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Alnylam Pharmaceuticals reports significant progress in discovery of novel technologies for systemic delivery of RNAi therapeutics

January 14, 2020

"Progress on LNP-Mediated Delivery of RNAi Therapeutics," by Martin Maier, Ph.D., Senior Director, Drug Discovery at Alnylam; "Progress in the Development of LNP Delivery Systems for siRNA: Advancing LNPs to the Clinic," by Mark Tracy, Ph.D., Senior Director, Pharmaceutical Operations at Alnylam; "Combinatorial Development of Biomaterials and Synthetic siRNA Delivery Systems," by Professor Daniel Anderson, Ph.D., Department of Chemical Engineering, Harvard-MIT Division of Health Sciences & Technology, David H. Koch Institute for Integrative Cancer Research; "Lipid Nanoparticle Delivery Systems for the Systemic Delivery of siRNA," by Pieter Cullis, Ph.D., Professor Biochemistry and Molecular Biology at UBC; and, "Rational Design of Cationic Lipids for siRNA Delivery: Identifying Critical Structure/Activity Relationships," by Michael Hope, Ph.D., Chief Scientific Officer of AlCana.

In addition, multiple posters were presented at the meeting providing further updates on the systemic delivery of RNAi therapeutics. Specifically, data were presented describing the discovery of additional novel lipids; the use of small molecules in both cis and trans configurations to enhance LNP-based delivery; targeting-based approaches with LNPs; and, the achievement of extra-hepatic delivery of siRNAs, including to immune cells and prostate cancer tumor xenografts, with LNPs.

Posters presented at the meeting included the following:

"Development of Targeted LNPs for the Delivery of RNAi Therapeutics," Soma De, Ph.D., Scientist at Alnylam; "Synthesis and Evaluation of Bicyclic Ketal-based Cationic Lipids for the Delivery of siRNA via Lipid Nanoparticle Systems," by Muthusamy Jayaraman, Ph.D., Principal Scientist at Alnylam; "Influence of Cationic Lipid Composition on Gene Silencing Properties of siRNA in Primary Antigen Presenting Cells," by Genc Basha, Research Associate at UBC; "siRNA Knockdown of the Androgen Receptor using Liposomal Nanoparticles to Treat Advanced Prostate Cancer," by Justin B. Lee, Graduate Student at UBC; "Reverse Headgroup Lipids: A New Class of Lipids With Enhanced Bilayer Destabilizing Characteristics," by Alex Leung, Graduate Student at UBC; "Identification of Small Molecules that Enhance Intracellular Delivery and Function of siRNA Presented in LN in vitro," by Paulo Lin, Postdoctoral Fellow at UBC; and "Identification of Small Molecules that Enhance Uptake of Liposomal Nanoparticles in Target Cells," by Chris Tam, Research Associate at UBC.

"The significant progress we are making in the delivery of RNAi therapeutics is certainly clear as demonstrated by the presentations and posters from Alnylam and collaboration scientists," said Laurence Reid, Ph.D., Senior Vice President and Chief Business Officer at Alnylam. "Importantly, Alnylam's exclusive relationships with key research efforts at MIT, UBC, and AlCana enable discovery of new delivery technologies - including intellectual property and know-how - that expand our overall RNAi therapeutics platform. Of course, advances in our RNAi platform strengthen the value we deliver to our existing partners and also form the foundation for Alnylam's future partnerships."

SOURCE Alnylam Pharmaceuticals