LIPID NANOPARTICLES COMPRISING NCULEIC ACIDS, IONIZABLE LIPIDS, STEROLS, LIPID ANCHORED POLYMERS AND HELPER LIPIDS, THEIR USES

• Main Authors: RAGHAV, Jimit, TOY, Randall, SLUBOWSKI, Christian, STANTON, Matthew, GUPTA, Anshul, ZHANG, Sandy, MILSTEAD, Andrew, MARTIN, Constance, GALLAGHER, Nolan, SILVER, Nathaniel
• Format: Patent
• Language: English; French
• Published: 06.09.2024
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; VINEGAR; WINE

Provided herein are lipid nanoparticle (LNP) compositions (e.g., pharmaceutical compositions) comprising a therapeutic nucleic acid (TNA), wherein the LNP comprises an ionizable lipid; a "helper" lipid, e.g., a ceramide or distearoylphosphatidylcholine (DSPC); a structural lipid, e.g., a sterol; and one or more types of lipid-anchored polymers, as well as uses thereof. L'invention concerne des compositions de nanoparticules lipidiques (NPL) (par exemple, des compositions pharmaceutiques) comprenant un acide nucléique thérapeutique (ANT), la NLP comprenant un lipide ionisable ; un lipide "auxiliaire", par exemple, un céramide ou de la distéaroylphosphatidylcholine (DSPC) ; un lipide structural, par exemple, un stérol ; et un ou plusieurs types de polymères à ancrage lipidique, ainsi que leurs utilisations.