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TKM-100201
- Development stage
- Discontinued
- Lead developer
- Arbutus Biopharma
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
TKM-100201 is an investigational RNA interference (RNAi) therapeutic developed for the treatment of Ebola virus infection. It consists of small interfering RNAs (siRNAs) formulated in stable nucleic acid lipid particles (SNALP), designed to target and silence specific Ebola virus genes. The siRNAs in TKM-100201 are directed against the mRNAs encoding the viral polymerase L protein, matrix protein VP40, and polymerase cofactor VP35 of Zaire ebolavirus (EBOV-Zaire). By inhibiting these essential viral proteins, TKM-100201 aims to prevent viral replication and spread within the host. The drug was initially developed by Protiva Biotherapeutics and later by Arbutus Biopharma. Preclinical studies demonstrated efficacy in nonhuman primate models with significant protection against lethal EBOV challenge when administered intravenously after exposure. Clinical development included a phase 1 trial in healthy volunteers; however, further development was discontinued following limited clinical success[1][2][3][5][6].
- Other names
- ebola snalp
02
Targets
Zaire ebolavirus RNA-directed RNA polymerase L mRNAVP35 (Ebola virus VP35)Zaire ebolavirus matrix protein VP40 mRNA
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