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186
resultados
Última actualización
03/09/2025 [06:53:00]
Resultados 50 a 75 de 186
Resumen de: WO2025178896A1
Disclosed herein are methods for rapidly screening polymer nanoparticles by exposing a cell to the polymer nanoparticle, the polymer nanoparticle comprising a detectably labeled mRNA, the mRNA encoding a detectable protein; and sorting the exposed cell by the presence or absence of a signal from the detectably labeled mRNA and the detectable protein via flow cytometry; and evaluating the polymer nanoparticle for its ability to deliver the mRNA to the cell and express the protein based on the presence or absence of the signals. Novel polymer nanoparticles, in particular, lipid nanoparticles are also provided herein.
Resumen de: WO2025178422A1
The present invention relates to a radiopharmaceutical using nanoparticles and a method for producing same. The present invention uses nanoparticles incorporating iodine bridges so that the nanoparticles can be selectively delivered to a tumor site, thereby improving the accuracy and efficiency of cancer treatments and minimizing various side effects caused by in vivo accumulation of detached isotopes.
Resumen de: US2025269059A1
Disclosed herein include methods, compositions, and kits suitable for use in gene editing. RT editing systems are provided herein comprising a fusion protein comprising a Cas9 nickase and a reverse transcriptase, and a template armed guide RNA (tagRNA) comprising from 5′ to 3′ a spacer sequence, a scaffold sequence, an editing template and a flap binding sequence. In some embodiments, the RT editing system further comprises an enhancer guide RNA (egRNA).
Resumen de: US2025268913A1
The invention relates to inhibitors of the PKC signaling pathway for use in the treatment of septic cholestasis, wherein the inhibitors are targeted into the liver by a selective nanostructured delivery system, wherein the selective nanostructured delivery system comprises at least one carbohydrate targeting moiety and at least one polymer and/or at least one lipid and/or at least one virus-like particle.
Resumen de: US2025268839A1
The invention relates to therapeutic nanobiologic compositions and methods of treating patients who have cancer, by promoting trained immunity, which is the long-term increased responsiveness, the result of metabolic and epigenetic re-wiring of myeloid cells and their stem cells and progenitors in the bone marrow and spleen and blood induced by a primary insult, and characterized by increased cytokine excretion after re-stimulation with one or multiple secondary stimuli.
Resumen de: US2025268840A1
There are disclosed methods of treating a subject or an object in need thereof, the method comprising administering compositions comprising nano-elements containing: a) at least one water-insoluble thermoplastic compound (WITC), capable of forming a core; and b) at least one active agent which can be disposed in said core or in shells surrounding the core. The nano-elements, having an average diameter in the sub-micron range, are constituted of materials having a low vapor pressure and are dispersible in a polar carrier. Methods for preparing these nano-elements, and administering them, so as to treat conditions corresponding to the active agents contained therein, are also provided.
Resumen de: US2025269062A1
The first systemic administration of a CRISPR/Cas9-based therapeutic for in vivo editing in a clinical trial is described. Described herein are methods for in vivo editing of a liver gene by systemically administering a lipid nanoparticle composition comprising an mRNA encoding a Cas nuclease and a guide RNA that targets the gene. For example, disclosed herein are methods for in vivo editing of a transthyretin gene by systemically administering a lipid nanoparticle composition comprising an mRNA encoding a Cas nuclease and a guide RNA that targets the TTR gene. Assessment of biosafety metrics and clinical efficacy metrics, as well as methods of treatment, are also described herein.
Resumen de: US2025268838A1
A modified nanoparticle for use in a therapeutic method, wherein the therapeutic method comprises the administration of the modified nanoparticle to an organism, the targeting of the modified nanoparticles to a specific site in the organism followed by an uptake of the modified nanoparticle into a cell, and wherein the modified nanoparticle is obtainable by a process comprising the steps of i) providing a nanoparticle and ii) contacting the nanoparticle with one or more antibodies as at a pH value of less than 7.0 so as to non-covalently bind the one or more antibodies via its/their Fc region onto the surface of the nanoparticle, wherein the nanoparticle provided in step i) is made of a material having at least one protonable or deprotonable group on the surface thereof and/or the one or more targeting moieties contacted with the nanoparticle in step ii) has at least one protonable or deprotonable group.
Resumen de: US2025268828A1
Disclosed herein are novel lipids that can be used in combination with other lipid components, such as helper lipids, structural lipids, and cholesterols, to form lipid nanoparticles for delivery of therapeutic agents, such as nucleic acids (e.g., circular polynucleotides), both in vitro and in vivo.
Resumen de: US2025268826A1
Disclosed are lipidoid compounds having the structure of formula (X) or formulawherein the groups are as defined in the application. Also disclosed are nanoparticle compositions comprising a lipidoid of the invention that are capable of delivering a therapeutic agent. The application also discloses pharmaceutical compositions comprising a lipidoid composition of the invention.
Resumen de: US2025268818A1
The present invention relates to the treatment of ocular diseases in a human subject. In particular, the invention relates to an intracameral administration of a sustained release biodegradable intracameral implant.
Resumen de: US2025269053A1
The present invention relates to lipid nanoparticles comprising mannose or a use thereof. The lipid nanoparticles according to an embodiment are specific for liver tissues and/or LSEC, have excellent biological affinity, and can deliver gene therapeutic agents, etc. at high efficiency, thus finding advantageous applications in the relevant technical fields such as lipid nanoparticle-mediated gene therapy, etc.
Resumen de: US2025269044A1
Provided herein is a dendrimer complex comprising a dendrimer and a prostate specific membrane antigen (PSMA) ligand conjugated to an outer surface of the dendrimer. The dendrimer complex may further include one or more drugs, imaging agents, and/or radioligands conjugated to an outer surface of the dendrimer. Such complexes are useful in methods for detecting or inhibiting proliferation of PMSA-expressing cells and for methods of treating cancer characterized by PMSA expression.
Resumen de: WO2025177008A1
The present invention relates to dry powder compositions, methods, apparatus and systems for manufacture of the same. In particular, the present invention relates to a system for producing a dry powder composition including a vacuum chamber depressurised by a vacuum pump and a spray head assembly configured to introduce a liquid formulation into the vacuum chamber, wherein the spray head assembly is ultrasonically stimulated during introduction of the liquid formulation into the vacuum chamber to initiate breakup of the liquid formulation into liquid droplets within the vacuum chamber. The present invention further relates to dry powder compositions comprising lyophilised aggregates produced with such apparatus.
Resumen de: WO2025176898A1
The present invention concerns, among others, a nanovesicle comprising an aqueous core and a shell formed from a plurality of conjugates, each conjugate comprising a head portion, a tail portion and an intermediate portion in-between, wherein the head portion contains a first drug component, the tail portion contains a polymer component and optionally a second drug component and/or a targeting ligand, and the intermediate portion contains a cleavable linker, and wherein the head portion has a higher hydrophobicity than the tail portion.
Resumen de: WO2025177141A1
The present invention relates to the field of plant-derived cellular vesicles, and in particular of nanovesicles obtained from plant protoplasts. A method for their preparation and uses thereof in the medical, cosmetic and nutraceutical fields are described.
Resumen de: US2025269031A1
The present invention proves mitochondria-targeting nanofibers that are formed by assembly of small-molecule building blocks of amphiphilicity to improve photodynamic cancer therapy. Monomers derived from a pheophorbide a and quinolinium conjugates are described, as well as the formation of nanoparticles, formulations of the compounds, and methods of treatment.
Resumen de: US2025269035A1
Disclosed herein are a lipid-charged molecule conjugate, an inhalable lipid nanoparticle, a preparation method therefor, and use thereof. The lipid-charged molecule conjugate of the present invention comprises a lipid unit and a charged unit. The charged unit is selected from a negative charge unit and/or a positive charge unit. The lipid nanoparticle of the present invention comprises an ionizable lipid, an auxiliary phospholipid, cholesterol, a pegylated lipid, and the lipid-charged molecule conjugate. After the lipid nanoparticle of the present invention is administered by means of atomization inhalation, the anti-atomization stability of the lipid nanoparticle and the nucleic acid delivery efficiency are improved. When the lipid nanoparticle was used to encapsulate mRNA vaccine, inhaled mRNA-LNP can activate humoral, cellular, and mucosal immune responses.
Resumen de: US2025268816A1
This invention relates generally to injectable drug delivery systems and methods of preparing same, and in particular to drug delivery systems comprising biodegradable polymeric nanogel formulations that form a depot (gel) in the body once injected, and can release therapeutic agents with enhanced extended-release times in the subject being treated. The polymer matrix drug delivery systems of the subject invention are comprised of a biodegradable polymer, a solvent or a combination of solvents, an alkylphenol, and therapeutic agents such as insulin, tetrandrine, other small molecule drugs (preferably hydrophobic drugs) to treat a variety of diseases including diabetes and hearing loss. A preferred alkylphenol is meta cresol which has demonstrated importance for dissolving and stabilizing the therapeutic agents, and forming gels with better shape and controlled drug release after injection.
Resumen de: US2025270250A1
The disclosure is generally related to spherical nucleic acids (SNAs) comprising a protein corona, wherein the SNA comprises (i) a nanoparticle core and (ii) one or more oligonucleotides attached to the surface of the nanoparticle core, wherein the protein corona comprises a plurality of proteins. The disclosure also provides methods of using the same. The disclosure further provides methods of improving stability and/or extending blood circulation half-life of a spherical nucleic acid (SNA), the SNA comprising a nanoparticle core and one or more oligonucleotides attached to the surface of the nanoparticle core, the method comprising adsorbing a plurality of proteins on the surface of the SNA.
Resumen de: US2025269054A1
Described herein are nanoparticle drug conjugates (NDCs), which, in certain embodiments, comprise a non-toxic, multi-modality, clinically proven silica-based nanoparticle platform with covalently attached drug molecules/moieties. The nanoparticle drug conjugates (NDCs) demonstrate imaging capability and targeting ligands which efficiently clear through the kidneys. Furthermore, the conjugates incorporate therapeutic agents for cancer detection, prevention, and/or treatment.
Resumen de: WO2025176843A1
Provided are gene therapy vectors designated MEV-GTVs, which are MEVs that contain an ITR-containing plasmid (also referred to as a minigene plasmid) that comprises Inverted Terminal Repeats (ITRs), such as viral ITRs, and nucleic acid encoding a product of interest, and optionally regulatory sequences. The ITRs serve to circularize the minigene plasmid. The plasmids do not contain additional viral components, so that resulting DNA is not encapsulated in a viral capsid or envelop and is not replicated by viral genes. The plasmids can be inserted into bacterial plasmids for propagation.
Resumen de: WO2025176738A1
The present invention relates to a mannose-HSA based nanocarrier system, and a pharmaceutical composition containing the same for delivery of immunomodulatory drugs. The present invention further relates to a method of manufacturing said mannose-HSA nanocarrier system.
Resumen de: WO2025176732A1
An aqueous dispersion having an aqueous mobile phase and a dispersed phase, wherein the dispersed phase comprises a lipid mixture including a cationically ionisable lipid; and the aqueous mobile phase comprises water; wherein the aqueous dispersion is substantially free of organic solvents, nucleic acids, organic acids, buffers, and inorganic ions, is described. Methods of preparing the aqueous dispersion, nucleic acid-lipid particles and methods of preparing them using the aqueous dispersion, and their use in medicine are disclosed.
Nº publicación: WO2025176847A1 28/08/2025
Solicitante:
AGS THERAPEUTICS SAS [FR]
AGS THERAPEUTICS SAS
Resumen de: WO2025176847A1
Provided are compositions containing microalgae extracellular vesicles (MEVs) formulated for administration to the eye for treatment of diseases, disorders, and conditions of the eye, particularly for diseases, disorders, and conditions that involve tissues and cells in the back of the eye, which generally cannot be treated with eyedrops. Upon administration to the eye, the MEVs deliver therapeutics to tissues that include the choroid, retinal pigment epithelium cells, and photoreceptor cells. The compositions contain extracellular vesicles from microalgae (MEVs) that are loaded with bioactive cargo for treating or diagnosing or monitoring a disease, disorder, or condition of the eye or involving the eye. Methods of treatment of diseases, disorders, and conditions of the eye are provided.
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