Patent classifications
C07F9/106
Endosomolytic Agents for Gene Therapy
Compounds of formula (I), wherein Ar is an aryl group optionally further substituted with one or more groups R.sup.3; A is a lipophilic, hydrophobic moiety; R.sup.1 is a phosphodiester, phosphotriester, thioether or amide group; X is an unsubstituted or substituted C.sub.6 to C.sub.24 alkylene or alkenylene group, which is optionally interrupted by one or more NR.sup.9, O or S linkages, R.sup.2 is YC(R.sup.4)(R.sup.5)CO.sub.2R.sup.6; and pharmaceutically acceptable salts or solvates thereof are useful as endosomolytic agents particularly for the delivery of nucleic acids useful in gene therapy.
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Compositions for Transfecting Resistant Cell Types
A transfection reagent composition comprising: 30-60 MOL. % of a cationic lipid, or a pharmaceutical acceptable salt thereof; 10-60 MOL % of a structural lipid; 10-20 MOL % of a triglyceride; and 0.1 to about 10 MOL. % of a stabilizing agent, is provided. The transfection agent is effective in transfecting cells, particularly neurons, with siRNA, mRNA and plasmid nucleic acid, Sand maintaining viability of the cells as well as activity of the delivered nucleic acid.
METHODS FOR MAKING ULTRASOUND CONTRAST AGENTS
Provided herein are improved methods for preparing phospholipid formulations including phospholipid UCA formulations.
PROCESS FOR THE PURIFICATION OF L-ALPHA-GLYCEROPHOSPHORYLCHOLINE
A process for the purification of L--glycerophosphorylcholine is described, wherein L--glycerophosphorylcholine is crystallized from DMSO or from a mixture of DMSO with at least another solvent, preferably selected from water, alcohol, halogenated solvents, ethers, esters and/or amides. Such a process allows to obtain L--glycerophosphorylcholine having a purity greater than 99.5%, preferably greater than 99.7%, even more preferably greater than or equal to 99.9%. A method for determining the purity of L--glycerophosphorylcholine is also described, comprising the elution of L--glycerophosphorylcholine through an HPLC column having an amino stationary phase, and subsequent detection of L--glycerophosphorylcholine itself, and any impurity thereof, by means of an Evaporative Light Scattering Detector type.
CATIONIC SULFONAMIDE AMINO LIPIDS AND AMPHIPHILIC ZWITTERIONIC AMINO LIPIDS
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:
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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.
METHODS FOR MAKING ULTRASOUND CONTRAST AGENTS
Provided herein are improve methods for preparing phospholipid formulations including phospholipid UCA formulations.
RED-SHIFTED PHOTOLIPIDS AND NANOPARTICLES FORMED FROM SAME
Provided are photoswitchable glycerophospholipids having a photoswitchable group having the following structure: or The photoswitchable glycerophospholipids can be isomerized by red spectral light. Also disclosed are nanovesicles (e.g., liposomes). Also disclosed are methods of making the photoswitchable glycerophospholipids and nanovesicles formed therefrom. The present disclosure further provides methods of using the nanovesicles. e.g., for targeted delivery of cargo. A photoswitchable glycerophospholipid may have the following structure: Structure I.
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Siloxane-containing phospholipids, compositions and uses thereof
The present application relates to siloxane-containing phospholipids such as the compounds of Formula I, methods of preparation, compositions and uses thereof. ##STR00001##
STABLE NANOLIPOPROTEIN PARTICLES AND RELATED COMPOSITIONS METHODS AND SYSTEMS
Nanolipoprotein particles having at least a scaffold protein component and a membrane lipid component and related compositions, methods and systems are described. The membrane lipid component includes at least one or more membrane forming lipids, one or more polymerized lipids and/or one or more polymerizable lipids.
METHODS FOR MAKING ULTRASOUND CONTRAST AGENTS
Provided herein are improved methods for preparing phospholipid formulations including phospholipid UCA formulations.