Resumen de: EP3485913A1

Provided are cationic lipids that can be used in combination with other lipid components, such as cholesterol and PEG-lipids, to form lipid nanoparticles with oligonucleotides,in particular siRNA. Such cationic lipids have low molecular weight and compriseg at least one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA. The cationic lipid scaffold which they fom has enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver.

Resumen de: WO2018013783A1

The disclosure provides compounds of Formula (I), wherein X, Y, and Z are defined herein. The disclosure also provides particles comprising one or more compounds described herein, compositions comprising one or more compounds or particles described herein and a pharmaceutically acceptable carrier, and methods of treating a subject in need thereof comprising administering one or more compounds, particles, or compositions described herein to the subject. X— Y— Z (I).

Resumen de: EP3485875A1

What is described is a process of forming lipid-nucleic acid nanoparticles simply and reproducibly under aseptic conditions comprising single use components.

Resumen de: US9833505B1

The present disclosure provides compositions and methods useful for preventing and treating Zika virus infection. As described herein, the compositions and methods are based on development of immunogenic compositions that include virus-like particles (VLPs) which comprise one or more Moloney Murine leukemia virus (MMLV) core proteins and include one or more Zika epitopes, such as, for example, from Zika envelope glycoprotein E and the Zika structural protein NS1 including variants thereof.

Resumen de: AU2019202835A1

The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.

Resumen de: AU2017342558A1

The disclosure provides methods of treating immunological conditions,

Resumen de: AU2017357758A1

The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a solution of pre-formed lipid nanoparticles and mRNA.

Resumen de: WO2019094868A1

This invention is directed to a biodegradable polymer that can be degraded in vivo. The biodegradable polymer comprises a biodegradable polymer segment having at least a biodegradable bond and two or more cationic components, wherein each of said cationic components is covalently attached to the biodegradable polymer segment and the two cationic components/molecules are separated by at least one biodegradable bond in the backbone. The biodegradable polymer can be used for targeting desired ceils in vivo including T cells, NK (natural killer) ceils, cancer cells, or a combination thereof, delivering genes, DNA, oligodeoxynucleotide, oligonucleotide, RNA, mRNA, RNAi, siRNA, microRNA, protein, peptide, antibody, fragment of an antibody, small molecule drug including chemotherapy drugs, or other bioactive agents into cells, or being used as a vaccine or drug for treating a disease such as a cancer in a subject.

Resumen de: WO2019094906A1

The present disclosure is directed to a nanotechnology-enabled screening strategy to identify small molecule TFEB agonists that shift maturation of autophagosomes to degradative autolysosomes.

Resumen de: WO2019094405A1

Disclosed herein are nanoparticles comprising a lipid core comprising a sterol; and a complex comprising a cationic agent and a therapeutic agent, wherein the complex is encapsulated within the lipid core. Methods to produce the nanoparticle comprise: combining a cationic agent, a therapeutic agent, and a first water-immiscible solvent with a first aqueous solution, thereby forming a mixture comprising a complex comprising the cationic agent and the therapeutic agent; combining the mixture with a second waterimmiscible solvent, thereby forming an aqueous phase and an organic phase, and separating the organic phase comprising the complex; combining the organic phase comprising the complex with a sterol and a first water-miscible organic solvent; and dispersing the complex in a second aqueous solution to form a herein disclosed nanoparticle. Methods for treating a disease and for reducing nanoparticle burst rate are also disclosed.

Resumen de: WO2019094625A1

The present disclosure provides pharmaceutical compositions, parenteral delivery methods and methods for the manufacture of a cannabidiol drug delivery system. The system comprises pharmaceutical compositions having therapeutically active amounts of cannabidiol in a nanoparticle formulation for delivery of an API to a mammal, preferably a human in the treatment of seizures and epilepsy. Also described herein are devices for their delivery such as an auto-injector device for rapid and safe delivery of cannabidiol as an auto-injector rescue. Other delivery devices are described for in a long-term treatment regimen for epilepsy. The platform has clinical applications in the treatment of subjects suffering from epilepsy or epileptic-induced seizures.

Resumen de: WO2019094769A1

Methods for reducing at least one of the bioavailability and absorption of at least one of ingested fat, carbohydrates, and unwanted substances from foodstuff in a subject are disclosed. The methods described herein include administering an amount of a nanobiopolymer, or combinations of nanobiopolymers, sufficient to reduce at least one of the bioavailability and absorption of ingested fat, carbohydrates, and unwanted substances to a subject, such as a human subject.

Resumen de: WO2019094669A2

Synthetic nanostructures, polypeptides that are useful, for example, in making synthetic nanostructures, and methods for using synthetic nanostructures are disclosed herein.

Resumen de: WO2019090411A1

The present disclosure relates to methods for preparing a dried preparation comprising lipids and therapeutic agents whereby the therapeutic agents are incorporated both before and after sizing of lipid vesicle particles to a mean particle size of ≤ 120 nm and a polydispersity index (PDI) of ≤0.1. The present application also provides stable, water-free pharmaceutical compositions comprising one or more lipid-based structures having a single layer lipid assembly, at least two therapeutic agents, and a hydrophobic carrier, as well as methods of treatment, uses and kits relating thereto, such as for example for inducing an antibody and/or CTL immune response.

Resumen de: WO2019093199A1

[Problem] The purpose of the present invention is to provide a water-in-oil emulsion composition having exceptional storage stability even without use of a surfactant as well as exceptional safety, the water-in-oil emulsion being useful in cosmetics, dermatological preparations, etc. [Solution] A water-in-oil emulsion composition including (a) 100 parts by mass of an oil-phase component, (b) 11-800 parts by mass of an aqueous-phase component, and (c) 0.5-40 parts by mass of hydrophobic spherical sol-gel silica fine particles having a volume median diameter (D50) of 5-1500 nm.

Resumen de: US2019144492A1

An oxysterol or oxysterol-like compound is provided, which finds use in treating and/or targeting cancer.

Resumen de: US2019142960A1

Described herein are polymeric drug-carrying nanogels that are capable of targeting to P-selectin for the treatment of cancer and other diseases and conditions associated with P-selectin. Furthermore, in certain embodiments, the nanogels presented here offer a drug release mechanism based on acidic pH in the microenvironment of a tumor, thereby providing improved treatment targeting capability and allowing use of lower drug doses, thereby reducing toxicity.

Resumen de: US2019142761A1

The invention provides an agent delivery system comprising an agent preparation encapsulated within a membrane composition. The invention further provides a membrane composition comprising a ganglioside and lipid for encapsulating agents, and processes for preparing the membrane compositions. The invention further provides agent preparations encapsulated within the membrane compositions, including a plurality of individual particles wherein each particle contains agent preparation individually encapsulated within membrane composition. The invention also provides processes for preparing encapsulated agent preparations or individual particles of encapsulated agent preparations. The agents may be biologically active including hydrophilic, hydrophobic, small molecule or large molecule therapeutic agents. The invention further provides pharmaceutical compositions comprising encapsulated agent preparations or individual particles of encapsulated agent preparations. The invention further provides methods for delivery of the encapsulated agent preparations or individual particles of encapsulated agent preparations to a subject, including oral delivery of large molecule therapeutic agents to the lymphatic system, blood circulatory system, and/or targeted delivery to cells, tissues or organs.

Resumen de: US2019142746A1

The present invention provides for compositions comprising a polymeric hydrogel impregnated with a toxin-absorbing or binding nanoparticle. The present invention also provides for the use of the above compositions for decreasing or neutralizing the effect of a toxin, or for treating or preventing an infection by a microbe that produces a toxin, in a subject. The exemplary toxin is a biological toxin such as a viral, bacterial, fungal, plant or animal toxin.

Resumen de: US2019142964A1

Low-generation dendrimers containing a high density of surface hydroxyl groups, and methods of synthesis thereof are provided. In particular, oligo ethylene glycol (OEG)-like dendrimers with a high surface functional groups at relatively low generations (e.g. ̃120 hydroxyls in the third generation, with a size of just 1-2 nm) is described. Dendrimer formulations including one or more prophylactic, therapeutic, and/or diagnostic agents, and methods of use thereof are also described. The formulations are suitable for topical, enteral, and/or parenteral delivery for treating one or more diseases, conditions, and injuries in the eye, the brain and nervous system (CNS), particularly those associated with pathological activation of microglia and astrocytes.

Resumen de: US2019142980A1

The present invention refers to cells from a subpopulation of progenitors of endothelial cells loaded with gold nanoparticles sensitive to excitation with infrared radiation with consequent release of thermal energy, to a composition having a plurality of these cells and to their use in the diagnosis and treatment of solid tumours thanks to the selective localization of the composition in the tumour mass, which can be thus identified easily for diagnostic purposes by marking the cells with appropriate marking agents, or treated by stimulation with pulsed laser beam up to the complete cauterization of the tumour mass for a targeted and highly effective therapeutic action.

Resumen de: US2019142739A1

Aspects of the invention relate to topical and ocular formulations of spherical nucleic acids (SNA), as well as methods of use thereof and compositions thereof. The formulations may include an inhibitor such as an inhibitor of tumour necrosis factor alpha (TNFa), platelet-derived growth factor subunit A (PDGFA), platelet-derived growth factor subunit B (PDGFB), platelet-derived growth factor subunit C (PDGFC), platelet-derived growth factor subunit D (PDGFD), platelet-derived growth factor receptor alpha (PDGFRA), platelet-derived growth factor receptor beta (PDGFRB), platelet-derived growth factor receptor like (PDGFRL), vascular endothelial growth factor A (VEGFA), vascular endothelial growth factor B (VEGFB), vascular endothelial growth factor C (VEGFC) vascular endothelial growth factor D (VEGFD), vascular endothelial growth factor receptor-1 (VEGFR1), vascular endothelial growth factor receptor-2 (VEGFR2), vascular endothelial growth factor receptor-3 (VEGFR3), beta-2 adrenergic receptor (ADRB2), connective tissue growth factor (CTGF), interleukin 1 beta (IL1 β), interleukin 1 receptor-1 (IL1 R1), interleukin 1 receptor-2 (IL1R2), and interleukin 1 receptor-3 (IL1R3). Aspects of the invention further relate to nanostructures comprising self-assembling therapeutic oligonucleotides, such as antisense oligonucleotides, that are linked to a molecular species, wherein the molecular species is positioned in a core of the nanostructure and the oligonucleotides extend radially

Resumen de: US2019142882A1

Methods and compositions for bacterial production of pure single-stranded DNA (ssDNA) composed of custom sequence and size have been developed. The methods enable scalability and bio-orthogonality in applications of scaffolded DNA origami, offering one-step purification of large quantities of pure ssDNA amendable for immediate folding of DNA nanoparticles. The methods produce pure ssDNA directly from bacteria. In some embodiments the E. coli helper strain M13cp combined with a phagemid carrying only an f1 -origin allows for, without the need for additional purification from contaminating dsDNA. This system is useful for generalized circular ssDNA synthesis, and here is applied to the assembly of DNA nanoparticles folded both in vitro and direct from phage.

Resumen de: US2019142790A1

Provided herein is a pharmaceutical composition comprising at least one isoflavonoid. Also provided herein are methods of treating cancer, sensitizing cancer cells, and inducing apoptosis in cancer cells by administering such compositions.

Resumen de: US2019142744A1

Described herein are nucleic acid based nanoparticles that can contain a cargo molecule. The nanoparticles described herein can be used to deliver a cargo molecule to a cell or area of interest.