport mechanism to cross the blood-brain barrier (BBB) following intravenous administration. Preclinical studies in mouse and non-human primate (NHP) models have demonstrated successful HTT gene suppression and protein reduction in the central nervous system. As of late 2024, the program is in the preclinical stage, with Phase 1b/2a clinical trials anticipated.
- Other names
- peptide:ASO6 complexHTT-ASOOCCT / HTT-ASO
02
Targets
HTT mRNA (HTT / Huntingtin gene)
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