Abstract: This invention provides single-stranded and multi-stranded compounds that are useful in various therapeutic modalities to regulate the expression of nucleic acid molecules in a cell. A range of compounds is provided, each containing one or more conformationally restricted nucleomonomers (CRN). In addition, compounds can contain one or more conformationally restricted nucleomonomers and one or more hydroxymethyl substituted nucleomonomers (unlocked nucleomonomers, UNA).

Abstract: Activity-generating delivery molecules comprising the structure R3—(C?O)-Xaa-NH—R4 wherein Xaa is any D- or L-amino acid residue with a non-hydrogen, substituted or unsubstituted side chain, R3—(C?O)— and —NH—R4 are independently a long chain group, each long chain group containing one or more carbon-carbon double bonds, and salts, compositions and methods of use thereof. The activity-generating delivery compounds and compositions are useful for generating activity of an active agent in a cell, tissue, or subject.

Abstract: This invention provides oligomer structures for therapeutic agents, the oligomers composed of one or more UNA nucleomonomers, along with nucleic acid monomers. The UNA oligomers may be used as therapeutic agents for treating or preventing disease. The UNA oligomers can be capable of modulating or silencing gene expression, through RNA interference or other modalities, and may have an antisense strand and a sense strand.

Abstract: This disclosure provides double-stranded RNA complexes having one or more hydroxymethyl substituted nucleomonomer(s) in the passenger strand (or sense strand) of an RNA complex. RNA complexes of the disclosure may be useful for therapeutic applications, diagnostic applications or research applications. RNA complexes include short interfering RNA complexes (siRNA) capable of modulating gene expression comprising an antisense strand and a continuous or a discontinuous passenger strand (“sense strand”). Further, one or more hydroxymethyl substituted nucleomonomer(s) of this disclosure may be positioned at the 3?-end, at the 5?-end, at both the 3?-end and 5?end.

Abstract: This invention provides single-stranded and multi-stranded compounds that are useful in various therapeutic modalities to regulate the expression of nucleic acid molecules in a cell. A range of compounds is provided, each containing one or more conformationally restricted nucleomonomers (CRN). In addition, compounds can contain one or more conformationally restricted nucleomonomers and one or more hydroxymethyl substituted nucleomonomers (unlocked nucleomonomers, UNA).

Abstract: Processes and compositions for liposomal delivery of therapeuticals prepared by contacting an aqueous solution of an active agent with a solution of liposome-forming components containing one or more DILA2 amino acid compounds or lipids in organic solvent to form an impinging stream. A protocol including flow rates, pH, and an incubation period are used to control formation of liposomal components for therapeutic applications. The impinging stream may be collected and incubated to prepare a liposomal formulation which encapsulates the active agent. The composition can be quenched with buffer and filtered by tangential flow and diafiltration and other means for finishing as a pharmaceutical composition.

Abstract: Activity-generating delivery molecules comprising the structure R3—(C?O)-Xaa-NH—R4 wherein Xaa is any D- or L-amino acid residue with a non-hydrogen, substituted or unsubstituted side chain, R3—(C?O)— and —NH—R4 are independently a long chain group, each long chain group containing one or more carbon-carbon double bonds, and salts, compositions and methods of use thereof. The activity-generating delivery compounds and compositions are useful for generating activity of an active agent in a cell, tissue, or subject.

Abstract: This disclosure provides single-stranded and multi-stranded nucleic acid compounds having one or more double-stranded regions that regulate the function or expression of nucleic acid molecules expressed in a cell or a cell regulatory system dependent upon a nucleic acid. The disclosure provides a range of nucleic acid compounds having one or more conformationally restricted nucleomonomers (CRN). Certain nucleic acid compounds may have one or more conformationally restricted nucleomonomers and one or more hydroxymethyl substituted nucleomonomers (UNA). The nucleic acid compounds are useful in various therapeutic modalities.

Abstract: Processes and compositions for liposomal delivery of therapeuticals prepared by contacting an aqueous solution of an active agent with a solution of liposome-forming components containing one or more DILA2 amino acid compounds or lipids in organic solvent to form an impinging stream. A protocol including flow rates, pH, and an incubation period are used to control formation of liposomal components for therapeutic applications. The impinging stream may be collected and incubated to prepare a liposomal formulation which encapsulates the active agent. The composition can be quenched with buffer and filtered by tangential flow and diafiltration and other means for finishing as a pharmaceutical composition.

Abstract: Activity-generating delivery molecules comprising the structure R3—(C?O)-Xaa-NH—R4 wherein Xaa is any D- or L-amino acid residue with a non-hydrogen, substituted or unsubstituted side chain, R3—(C?O)— and —NH—R4 are independently a long chain group, each long chain group containing one or more carbon-carbon double bonds, and salts, compositions and methods of use thereof. The activity-generating delivery compounds and compositions are useful for generating activity of an active agent in a cell, tissue, or subject.

Abstract: This disclosure provides double-stranded RNA complexes having one or more hydroxymethyl substituted nucleomonomer(s) in the passenger strand (or sense strand) of an RNA complex. RNA complexes of the disclosure may be useful for therapeutic applications, diagnostic applications or research applications. RNA complexes include short interfering RNA complexes (siRNA) capable of modulating gene expression comprising an antisense strand and a continuous or a discontinuous passenger strand (“sense strand”). Further, one or more hydroxymethyl substituted nucleomonomer(s) of this disclosure may be positioned at the 3?-end, at the 5?-end, at both the 3?-end and 5?end.

Abstract: The present invention relates to compositions and methods which enhance the delivery of oligonucleotides and other nucleosidic moieties via topical routes of administration. Preferred compositions include liposomes or penetration enhancers for the delivery of such moieties to dermal and/or epidermal tissue in an animal for investigative, therapeutic or prophylactic purposes.

Abstract: The present invention relates to compositions and methods which enhance the delivery of oligonucleotides and other nucleosidic moieties via topical routes of administration. Preferred compositions include liposomes or penetration enhancers for the delivery of such moieties to dermal and/or epidermal tissue in an animal for investigative, therapeutic or prophylactic purposes.

Abstract: Processes and compositions for liposomal delivery of therapeuticals prepared by contacting an aqueous solution of an active agent with a solution of liposome-forming components containing one or more DILA2 amino acid compounds or lipids in organic solvent to form an impinging stream. A protocol including flow rates, pH, and an incubation period are used to control formation of liposomal components for therapeutic applications. The impinging stream may be collected and incubated to prepare a liposomal formulation which encapsulates the active agent. The composition can be quenched with buffer and filtered by tangential flow and diafiltration and other means for finishing as a pharmaceutical composition. An efficiency for delivering a drug cargo is provided.

Abstract: The present invention relates to compositions and methods which enhance the delivery of oligonucleotides and other nucleosidic moieties via topical routes of administration. Preferred compositions include liposomes or penetration enhancers for the delivery of such moieties to dermal and/or epidermal tissue in an animal for investigative, therapeutic or prophylactic purposes.

Abstract: Compounds and components including sequences for mucosal epithelial transport of an active agent are given. Tight junction modulating peptide components are described for use in transport and delivery. Permeability can be enhanced with reversibility. Compounds and components for enhanced delivery may be peptide or protein variants, conjugates, or other analog types and structures.

Abstract: Methods and uses of RNAi-inducing agents for medicaments and treating or preventing a viral infection.

Abstract: What is described is a pharmaceutical formulation comprising a mixture of a pharmaceutically effective amount of glucose-regulating peptide (GRP) and enhancers, wherein the pharmaceutical formulation is used in the treatment of a metabolic syndrome.

Abstract: A method for modulating hematopoietic stem cells and treating a hematologic disease in a mammal comprising administering intranasally a therapeutically effective amount of a PTH formulation. The PTH formulation may contain teriparatide.

Abstract: The present invention relates to compositions and methods which enhance the delivery of oligonucleotides and other nucleosidic moieties via topical routes of administration. Preferred compositions include liposomes or penetration enhancers for the delivery of such moieties to dermal and/or epidermal tissue in an animal for investigative, therapeutic or prophylactic purposes.