GalNAc-siRNA Conjugates: Leading the Way for Delivery of …
GalNAc-siRNA conjugates are a simple solution to the siRNA delivery problem for liver hepatocytes and have shown the RNAi (and antisense oligonucleotide) field the path forward for targeting other tissue types.
Drug delivery systems for RNA therapeutics - Nature
2022年1月4日 · Like the mRNA-LNP vaccines, GalNAc–siRNA conjugates can easily be redesigned to treat different diseases, since only the siRNA sequence needs to change.
RNAi-based drug design: considerations and future directions
2024年4月3日 · In this Review, we discuss principal considerations and future directions in the design of therapeutic siRNAs, with a particular emphasis on chemistry, the application of informatics, delivery...
Delivery of Oligonucleotides to the Liver with GalNAc: From …
2020年8月5日 · Summary of GalNAc-siRNA or GalNAc-ASO Conjugates in the Clinic from the Leading Oligonucleotide Platform Companies
Pharmacokinetics and Pharmacodynamics of GalNAc-Conjugated …
To support the continuous development of siRNAs, it is important to have a thorough and deep understanding of the PK/PD and clinical pharmacology related features of siRNAs.
Less Is More: Novel Hepatocyte-Targeted siRNA Conjugates for …
We applied a versatile monovalent serinol-GalNAc conjugation strategy. First, 1–4 serial serinol-linked GalNAc units were conjugated to terminal positions of small interfering RNA (siRNA) molecules.
GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic …
2023年5月15日 · Building on prior evidence that the addition of a GalNAc-Lipid targeting ligand can facilitate hepatic delivery of siRNA and ASO therapeutics, we describe a series of experiments to optimize...
Delivery of Oligonucleotides to the Liver with GalNAc: From …
2020年8月5日 · Targeted delivery of oligonucleotides to liver hepatocytes using N -acetylgalactosamine (GalNAc) conjugates that bind to the asialoglycoprotein receptor has become a breakthrough approach in the therapeutic oligonucleotide field.
Initial Results from a Phase 1/2 Study Evaluating Divesiran, a Novel ...
2024年11月5日 · Divesiran (SLN124), a liver-targeted GalNAc-conjugated double-stranded 19-mer siRNA increases hepatic hepcidin synthesis and plasma levels by silencing TMPRSS6 (a negative regulator of hepcidin production), and restricting iron availability for erythropoiesis. Previously, in healthy volunteers divesiran was reported to be well-tolerated and ...
Advanced siRNA Designs Further Improve In Vivo Performance of GalNAc …
2018年3月7日 · To achieve this, we employed an iterative screening approach across multiple siRNAs to arrive at advanced designs with low 2'-deoxy-2'-fluoro content that yield significantly improved potency and duration in preclinical species, including non-human primate.
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