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EDN-OL1
- Development stage
- Discontinued
- Lead developer
- Edunn Biotechnology
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → 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
- Intravenous
01
Overview
EDN-OL1 is a patented antisense phosphorothioate oligonucleotide developed by Edunn Biotechnology for the treatment of Alzheimer's disease and CNS symptoms associated with Down syndrome. The drug is designed to cross the blood-brain barrier following intravenous administration. Its mechanism of action involves targeting the amyloid precursor protein (APP) mRNA to reduce the production of APP and subsequent amyloid-beta proteins. In preclinical murine models, EDN-OL1 demonstrated the ability to restore cognitive function and potentially halt or decrease the rate of cognitive decline. Development was previously supported through a collaboration with the National Center for Advancing Translational Sciences (NCATS), which has since concluded due to a change in the company's business strategy.
02
Targets
Amyloid-beta precursor protein messenger RNA
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