Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY), a
leading RNAi therapeutics company, and collaborators announced today the
publication of a scientific paper documenting the discovery of novel
lipids used in second generation lipid nanoparticles (LNPs) for systemic
delivery of RNAi therapeutics. These results were published in a paper
titled "Maximizing
the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In
Vivo" (DOI: 10.1002/anie.201203263) in the journal Angewandte
Chemie International Edition. The findings describe the rational
design, synthesis, and evaluation of novel lipids, including MC3, that
have led to the discovery of Alnylam's proprietary second generation LNP
platform for systemic delivery of RNAi therapeutics. This research was
conducted in collaboration with scientists from AlCana Technologies,
Inc. and the University of British Columbia (UBC).
"This new paper documents the work performed by Alnylam, AlCana, and UBC
scientists leading to the discovery of novel lipids, including MC3, that
demonstrate remarkably improved in vivo potency for systemic
delivery of RNAi therapeutics. In addition, our results have led to the
identification of a key structural feature of novel ionizable lipids,
specifically the pKa of the amino group of the lipid, required for
optimal in vivo efficacy," said Muthiah Manoharan, Ph.D., Senior
Vice President, Drug Discovery. "The MC3 lipid and resulting second
generation LNPs have now advanced into clinical stages of evaluation.
These novel LNPs exhibit significant improvements in potency and
broadened therapeutic index for RNAi therapeutics, as evidenced by our
recent human data with ALN-PCS, and also define Alnylam's LNP platform
for advancement of ALN-TTR02 and potentially other 'Alnylam 5x15'
programs."