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229
results.
Updated on
09/07/2026 [07:11:00]
Results 200 to 225 of 229
Absstract of: US20260174706A1
0000 The present invention relates to a composition for transpulmonary delivery of nucleic acid drugs and a use thereof. The composition for transpulmonary delivery of the present invention is specific to lung tissue and specific cells thereof, has excellent biocompatibility, and can deliver gene therapy products, etc., with high efficiency, and thus can be useful in relevant technical fields such as in the treatment of lipid nanoparticle-mediated pulmonary diseases.
Absstract of: US20260176625A1
0000 Provided herein is a method for preparing the nanoparticle comprising: (i) combining an aqueous phase comprising the nucleic acid with an organic solvent-lipid mixture comprising lipids, wherein the lipids comprise an ionizable lipid and at least one helper lipid and optionally a hydrophilic-polymer lipid conjugate; wherein the aqueous phase comprises a buffer having a concentration of at least 100 mM and has an aqueous phase pH that is lower than a pKa of the ionizable lipid such that the ionizable lipid is substantially charged; wherein the lipid nanoparticle is formed during or subsequent to the combining; and (ii) exchanging a solution external to the lipid nanoparticle with a higher pH solution, thereby producing the nucleic-acid lipid nanoparticle, wherein the nucleic-acid lipid nanoparticle comprises one or more bleb compartments.
Absstract of: US20260176618A1
Aspects of the disclosure relate to mRNAs comprising a relatively low abundance of cytidine: adenosine (CA) dinucleotides that benefit from increased stability relative to mRNAs containing more CpA dinucleotides. The disclosure also relates to methods of modifying an mRNA sequence to improve stability. In some aspects, the disclosure relates to mRNAs comprising modified mRNA sequences with relatively reduced numbers of CpA dinucleotides, and compositions comprising mRNAs with relatively reduced numbers of CpA dinucleotides.
Absstract of: US20260174902A1
0000 The present disclosure provides nucleic acids, compositions and vectors containing and their use for effecting gene editing and/or gene expression alteration on sickle cell disease (SCD)-associated genes, e.g., in vivo. The present disclosure further provides compositions for and methods of effecting gene editing and/or gene expression alteration, such as on SCD-associated genes in vivo, and methods of preventing, ameliorating, or treating SCD.
Absstract of: US20260174694A1
Provided herein is a lipid nanoparticle comprising an encapsulated oligonucleotide molecule, wherein the oligonucleotide molecule is single-stranded or double-stranded and has a length of between 5 and 500 nucleotides; and 20 to 70 mol % of a neutral lipid content relative to total lipid present in the lipid nanoparticle, an ionizable lipid; a sterol; and optionally a hydrophilic polymer-lipid conjugate.
Absstract of: US20260174820A1
The present invention provides an ionizable polymer comprising a constitutional unit according to formula (I). wherein R1 and R2 are as defined in the specification. The ionizable polymer of the invention finds use in the intracellular delivery of polynucleotides in vitro and in vivo. The present invention also provides compositions comprising the ionizable polymer of the invention and a polynucleotide. The compositions disclosed herein find utility as medicaments, in particular as medicaments for the prevention or treatment of an infectious disease, a cancer, or a protein-deficiency disease.
Absstract of: US20260174700A1
The invention relates to formulation for oral delivery of peptides and/or drug molecules and a method of manufacturing the same. The oral formulation is useful for delivering peptides to the gastrointestinal tract, preferably at the ileo-caecal junction of colon.
Absstract of: US20260174841A1
The present disclosure provides a method of enhancing an immune response in a subject in need thereof, including administering an effective amount of an antigen with an immune modulator to the subject, wherein the immune modulator is a quaternary immunomodulatory nanoparticle (QIN). The present disclosure also provides the use of the QIN in the manufacture of a chimeric nanoparticle, a pharmaceutical composition and a medicament for treating a subject suffering from a cancer or virus infection.
Absstract of: US20260174865A1
The present invention relates to a system for the release of bioactive compounds comprising biodegradable colloidal particles with an average size less than 500 nm, where the biodegradable colloidal particles comprise an anionic polysaccharide and a cationic polysaccharide. Furthermore, its method of extraction includes: (a) estimating the stoichiometric charge ratio between an anionic polysaccharide, and a cationic polysaccharide; (b) preparing a solution with the anionic polysaccharide based on the stoichiometric charge ratio and adjusting to an acidic pH; (c) preparing a solution with the cationic polysaccharide based on the stoichiometric charge ratio and adjusting to an acidic pH; (d) adding the solution with the cationic polysaccharide to the solution with the anionic polysaccharide to obtain biodegradable colloidal particles; (e) reducing the particle size of the biodegradable colloidal particles; (f) separating the biodegradable colloidal particles from the supernatant; and (g) resuspending the biodegradable colloidal particles and storing them.
Absstract of: US20260176231A1
The subject of the present invention is a lipidoid of general formula Iwherein X, Y, Z and R are as defined in the claims.This lipidoid is useful as a transfection agent. The invention further describes transfection agents, transfection particles containing this lipidoid, and their use.
Absstract of: AU2026204465A1
COMPOSITIONS AND METHODS FOR TREATING OCULAR DISEASES 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.5 COMPOSITIONS AND METHODS FOR TREATING OCULAR DISEASES un u n
Absstract of: AU2026204376A1
Antibodies and antigen binding fragments thereof that specifically recognize and bind an epitope of elastin that is exposed and accessible in degraded elastic fiber are described. The antibodies and/or antigen binding fragments can be operably linked to a secondary component, including biologically active agents such as therapeutics and/or imaging agents. Optionally, the antibodies and/or antigen binding fragments thereof can be attached to a surface of a carrier, such as a particle, for specific binding and delivery of the carried agents to degraded elastic fiber. fiber. un u n
Absstract of: US20260174884A1
The present invention provides, among other things, methods and compositions of formulating nucleic acid-containing nanoparticles for efficient delivery of payload in vivo such that the method and compositions can be used to generate mRNA vaccines.
Absstract of: US20260174897A1
Covalently linked linear polyethylenimine (PEI) clusters are provided that change conformation depending upon changes in counterion concentrations. The structures may be used for the storage, delivery, and/or transport of substances.
Absstract of: US20260174704A1
0000 The disclosure relates to cancer ribonucleic acid (RNA) vaccines, as well as methods of using the vaccines and compositions comprising the vaccines.
Absstract of: US20260174909A1
0000 The invention relates to compositions including polynucleotides encoding polypeptides which have been chemically modified by replacing the uridines with 1-methyl-pseudouridine to improve one or more of the stability and/or clearance in tissues, receptor uptake and/or kinetics, cellular access by the compositions, engagement with translational machinery, mRNA half-life, translation efficiency, immune evasion, protein production capacity, secretion efficiency, accessibility to circulation, protein half-life and/or modulation of a cell's status, function, and/or activity.
Absstract of: EP4763227A1
The present invention relates to a composition comprising a solid carrier, a protein branched-chain amino acid-degrading enzyme or a fragment thereof immobilized on the surface of the solid carrier, a protective layer to protect the protein branched-chain amino acid-degrading enzyme or a fragment thereof by embedding the protein branched-chain amino acid-degrading enzyme or a fragment thereof, and a functional constituent immobilized on the surface of the protective layer, wherein the functional constituent immobilized on the surface of the protective layer is a polymer comprising repeat units wherein each repeat unit comprises at least one amino group and/or at least one thiol group. The present invention also relates to methods of producing said composition and uses of the composition for the prevention, delay of progression or treatment of branched-chain amino acid metabolism related diseases.
Absstract of: EP4763189A1
The present invention relates to a composition comprising a solid carrier, a lipase or a fragment thereof immobilized on the surface of the solid carrier wherein the lipase or a fragment thereof is in the open conformation, a protease or a fragment thereof immobilized on the surface of the solid carrier, an amylase or a fragment thereof immobilized on the surface of the solid carrier, an agent which interacts with the lid domain of the lipase or a fragment thereof, a protective layer to protect the lipase or a fragment thereof, the protease or a fragment thereof and the amylase or a fragment thereof by embedding the lipase or a fragment thereof, the protease or a fragment thereof and the amylase or a fragment thereof, and a functional constituent immobilized on the surface of the protective layer, wherein the functional constituent immobilized on the surface of the protective layer is a polymer comprising repeat units wherein each repeat unit comprises at least one amino group and/or at least one thiol group; b) an amino acid; c) a non-reducing sugar and/or a non-reducing sugar alcohol; and d) a surfactant. The present invention also relates to solid, lyophilized and liquid compositions of said nanoparticle composition, capsules comprising said nanoparticle composition, methods of producing said nanoparticle composition and uses thereof.
Absstract of: EP4763197A1
The present invention relates to a composition comprising a solid carrier, a protein ATP-hydrolyzing enzyme or a fragment thereof immobilized on the surface of the solid carrier, a protective layer to protect the protein ATP-hydrolyzing enzyme or a fragment thereof by embedding the protein ATP-hydrolyzing enzyme or a fragment thereof, and a functional constituent immobilized on the surface of the protective layer, wherein the functional constituent immobilized on the surface of the protective layer is a polymer comprising repeat units wherein each repeat unit comprises at least one amino group and/or at least one thiol group. The present invention also relates to methods of producing said composition and uses of the composition for the prevention, delay of progression or treatment of dysbiosis or a dysbiosis-related disease. The present invention also relates to a pharmaceutical combination comprising (i) said composition; and (ii) an immune checkpoint modulator and uses thereof for the prevention, delay of progression or treatment of cancer and/or for use in adoptive (T) cell therapy.
Absstract of: WO2025143118A1
The present invention pertains to: a pharmaceutical composition for treating spinal cord injury, the pharmaceutical composition containing an mRNA encoding NeuroD1 protein; a method for producing a non-human primate animal model with which it is possible to assess spinal cord injury (in particular, chronic-stage spinal cord injury); and a method for assessing, by using the model, the effect of a test substance as a therapeutic agent for spinal cord injury.
Absstract of: EP4763287A2
The invention relates to novel polymer conjugated lipids and to novel compositions comprising said novel polymer conjugated lipids useful for the delivery of nucleic acids into living cells.
Absstract of: WO2025062064A1
The present disclosure provides a structural protein comprising an amino acid sequence unit comprising a motif (VPGVG)n, wherein n is 2 or more, and an amino acid sequence selected from SEQ ID NO:1–10. The present disclosure also provides a polynucleotide, an expression vector and a host cell, as well as a method for producing the structural protein. The present disclosure also provides a medical product, an electrospun filament comprising the structural protein, a protein-based micro-robot, photonic crystals, metamaterial and thermoresponsive glass comprising one or more of the structural proteins. The present disclosure also provides a method for preparing a product. The present disclosure also provides use of one or more of the structural proteins for preparing a product.
Absstract of: KR102647261B1
The present invention relates to a composition for drug delivery comprising a telomerase activator and nanoparticles, and a composition for preventing, alleviating, or treating hair loss comprising the same. More specifically, the present invention relates to a composition having an effect of promoting the regeneration and growth of pores as a nanoliposome, a nanosilica particle, or a nanobubble composition in which drugs such as a plant extract component TA-65 which increases telomerase activity and derivatives thereof, a telomerase activating compound GPC, and derivatives thereof are encapsulated.
Absstract of: WO2025036956A1
The present invention relates to compositions designed for localized delivery within a subject's body. More particularly, the invention pertains to therapeutic compositions that remain localized to specific organs or tissues and do not exhibit systemic distribution. These compositions include specific carriers and therapeutic agents suitable for various medical applications.
Nº publicación: EP4762030A1 24/06/2026
Applicant:
BEAM THERAPEUTICS INC [US]
Beam Therapeutics Inc.
Absstract of: WO2025038855A1
The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use such as a compound of Formula (A) or a pharmaceutically acceptable salt thereof.
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