Abstract: This invention includes ionizable compounds, and compositions and methods of use thereof. The ionizable compounds can be used for making nanoparticle compositions for use in biopharmaceuticals and therapeutics. More particularly, this invention relates to compounds, compositions and methods for providing nanoparticles to encapsulate active agents, such as nucleic acid agents, and to deliver and distribute the active agents to cells, tissues, organs, and subjects.

Abstract: This invention provides methods and compositions for inhibiting Ebola virus and Ebola infection utilizing gene silencing siRNAs. The siRNAs can be targeted to one or more Ebola genes including NP, VP30, VP35, L polymerase, glycoprotein (GP), VP24, VP40, and intergenic overlapping regions selected from NP/VP35, VP35/VP40, GP/VP30, and VP24/L. A composition containing the siRNAs can be administered in a delivery vehicle such as liposomes.

Abstract: This invention provides methods and compositions for preventing, treating or ameliorating one or more symptoms of a malignant tumor, which may be associated with KRAS mutation in a mammal in need thereof, by administering to the mammal a therapeutically effective amount of a composition comprising one or more RNAi molecules that are active in reducing expression of GST-?.

Abstract: This invention provides compositions for use in distributing active agents for treating a malignant tumor in a subject. The compositions contain RNAi molecules targeted to a human GST-?, along with RNAi molecules targeted to a human p21, and a pharmaceutically acceptable carrier. The carrier can include nanoparticles composed of an ionizable lipid, a structural lipid, one or more stabilizer lipids, and a lipid for reducing immunogenicity of the nanoparticles. This invention further provides methods for preventing or treating a malignant tumor by administering a therapeutically effective amount of an RNAi composition.

Abstract: This invention provides methods and compositions for preventing, treating or ameliorating one or more symptoms of a malignant tumor associated with KRAS mutation in a mammal in need thereof, by identifying a tumor cell in the mammal, the tumor cell comprising at least one of: (i) a mutation of the KRAS gene, and (ii) an aberrant expression level of KRAS protein; and administering to the mammal a therapeutically effective amount of a composition comprising one or more RNAi molecules that are active in reducing expression of GST-?.

Abstract: This invention provides methods and compositions for preventing, treating or ameliorating one or more symptoms of a malignant tumor associated with KRAS mutation in a mammal in need thereof, by identifying a tumor cell in the mammal, the tumor cell comprising at least one of: (i) a mutation of the KRAS gene, and (ii) an aberrant expression level of KRAS protein; and administering to the mammal a therapeutically effective amount of a composition comprising one or more RNAi molecules that are active in reducing expression of GST-?.

Abstract: This invention provides compositions for making a solid lyophile of one or more nucleic acid active agents, which can be reconstituted as a drug product. The composition can include an aqueous suspension of lipid nanoparticles in a pharmaceutically acceptable solution, wherein the lipid nanoparticles encapsulate one or more nucleic acid active agents, a dextrin compound, and a saccharide compound. The nucleic acid active agents can be RNAi molecules capable of mediating RNA interference, as well as other RNAs and oligonucleotides.

Abstract: This invention includes ionizable compounds, and compositions and methods of use thereof. The ionizable compounds can be used for making nanoparticle compositions for use in biopharmaceuticals and therapeutics. More particularly, this invention relates to compounds, compositions and methods for providing nanoparticles to encapsulate active agents, such as nucleic acid agents, and to deliver and distribute the active agents to cells, tissues, organs, and subjects.

Abstract: This invention provides methods and compositions for preventing, treating or ameliorating one or more symptoms of a malignant tumor associated with KRAS mutation in a mammal in need thereof, by identifying a tumor cell in the mammal, the tumor cell comprising at least one of: (i) a mutation of the KRAS gene, and (ii) an aberrant expression level of KRAS protein; and administering to the mammal a therapeutically effective amount of a composition comprising one or more RNAi molecules that are active in reducing expression of GST-?.

Abstract: This invention provides compositions for use in distributing active agents for treating a malignant tumor in a subject. The compositions contain RNAi molecules targeted to a human GST-?, along with RNAi molecules targeted to a human p21, and a pharmaceutically acceptable carrier. The carrier can include nanoparticles composed of an ionizable lipid, a structural lipid, one or more stabilizer lipids, and a lipid for reducing immunogenicity of the nanoparticles. This invention further provides methods for preventing or treating a malignant tumor by administering a therapeutically effective amount of an RNAi composition.

Abstract: Pharmaceutical compositions and methods of use of a composition containing a double-stranded RNA (dsRNA), cationic lipids, non-cationic lipids, and lipophilic delivery-enhancing compounds.

Abstract: A complex of a double stranded (ds) ribonucleic acid and a peptide produced by a method comprising dissolving the nucleic acid in an aqueous solution, dissolving the peptide in an aqueous solution, mixing the solubilized ds nucleic acid and the solubilized peptide, and treating the mixture by freezing and thawing, heating and cooling, or salting and desalting.

Abstract: The present invention is directed to pharmaceutical compositions containing a therapeutically effective amount of an Active Pharmaceutical Ingredient (“API”), a pharmaceutically acceptable cyclodextrin and a pharmaceutically acceptable preservative. The invention is also directed to pharmaceutical compositions of the compounds of Formula (I) wherein R2 is selected from the group consisting of methyl, ethyl, isopropyl, sec-butyl and tert-butyl and a pharmaceutically acceptable cyclodextrin and preservative. Formula (I): In particular, the invention is directed to pharmaceutical compositions of the compound of Formula 1a, and a pharmaceutically acceptable cyclodextrin and a preservative.