alexa Rational design of cationic lipids for siRNA delivery
Materials Science

Materials Science

Journal of Nanomedicine & Biotherapeutic Discovery

Author(s): Semple SC, Akinc A, Chen J, Sandhu AP, Mui BLL

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We adopted a rational approach to design cationic lipids for use in formulations to deliver small interfering RNA (siRNA). Starting with the ionizable cationic lipid 1,2-dilinoleyloxy-3-dimethylaminopropane (DLinDMA), a key lipid component of stable nucleic acid lipid particles (SNALP) as a benchmark, we used the proposed in vivo mechanism of action of ionizable cationic lipids to guide the design of DLinDMA-based lipids with superior delivery capacity. The best-performing lipid recovered after screening (DLin-KC2-DMA) was formulated and characterized in SNALP and demonstrated to have in vivo activity at siRNA doses as low as 0.01 mg/kg in rodents and 0.1 mg/kg in nonhuman primates. To our knowledge, this represents a substantial improvement over previous reports of in vivo endogenous hepatic gene silencing.

This article was published in Nat Biotechnol and referenced in Journal of Nanomedicine & Biotherapeutic Discovery

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