nor-MC3 and nor-KC2: cationic ionizable lipids for the delivery of therapeutic nucleic acids

Deaglan Arnold; Nagavenkata Durga Prasad Atmuri; Fariba Saadati; Ardalan Nabi; Daniel Z. Kurek; Anthony Tam; Taniya Adak; Dominik Witzigmann; Glenn Sammis; Pieter R. Cullis; Jayesh Kulkarni; Marco A. Ciufolini

doi:10.1039/D5PM00350D

nor-MC3 and nor-KC2: cationic ionizable lipids for the delivery of therapeutic nucleic acids

Deaglan Arnold,^a Nagavenkata Durga Prasad Atmuri,^a Fariba Saadati,^a Ardalan Nabi,^a Daniel Z. Kurek,^a Anthony Tam,^a Taniya Adak,^b Dominik Witzigmann,^a Glenn Sammis,

^b Pieter R. Cullis,

^ac Jayesh Kulkarni*^a and Marco A. Ciufolini

*^a

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* Corresponding authors

^a NanoVation Therapeutics, Inc., 2665 East Mall 2nd floor, Vancouver, BC V6T 1Z4, Canada
E-mail: j.kulkarni@nanovationtx.com, m.ciufolini@nanovationtx.com

^b Department of Chemistry, University of British Columbia, 2036 Main Mall, Vancouver, BC V6T 1Z1, Canada

^c Department of Biochemistry and Molecular Biology, University of British Columbia, Life Sciences Center, 2350 Health Sciences Mall, Vancouver, BC V6T 1Z3, Canada

Abstract

nor-MC3 and nor-KC2, analogues of D-Lin-MC3-DMA (cationic ionizable lipid in Onpattro®) and D-Lin-KC2-DMA (valuable research tool) wherein C₁₇ lipophilic chains replace C₁₈ ones, are at least as efficacious as the originals, but more economical and safer to produce.

RSC Pharmaceutics

nor-MC3 and nor-KC2: cationic ionizable lipids for the delivery of therapeutic nucleic acids

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nor-MC3 and nor-KC2: cationic ionizable lipids for the delivery of therapeutic nucleic acids

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