Alnylam Pharmaceuticals’ new research on expanded delivery of RNAi Therapeutics showed effective silencing of target genes in distinct cell types and tissues beyond the liver with systemic delivery of RNAi therapeutics.
The pre-clinical research was presented at the 8th International M Judah Folkman Conference in Cambridge, Massachusetts, highlighting significant advances in the systemic delivery of RNAi therapeutics.
In the study, the RNAi-mediated target gene silencing was observed in endothelial cells across a broad range of tissues, with duration of action lasting for over two months after a single dose.
Additionally, the new work also showed that siRNAs designed toward Tie2 – an endothelium-specific receptor-like tyrosine kinase – formulated in a novel LNP achieved dose dependent silencing of Tie2 mRNA.
The in-vivo efficacy was observed in a range of tissues including liver, heart, intestine, spleen and skeletal muscle, reflecting silencing of the Tie2 mRNA in the vascular endothelium of these tissues.
Tie2 mRNA silencing was also found to be durable, lasting more than two months after a single dose administration.
Alnylam Pharma research senior director said that their key achievements over the past 12 months is the major progress they and their collaborators have made in advancing systemic delivery of RNAi therapeutics.
“In this regard, we have already shown markedly improved potency of RNAi therapeutics, with in vivo-gene silencing at single microgram per kilogram doses, in addition to broadened activity that includes tumors and immune cells,” Fitzgerald said.