Alnylam Pharmaceuticals Inc (ALNY)

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In addition to new data on the Company’s ESC+ platform, Alnylam scientists and collaborators presented additional pre-clinical findings showing continued leadership in RNAi technologies and delivery. First, Alnylam scientists presented further advances toward optimizing the Company’s GalNAc-siRNA conjugate platform. These included studies to further improve the mechanistic understanding of conjugate duration of activity as well as the development of advanced ESC designs with significantly improved metabolic stability and in vivo efficacy. Further, new data on Alnylam’s Reversir™ platform were presented. Specifically, optimizations were implemented that enable rapid reversal of siRNA-mediated mRNA silencing, providing the means to fine-tune the pharmacology of GalNAc-siRNA conjugates. Finally, pre-clinical data demonstrating extra-hepatic siRNA delivery, involving Centyrins, a novel class of highly stable FN3 domain proteins, were also presented as part of a research collaboration with Janssen Research & Development, LLC. Centyrin-siRNA conjugates showed excellent cross-tumor penetration and mediated robust target knockdown in a mouse xenograft tumor model. Also, the generalizability of this approach was demonstrated in vitro using Centyrins for a number of different receptors and gene targets.