GalNAc-siRNA conjugates represent a truly novel class of RNA therapeutics enabling liver hepatocyte-specific delivery of small-interfering RNA (siRNA) payloads at an unprecedented level of efficacy. In vitro, and also in vivo,GalNAc conjugated multivalent siRNA is more potent than siRNA alone and th...
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Review of GalNAc-siRNA Conjugates Across Multiple Programs Demonstrates No Evidence of Thrombocytopenia or Pro-inflammatory EffectsCoagulation dysfunction, predominantly mediated by thrombocytopenia and immunostimulatory effects, including complement activation, has been associated with a number of oligonucleotides ...
Alnylam's RNAi therapeutics use novel delivery methods including lipid nanoparticles (LNPs) and conjugates to deliver our siRNAs-based medicines to target tissues.
Identical siRNAs delivered in lipid nanoparticles or as GalNAc conjugates were dose-adjusted to achieve similar knockdown, but only GalNAc–siRNAs supported an extended duration of activity, illustrating the importance of receptor-mediated siRNA trafficking in the process. Taken together, we provide ...
No apparent functional impact on mitochondria and no significant accumulation of 2′-F-monomers were observed after weekly administration of two GalNAc–siRNA conjugates in rats for ∼2 years. Taken together, the results support the conclusion that 2′-F nucleotides can be safely applied for the...
The strong correlation between human and NHP TTR knockdown suggests that NHPs are predictive of human PD for GalNAc-siRNA conjugates and provides a valuable preclinical model for translational research of other GalNAc-conjugated siRNA-based therapeutic agents. The GalNAc-siRNA conjugate is the first ...
5 -(e)-vinylphosphonate: A stable phosphate mimic can improve the RNAi activity of sirna-galnac conjugates. Chembiochem 2016, 17, 985-989. [CrossRef] [PubMed]Parmar, R. et al. 5′-(E)-Vinylphosphonate: a stable phosphate mimic can improve the RNAi activity of siRNA-GalNAc conjugates. ...
Multiple bioanalytical assays were used to compare the conjugates to determine the pharmacokinetics and pharmacodynamics including LC-MS, qPCR, RISC loading and protein binding. Reducing the number of GalNAc sugars to two had no impact on uptake, distribution, knockdown or RISC loading. A contrasting...