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huntingtin antisense oligonucleotide
- 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
Huntingtin antisense oligonucleotide (HTT-ASO) is a preclinical therapeutic candidate being developed by Ophidion for the treatment of Huntington's disease. The drug consists of an antisense oligonucleotide designed to selectively bind to huntingtin (HTT) mRNA, facilitating its degradation and thereby reducing the expression of the mutant huntingtin protein responsible for neurodegeneration. A key feature of this program is the use of Ophidion's proprietary Oligonucleotide Blood-Brain Barrier Crossing Technology (OCCT), a nanoparticle-based delivery system that enables the ASO to cross the blood-brain barrier following systemic administration. This approach aims to provide a less invasive and more effective method of delivering genetic therapies to the central nervous system compared to direct intracranial or intrathecal injections.
- Other names
- Huntingtin ASO
02
Targets
HTT mRNA (HTT / Huntingtin gene)
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