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The present invention relates to stabilizing an oil in water nanoemulsion in a pressurized container. The nanovesicles comprised in the nanoemulsion are particularly stable in regard to vesicle size and vesicle size homogeneity after long-term storage at different temperatures.
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The present invention relates to oil in water nanoemulsions which are essentially free of propylene glycol. The nanovesicle formulations are particularly stable in regard to shelf life at different storage temperatures.
Resumen de: AU2024252371A1
The present disclosure relates to novel compounds, methods, and cell-targeting formulations, e.g.. a lipid nanoparticle (LNP) for targeted delivery to a tissue or a cell type. The compound and formulation provided herein are designed to have a targeting moiety configured to provide selective delivery features for the formulation and a lipid tail for being incorporated into the bilayer membrane of the formed lipid nanoparticle.
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The present application discloses compositions comprising nanoparticles of vitamin K2, and their methods of use.
Resumen de: AU2024236558A1
The present disclosure describes improved LNP-based nucleobase editing systems and therapeutics for use in treating a disease. In particular, the disclosure describes improved LNPs, including novel and improved ionic lipids for making LNPs, that enhance the targeted delivery of LNP-based nucleobase editing systems and therapeutics based on linear and/or circular mRNAs. The improved LNPs protect linear and/or circular mRNA payloads from degradation and clearance while achieving targeted systemic or local delivery for use as enhanced nucleobase editing systems and/or therapeutic agents.
Resumen de: MX2025009878A
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Resumen de: WO2025212858A1
Disclosed are in vitro transcribed (IVT) messenger RNAs (mRNAs) comprising a polynucleotide that encodes human TJP2 gene, a 5' terminal cap, and a poly-adenylation (poly- A) tail at the 3' terminal end. In aspects, the polynucleotide is substantially free of uridine bases and comprises pseudouridine bases. Methods, compositions and uses employing the IVT mRNA for treating or preventing progressive familial intrahepatic Cholestasis type IV (PFIC type 4) in a patient in need thereof is also disclosed.
Resumen de: WO2025212932A1
Described herein are methods of synthesizing ionizable amine lipids, such as Lipid A: Formula (I) with increased overall purity, process robustness and an applied impurity control strategy, leading to a substantially pure Lipid A product. Intermediate compounds and methods of synthesizing intermediate compounds are also described.
Resumen de: WO2025212530A1
The present disclosure provides nanoparticle compositions in which individual nanoparticles comprise payloads, as well as methods of making and using such nanoparticle compositions, and various compositions and/or technologies relating to such nanoparticle compositions, their production, and/or their use.
Resumen de: WO2025210521A1
The present disclosure provides lipid nanoparticles that are particularly beneficial in inducing an immune response in a subject, particularly by stimulating CD8+ T cells associated with protein or protein fragments of interest. The lipid nanoparticles can have a negative zeta potential and/or particular configurations and/or relative amounts of a phospholipid, an ionizable lipid, and a conjugated lipid. The disclosure also provides vaccines, other pharmaceutical compositions, and methods including the provided lipid nanoparticles.
Resumen de: WO2025213139A1
Provided herein are compositions and methods for gene therapy for disorders of arterial calcification as well as Generalized Arterial Calcification of Infancy (GACI). The methods include a gene addition strategy to deliver a DNA construct to target tissues (such as liver and smooth muscle cells) to express soluble recombinant ENPP1 (srENPP1) or transmembrane full-length recombinant ENPP1 (rENPP1).
Resumen de: EP4628070A1
One aspect according to the present invention relates to: a gold nanozyme including glycol chitosan and gold particles; and a pharmaceutical composition for preventing or treating inflammatory bowel disease, the composition comprising the gold nanozyme. The gold nanozyme and the pharmaceutical composition comprising same according to one aspect of the present invention was found to inhibit the expression of inflammatory factors, inhibit the generation of reactive oxygen species (ROS) and reactive nitrogen species (RNS) inside cells, and reduce the production of nitric oxide (NO) inside cells. In addition, the gold nanozyme was found to inhibit the secretion of Hight Mobility Group Box 1 (HMGB1) when further including glycyrrhizin. In addition, the gold nanozyme and the composition comprising same were found to enable the recovery of the length, weight, etc., of damaged intestines, and thus can be used in the inflammatory bowel disease prevention and/or treatment industry/market.
Resumen de: WO2024118378A1
Compositions and methods are disclosed for treating metabolic syndrome-associated heart disease cardiomyopathy and/or heart failure, wherein the method comprises the step of increasing the concentration of LIPTER RNA in the cardiomyocytes of said patient.
Resumen de: WO2024119103A1
Provided herein are lipid nanoparticles (LNPs) comprising a therapeutic nucleic acid (TNA) and uses thereof. The LNPs comprise an ionizable lipid; a structural lipid, e.g., a sterol; and one or more types of lipid-anchored polymers, and do not comprise a helper lipid.
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The present disclosure provides novel polymer-conjugated lipids, e.g., comprising DODA conjugated to a polyglycerol or a polyglycerol derivative. The present disclosure also provides lipid nanoparticles (LNPs) formulation using the polymer-conjugated lipids and methods of treating a disease by administering the LNP formulations.
Resumen de: AU2023406303A1
Provided herein are lipid nanoparticle (LNP) compositions (e.g., pharmaceutical compositions) comprising a therapeutic nucleic acid (TNA), wherein the LNP comprises an ionizable lipid; a "helper" lipid, e.g., a ceramide or distearoylphosphatidylcholine (DSPC); a structural lipid, e.g., a sterol; and one or more types of lipid-anchored polymers, as well as uses thereof.
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The present disclosure provides stealth lipid nanoparticle (LNP) compositions engineered to target specific tissues or cell-types,
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The present disclosure describes compositions, nanoparticles (such as lipid nanoparticles), and/or lipid nanoparticle compositions and methods of their use.
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This disclosure provides a bispecific canine antigen-binding molecule comprising a first antigen binding domain or antigen-binding portion thereof that specifically binds canine CD3, and a second antigen binding domain or antigen-binding portion thereof that specifically binds canine CD20, compositions comprising the same, and methods of their use. The disclosure also provides a canine antibody or antigen-binding portion thereof that binds canine CD3, compositions comprising the same, and methods of their use. The disclosure also provides a canine antibody or antigen-binding portion thereof that binds canine CD20, compositions comprising the same, and methods of their use.
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Provided herein are pharmaceutical compositions comprising surface functionalized carbon nanotube and an active agent. The active agent can be attached to the carbon nanotube covalently or noncovalently. Also provided are methods of preparing the pharmaceutical compositions and methods of use thereof.
Resumen de: EP4628102A2
The drugs available for the treatment of cutaneous leishmaniasis have unsatisfactory efficacy, frequent and serious adverse effects, and require long treatment regimens. Thus, the search for new treatment alternatives for cutaneous leishmaniasis is considered a priority by the World Health Organization. Parenteral administration of pentavalent antimonials for the treatment of all forms of leishmaniasis, including cutaneous leishmaniasis, has several limitations. The therapy is long, requires repeated doses, and adverse reactions are frequent. Topical treatment is an attractive alternative for cutaneous leishmaniasis, offering significant advantages over systemic therapy: fewer adverse effects, ease of administration, and lower costs. The present inventors aimed to provide a fixed-dose topical composition containing at least one antileishmanial compound, providing adequate absorption of the active ingredient. Another objective of the present invention is to provide a topical, fixed-dose formulation containing a combination of antileishmanial compounds that has sufficient efficacy and safety to be used in the treatment of cutaneous leishmaniasis.
Resumen de: EP4628496A1
A steroid-cationic lipid compound as represented by formula (I). The LNP prepared from the compound can deliver a bioactive substance to a target cell or organ in an effective and stable manner, and the mRNA LNP prepared from the compound has good levels of stability and transfection efficiency, and can trigger a relatively high specific antibody response and cellular immune response in an animal.
Resumen de: EP4628494A1
A phosphatidylamine compound including a plurality of tertiary amino group structures and the composition and use thereof are provided. The phosphatidylamine compound is a phospholipid compound including two or more tertiary amino group structures, the structure of which is represented by the following formula (I), where, the definition of each substituent is detailed in the instructions. The compound works together with other lipid components such as cholesterol, DSPC/DOPE, DMG-PEG2000, and other helper lipids to form lipid nanoparticles (LNPs), which may be used for efficient delivery of drug molecules such as nucleic acids (siRNA, mRNA, pDNA), thereby realizing diagnosis and treatment of diseases such as cancer, fibrosis (e.g., liver, lung, kidney).
Resumen de: AU2023406947A1
The present application discloses modified single-stranded DNA molecules, as well as their cell-free methods of synthesis and their use as therapeutic agents.
Nº publicación: EP4626858A1 08/10/2025
Solicitante:
UNIV CINCINNATI [US]
University of Cincinnati
Resumen de: WO2024119037A1
Provided herein are an ionizable lipid compound, a lipid nanoparticle comprising the ionizable lipid compound, a composition comprising an mRNA formulated in the lipid nanoparticle, and a method of delivering an mRNA to a subject or a cell by administering the composition including an mRNA formulated in the lipid nanoparticle to the subject or cell.