Drug intelligence / Profile preview

Sirna-AV34

Development stage
Unknown
Lead developer
Alnylam Pharmaceuticals
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, Nanoparticles → Drug Delivery Systems
Administration
Parenteral
01

Overview

**Sirna-AV34** was a preclinical, systemically delivered nanoparticle formulation of multiple chemically modified small interfering RNA sequences developed by Sirna Therapeutics for chronic hepatitis C virus infection. The siRNA components were designed to target highly conserved regions of the hepatitis C viral genome, inducing RNA interference-mediated degradation of viral RNA and suppressing viral replication while reducing the opportunity for resistant viral variants to emerge. The program was reported as a clinical-development candidate, with preclinical efficacy in rodent and non-human primate models and manufacturing activities intended to support Phase I development.

02

Targets

HCV RNA (Hepatitis C virus RNA)

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