Nanomaterials comprising triols
AssigneeBeam Therapeutics Inc.
InventorsCory Dane Sago, Gregory Lawrence Hamilton, Neeraj Narendra Patwardhan
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Abstract
The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use.
Claims24
A method administering a therapeutic and/or prophylactic agent to a subject, the method comprising administering a lipid nanoparticle (LNP) preparation to the subject,
- wherein the LNP preparation comprises:
- a therapeutic and/or prophylactic agent comprising one or more nucleic acids;
- an ionizable lipid, or a pharmaceutically acceptable salt thereof;
- a phospholipid;
- and
- a cholesterol;
- and
- wherein the ionizable lipid is a compound of Formula IX-B-i:
- or a pharmaceutically acceptable salt thereof, wherein:
- each L2 and L2′ is independently a bivalent saturated, straight, or branched C1-12 hydrocarbon chain;
- R1 is optionally substituted C1-20 aliphatic;
- each R1′ is independently optionally substituted C1-20 aliphatic;
- Y2 is a bivalent saturated, straight, or branched C1-6 hydrocarbon chain;
- Y3 is optionally substituted C1-20 aliphatic;
- and
- —X2—X3 is
- and
- wherein the one or more nucleic acids is or comprises an mRNA.
A method of producing a polypeptide of interest in a mammalian cell, the method comprising contacting the cell with a lipid nanoparticle (LNP) preparation to a subject,
- wherein the LNP preparation comprises:
- a therapeutic and/or prophylactic agent comprising one or more nucleic acids;
- an ionizable lipid, or a pharmaceutically acceptable salt thereof;
- a phospholipid;
- and
- a cholesterol;
- and
- wherein the ionizable lipid is a compound of Formula IX-B-i:
- or a pharmaceutically acceptable salt thereof, wherein:
- each L2 and L2′ is independently a bivalent saturated, straight, or branched C1-12 hydrocarbon chain;
- R1 is optionally substituted C1-20 aliphatic;
- each R1′ is independently optionally substituted C1-20 aliphatic;
- Y2 is a bivalent saturated, straight, or branched C1-6 hydrocarbon chain;
- Y3 is optionally substituted C1-20 aliphatic;
- and
- —X2—X3 is
- and
- wherein the one or more nucleic acids is or comprises an mRNA, and wherein the mRNA encodes the polypeptide of interest, whereby the mRNA is capable of being translated in the cell to produce the polypeptide of interest.
A method of base editing a genome of a cell in a subject, the method comprising administering to the subject a composition comprising a lipid nanoparticle (LNP) preparation,
- wherein the LNP preparation comprises:
- a therapeutic and/or prophylactic agent comprising one or more nucleic acids;
- an ionizable lipid, or a pharmaceutically acceptable salt thereof;
- a phospholipid;
- and
- a cholesterol;
- and
- wherein the ionizable lipid is a compound of Formula IX-B-i:
- or a pharmaceutically acceptable salt thereof, wherein:
- each L2 and L2′ is independently a bivalent saturated, straight, or branched C1-12 hydrocarbon chain;
- R1 is optionally substituted C1-20 aliphatic;
- each R1′ is independently optionally substituted C1-20 aliphatic;
- Y2 is a bivalent saturated, straight, or branched C1-6 hydrocarbon chain;
- Y3 is optionally substituted C1-20 aliphatic;
- and
- —X2—X3 is
- and
- wherein the one or more nucleic acids comprises an mRNA encoding a base editor and a chemically modified sgRNA.
Dependencies are inferred from the claim text.