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PrP-siRNA
- Development stage
- Phase 1
- Lead developer
- Broad Institute
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
PrP-siRNA is an investigational divalent short interfering RNA (siRNA) developed by the Broad Institute of MIT and Harvard for the treatment of symptomatic prion disease. The therapeutic utilizes a novel divalent architecture—consisting of two chemically modified siRNA molecules joined by a linker—to enhance distribution and gene silencing throughout the central nervous system following intrathecal delivery. By hybridizing with the mRNA encoded by the PRNP gene, the drug triggers the degradation of the transcript, thereby reducing the expression of the prion protein (PrP). This reduction is intended to slow or halt the neurodegenerative progression of prion diseases, which are characterized by the misfolding and aggregation of PrP. The drug is currently being evaluated in the Phase 1 PRiSM trial, marking the first time a divalent siRNA has entered human clinical trials.
- Other names
- divalent PrP-siRNAdivalent siRNA targeting PRNP
02
Targets
PRNP (Cellular Prion Protein)
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