Treatment of Erythropoietin Deficiency in Mice with Systemically Administered...
W. Querbes et al., Blood. 18 May2012 (Epub) Abstract: Anemia linked to a relative deficiency of renal erythropoietin production is a significant cause of morbidity and medical expenditures in the...
View ArticleMolecularly Self-assembled Nucleic Acid Nanoparticles for Targeted in Vivo...
H.Lee et al.,Nat Nanotechnol. 2012 Jun 3;7(6):389-93. doi: 10.1038/nnano.2012.73. Abstract: Nanoparticles are used for delivering therapeutics into cells. However, size, shape, surface chemistry and...
View ArticleHarnessing a Physiologic Mechanism for siRNA Delivery with Mimetic...
T.Nakayama et al., Molecular Therapy.2012 Feb 1;Epub 2012 Jun 05. Abstract: Therapeutics based on RNA interference (RNAi) have emerged as a potential new class of drugs for treating human disease by...
View ArticleMaximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene...
M. Jayaraman et al., Wiley. 2012 Jul 10 (Epub). Abstract: Special (lipid) delivery: The role of the ionizable lipid pKa in the in vivo delivery of siRNA by lipid nanoparticles has been studied with a...
View ArticleNew Data from Conjugate Delivery Platform
New data from our conjugate delivery platform was presented at the XX International Roundtable on Nucleosides, Nucleotides and Nucleic Acids held in August 2012. This delivery platform enables...
View Article8th Annual Meeting of the Oligonucleotide Therapeutics Society
At the 8th Annual Meeting of the Oligonucleotide Therapeutics Society, held in Boston October 28-31, 2012, we presented exciting data covering several of the company’s therapeutic programs and delivery...
View ArticleRNAi Roundtable: Conjugate Delivery
In December 2012, we hosted a roundtable discussion focused on our activities and progress with our conjugate platform to deliver RNAi therapeutics subcutaneously. The roundtable included a review of...
View ArticleImage-based analysis of lipid nanoparticle–mediated siRNA delivery,...
Gilleron et al., Nat Biotechnol. 2013 Jun23 (Epub doi:10.1038/nbt.2612) Abstract: Delivery of short interfering RNAs (siRNAs) remains a key challenge in the development of RNA interference (RNAi)...
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Efficiency of siRNA delivery by lipid nanoparticles is limited by endocytic...
Sahay et al., Nat Biotechnol. 2013 Jun23 (Epub doi:10.1038/nbt.2614) Abstract: Despite efforts to understand the interactions between nanoparticles and cells, the cellular processes that determine the...
View ArticleBiodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for...
Maier et al., Mol Ther. 2013 Jun25 (Epub doi: 10.1038/mt.2013.124) Abstract In recent years, RNA interference (RNAi) therapeutics, most notably with lipid nanoparticle-based delivery systems, have...
View ArticlePre-Clinical Data on Pharmacology of GalNAc-siRNA Conjugates
We presented new pre-clinical data on the pharmacology of GalNAc-siRNA conjugates at the 12th US-Japan Symposium on Drug Delivery Systems held December 16 – 20, 2013 in Lahaina, Maui, Hawaii. These new...
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