Lipid composition for the delivery of therapeutic cargos

DWPI Title: Construct for in vitro gene editing, comprises core comprising external surface and set of pores, cargo arranged in pores and clustered regularly interspaced short palindromic repeats-associated protein 9 component, or nucleic acid sequence encoding component, and coating coupled to core
Abstract: A construct includes a core comprising an external surface and a plurality of pores, a cargo disposed in a pore of the plurality of pores, the cargo comprising a CRISPR Cas9 component or a nucleic acid sequence encoding a CRISPR Cas9 component; and a coating coupled to the core, wherein the coating comprises a cationic lipid, a pegylated lipid, a zwitterionic lipid, and a sterol. The coating may comprise a molar ratio of about 1 cationic lipid to 1 zwitterionic lipid to 0.9 sterol to 0.15 PEGylated lipid, wherein each molar ratio optionally varies by about plus or minus 10%. A method of treatment is also provided.
Use: Construct for in vitro gene editing.
Advantage: The construct demonstrates a tailored coating layer resulting in an improved and efficient delivery, improves colloidal stability and subsequent circulation time with biocompatibility and lower cytoxicity, and is biocompatible and non-toxic.
Novelty: Construct comprises a core comprising an external surface and a set of pores. An average dimension of the set of pores is greater than 2 nm. A cargo is arranged in a pore of the set of pores. The cargo comprises a clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9) component, or a nucleic acid sequence encoding a CRISPR Cas9 component, and a coating coupled to the core. The coating comprises a cationic lipid, a pegylated lipid, a zwitterionic lipid, and a sterol.
Filed: 4/3/2020
Application Number: US16839194A
Tech ID: SD 15013.0
This invention was made with Government support under Contract No. DE-NA0003525 awarded by the United States Department of Energy/National Nuclear Security Administration. The Government has certain rights in the invention.
Data from Derwent World Patents Index, provided by Clarivate
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