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NMT.001 (Neumirna Therapeutics)
- Development stage
- Preclinical
- Lead developer
- Neumirna Therapeutics
- Modality
- 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
- Intrathecal
01
Overview
NMT.001 (formerly known as NEUmiRNA lead ASO) is a preclinical-stage, chemically modified mixmer antisense oligonucleotide (ASO) designed to selectively inhibit microRNA-134 (miR-134). Developed by Neumirna, NMT.001 targets miR-134, a brain-enriched microRNA that is upregulated in temporal lobe epilepsy and regulates neuronal microstructure and excitability. By silencing miR-134, NMT.001 aims to restore balanced gene expression networks, offering a potentially disease-modifying and curative treatment for drug-resistant epilepsy (DRE) and its associated cognitive and behavioral comorbidities.
- Other names
- NEUmiRNA lead ASOanti-miR-134 ASOanti-miR134 ASOanti-miR 134 ASO
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