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FM-hsiRNAHTT
- Development stage
- Preclinical
- Lead developer
- UMass Chan Medical School
- 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
- Administration
- Intratumoral, Intrathecal
01
Overview
FM-hsiRNAHTT is a fully chemically stabilized, hydrophobically-modified self-delivering small interfering RNA (hsiRNA) designed to target and silence the huntingtin (HTT) mRNA. Developed by the Khvorova laboratory at the University of Massachusetts Medical School, this platform utilizes chemical modifications to enhance metabolic stability and facilitate cellular uptake without the need for additional delivery vehicles. While HTT is the primary target in Huntington's disease research, this specific compound has been utilized in preclinical studies as a tool for in vivo target validation in aggressive glioblastoma (GBM) models, demonstrating robust and long-lasting gene silencing following intratumoral administration.
- Other names
- hsiRNAHTT
02
Targets
HTT mRNA (HTT / Huntingtin gene)
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