A61K9/1271

Biodegradable compound, lipid particle, composition comprising lipid particle, and kit

The present embodiment provides a compound represented by the formula (1):
Q-CHR.sub.2  (1)
(Q is a nitrogen-containing aliphatic group containing two or more tertiary nitrogens but no oxygen, and R is an aliphatic group containing a biodegradable group). From the compound in combination with other lipids such as a lipid capable of reducing aggregation, lipid particles can be formed. Further, the compound can be used for a pharmaceutical composition to deliver an activator into cells.

NUCLEIC ACID EXPRESSION USING SUBCUTANEOUS ADMINISTRATION

Provided herein are compositions, systems, kits, and methods for treating a subject, and/or a subject's pre-adipocytes and/or adipocytes, with a composition containing a nucleic acid sequence encoding a protein or other biologically active nucleic acid-encoded molecule (BANEM), or a vector containing the nucleic acid sequence, wherein the treating comprises: a) injecting the composition into one or more subcutaneous (SC) regions of the subject such that one or more protein, or other BANEM, is detectable in a blood, serum, or plasma sample from the subject; and/or b) injecting the composition into one or more SC regions of the subject such that in-vivo transfected pre-adipocytes and/or adipocytes (e.g., transfected cells of fat cell origin) are generated; and/or c) performing the following: i) contacting pre-adipocytes and/or adipocytes (e.g., cells of fat cell origin) from the subject ex-vivo with the composition such that ex-vivo transfected pre-adipocytes and/or adipocytes are generated, and ii) injecting the ex-vivo transfected pre-adipocytes and/or adipocytes into one or more SC regions of the subject.

Lipids for use in lipid nanoparticle formulations
11542225 · 2023-01-03 · ·

Compounds are provided having the following structure (I) or a pharmaceutically acceptable salt, tautomer or stereoisomer thereof, wherein G.sup.1, G.sup.1′, G.sup.2, G.sup.2′, G.sup.3, L.sup.1, L.sup.1′, L.sup.2, L.sup.2′, X, X′, Y and Y′ are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided. ##STR00001##

Comprehensive vaccine design for commensal disease progression

Provided are vaccine compositions and methods against Streptococcus pneumoniae. The composition comprises liposomes which have polysaccharides from one or more serotypes and have proteins non-covalently attached to the surface and exposed to the exterior.

VARICELLA ZOSTER VIRUS (VZV) VACCINE
20220409720 · 2022-12-29 · ·

Aspects of the disclosure relate to nucleic acid vaccines. The vaccines include at least one RNA polynucleotides having a open reading reading frame encoding at least varicella zoster virus (VZV) antigen. Methods for preparing and using such vaccines are also described.

CAROTENOID COMPOSITIONS AND USES THEREOF

Provided herein are pharmaceutical compositions comprising carotenoids, including liposomes that encapsulate carotenoids including ionizable carotenoids such as trans-crocetin. The provided compositions have uses in treating diseases, disorders and conditions associated with, but not limited to, infection, endotoxemia, inflammation, sepsis, ischemia, hypoxia, shock, stroke, lung injury, wound healing, traumatic injury, reperfusion injury, cardiovascular disease, kidney disease, liver disease, inflammatory disease, metabolic disease, pulmonary disorders, blood related disorders and hyperproliferative diseases such as cancer. Methods of making, delivering, and using the pharmaceutical compositions are also provided.

NANODROPLET WITH LAYER-BY-LAYER ASSEMBLY
20220409750 · 2022-12-29 ·

The present disclosure provides novel modified nanodroplets with a layerby-layer (LBL) assembly formulation (“LBLnNDs”). The LBLnNDs of the present disclosure comprise gaseous perfluorocarbon core, polymer shell, and multiple alternating positively and negatively charged biopolymer layers dispersed layerby-layer onto the shell of the LBLnNDs. Methods of making and use of the LBLnNDs are also provided.

COMPOSITIONS AND METHODS FOR USE IN TYPE 1 DIABETES
20220409702 · 2022-12-29 ·

Provided are methods for preventing, treating, suppressing type 1 diabetes, or reversing one or more symptoms of T1D, or delaying the onset of T1D comprising administration to a subject a composition comprising liposomes and insulin intercalated in the bilayer of the liposomes, wherein the bilayer comprises lyso-PS.

Polyglutamated antifolates and uses thereof

The disclosure relates generally to polyglutamated antifolates, formulations containing liposomes filled with the polyglutamated antifolates, methods of making the polyglutamated antifolates and liposome containing formulations, and methods of using the polyglutamated antifolates and liposome containing formulations to treat hyperproliferative disorders (e.g., cancer) and disorders of the immune system (e.g., an autoimmune disease such as rheumatoid arthritis).

IONIZABLE AMINE LIPIDS AND LIPID NANOPARTICLES

The disclosure provides ionizable amine lipids and salts thereof (e.g., pharmaceutically acceptable salts thereof) useful for the delivery of biologically active agents, for example delivering biologically active agents to cells to prepare engineered cells. The ionizable amine lipids disclosed herein are useful as ionizable lipids in the formulation of lipid nanoparticle-based compositions.