Abstract: Described herein are novel lipid compositions comprising unsaturated dendrimers and methods of synthesis of unsaturated dendrimers. The lipid composition can comprise an ionizable cationic lipid, a phospholipid, and a selective organ targeting lipid. Also described herein are pharmaceutical formulations comprising an unsaturated dendrimer, a lipid composition, and a therapeutic agent. Further described in here are methods of mRNA delivery comprising a lipid composition and a therapeutic agent. Further described herein are high-potency dosage forms of a therapeutic formulated with a lipid composition.

Abstract: Modular dendrimers with cationic groups and lipophilic groups are provided herein. In some aspects, the dendrimers provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.

Abstract: Modular dendrimers with cationic groups and lipophilic groups are provided herein. In some aspects, the dendrimers provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.

Abstract: Modular dendrimers with cationic groups and lipophilic groups are provided herein. In some aspects, the dendrimers provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.

Abstract: Modular dendrimers with cationic groups and lipophilic groups are provided herein. In some aspects, the dendrimers provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.

Abstract: Modular dendrimers with cationic groups and lipophilic groups are provided herein. In some aspects, the dendrimers provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.

Abstract: Modular dendrimers with cationic groups and lipophilic groups are provided herein. In some aspects, the dendrimers provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.

Abstract: Disclosed herein are methods for modulating an amount or activity of a gene or a gene product in a cell. The methods herein may comprise contacting a cell with a therapeutic agent assembled with a lipid composition, which lipid composition may comprise a dendrimer or dendron which may comprise one or more degradable diacyl group, in which may result in modulating the amount or activity of the gene or the gene product in the cell. The therapeutic agent modulating a gene or gene product in a cell may be sufficient to treat a disease or disorder in a subject. Further disclosed herein are pharmaceutical compositions, kits, and lipid compositions for modulating an amount or activity of a gene or a gene product in a cell.

Abstract: Ionizable phospholipids and compositions and methods relating thereof are provided herein. In some aspects, the ionizable phospholipids provided herein may be formulated in compositions which contain a nucleic acid and one or more helper excipients. In some aspects, these compositions may also be used to treat diseases or disorders with a therapeutic nucleic acid.

Abstract: The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.

Abstract: The present disclosure provides one or more amino lipids such as an amino lipids containing a sulfonic acid or sulfonic acid derivative of the formulas: wherein the variables are as defined herein. These amino lipids may be used in compositions with one or more helper lipids and a nucleic acid therapeutic agent. These compositions may be used to treat a disease or disorder such as cancer, cystic fibrosis, or other genetic diseases.

Abstract: The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.

Abstract: The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.

Abstract: The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.

Abstract: The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.

Abstract: The present disclosure provides one or more amino lipids such as an amino lipids containing a sulfonic acid or sulfonic acid derivative of the formulas: wherein the variables are as defined herein. These amino lipids may be used in compositions with one or more helper lipids and a nucleic acid therapeutic agent. These compositions may be used to treat a disease or disorder such as cancer, cystic fibrosis, or other genetic diseases.

Abstract: The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.

Abstract: The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.

Abstract: Embodiments of the instant disclosure relate to novel antiviral compositions and methods for treating viral infections. In accordance with these embodiments, antiviral compositions can include at least one mRNA encoding for a TRIM7 protein encapsulated into a lipid nanoparticle (LNP). In other embodiments, methods of making antiviral compositions are disclosed as well as methods of administering a composition having at least one mRNA encoding for a TRIM7 protein encapsulated into LNP into a subject.

Abstract: Disclosed herein are methods for modulating an amount or activity of a gene or a gene product in a cell. The methods herein may comprise contacting a cell with a therapeutic agent assembled with a lipid composition, which lipid composition may comprise a dendrimer or dendron which may comprise one or more degradable diacyl group, in which may result in modulating the amount or activity of the gene or the gene product in the cell. The therapeutic agent modulating a gene or gene product in a cell may be sufficient to treat a disease or disorder in a subject. Further disclosed herein are pharmaceutical compositions, kits, and lipid compositions for modulating an amount or activity of a gene or a gene product in a cell.