Resumen de: WO2021170841A1
Disclosed are pharmaceutical compositions including a plurality of nanoparticles including a compound of the following structure (1) or a pharmaceutically acceptable salt thereof. Also disclosed are methods of their use and preparation.
Resumen de: AU2024398058A1
The present disclosure describes improved LNP-based RNA vaccines, nucleobase editing systems, and therapeutics for use in treating and/or immunization against disease. In particular, the disclosure describes improved LNPs, including novel and improved ionizable lipids for making LNPs, that enhance the targeted delivery of LNP-based RNA vaccines 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 vaccines and/or therapeutic agents.
Resumen de: EP4825843A1
0001 The present disclosure relates to an imidazole lipid and a use thereof. Specifically, provided are a compound represented by formula I or a salt thereof, and a lipid particle or a pharmaceutical composition containing the compound. The compound can be used as a delivery system, and is used for preparing drugs for preventing and/or treating cancers, infectious diseases, autoimmune diseases, neurodegenerative diseases, inflammation, etc. The definition of each group in formula (I) is as described in the description.
Resumen de: WO2025125384A1
The present invention relates to compositions comprising metal-organic framework (MOF) nanoparticle or nanoparticles, methods of preparing said compositions, methods of treatment using said compositions, and uses of said compositions.
Resumen de: WO2025129174A1
Tuberculosis (TB) remains a leading cause of death from infectious disease worldwide, in part due to the limited efficacy of currently available vaccine. Disclosed herein are compositions and methods for providing immunogenic protection against TB.
Resumen de: WO2025128916A1
Systems for analyzing biological components are provided. The systems may include a fluidic device and an energy source in communication with the fluidic device. The energy source may supply energy to the fluidic device to form a polymer matrix on or adjacent to a biological component within the fluidic device. Methods of using the systems to analyze biological components are also provided.
Resumen de: EP4825838A1
The present disclosure relates to a cationic lipid and use thereof. Specifically provided is a compound represented by formula I or a salt thereof, wherein R1 to R4, H1, H2, and L1 are as defined in the present disclosure. The cationic lipid is used for delivering polynucleotides or nucleic acids and protecting the polynucleotides or nucleic acids from being degraded and removed in serum, and has excellent delivery efficiency.
Resumen de: WO2025128687A1
Described herein are peptide amphiphiles (PAs) for inclusion in droplets and hydrogels.
Resumen de: WO2025129124A1
Compositions and methods for treating a cancer or an infectious disease by delivering a compound comprising a polymeric backbone and a STING agonist covalently linked by a stimulus responsive linker are provided.
Resumen de: WO2025129128A1
The present disclosure provides compositions and methods for non-viral delivery of therapeutics, and methods of preparing said compositions.
Resumen de: EP4825839A1
0001 The present application relates to a cationic lipid containing a disulfide bond of Formula (1), wherein, -S-S- is a disulfide bond; L<1> and L<2> are each independently a degradable divalent linking group, X<1> and X<2> are each independently an acyclic divalent linking group containing a tertiary amine group; G<1> and G<2> are each independently a linking bond or a trivalent branching group; when G<1> is a linking bond, k1 is 1; when G<1> is a trivalent branching group, k1 is 2; when G<2> is a linking bond, k1 is 1; when G<2> is a trivalent branching group, k2 is 2; R<1> and R<2> are each independently a substituted or unsubstituted C<5-30> hydrocarbon group or C<5-30> hydrocarbon derivative residue. The lipid composition prepared with the cationic lipid of the present application, under the premise of good ability to complex with nucleic acid drugs, stability in the systemic circulation, and low toxicity, can more effectively exert the therapeutic effect of the LNP-nucleic acid pharmaceutical composition formulation, thereby improving immunological or therapeutic outcomes.
Resumen de: MX2026007279A
An immunomodulatory combination that includes a first lipid conjugate, a second lipid conjugate, and one or more nucleic acids. The first lipid conjugate includes a corticosteroid covalently linked to a first lipophilic moiety by a first cleavable linkage or through a first cleavable linker. The second lipid conjugate includes an mTOR/mTORC inhibitor covalently linked to a second lipophilic moiety by a second cleavable linkage or through a second cleavable linker. The one or more nucleic acids encode one or more epitopes targeted by autoreactive T cells, wherein the one or more epitopes comprise one or more class II epitopes. The first lipid conjugate, the second lipid conjugate, and the one or more nucleic acids are co-formulated together in a delivery vehicle, or are formulated in two or three separate delivery vehicles. The immunomodulatory combination may be used for treating autoimmune diseases or conditions involving autoreactive T cells, which includes both CD4+ T cells and CD8+ T cells.
Resumen de: WO2025128902A1
Provided are ionizable cationic lipids and lipid nanoparticles for the delivery of nucleic acids to cells (e.g., immune cells), and methods of making and using such lipids and targeted lipid nanoparticles.
Resumen de: WO2025128696A1
Compounds are provided having the following Formula (I): or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein R1, R2, R3a, R3b, R3c, R3d, L1, L2, and n are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided.
Resumen de: WO2025123134A1
The present application relates to ionizable lipids that include ester functional groups. The present application further relates to compositions and uses thereof for the delivery of agents such as nucleic acids and drugs.
Resumen de: WO2025073056A1
The present disclosure provides a method for delivery of cargo to a haematopoietic stem or progenitor cell of a subject, the method comprising contacting a lipid nanoparticle encapsulating the cargo with the haematopoietic stem or progenitor cell ex vivo or in vivo, thereby causing cellular uptake of the cargo, the lipid nanoparticle having between 30 mol% and 70 mol% of a neutral or zwitterionic amphipathic lipid having a net-neutral charge at physiological pH, an ionizable cationic lipid, and a sterol, wherein the lipid nanoparticle is substantially uncharged at physiological pH and has an apparent pKa of between 6.0 and 7.5. Further provided are lipid nanoparticle compositions and uses thereof.
Resumen de: WO2025064507A1
The present disclosure relates to compositions and methods for treating phenylketonuria (PKU). In particular, the present disclosure relates to AAV donor polynucleotide vectors and LNP compositions comprising a targeting gRNA pair and a nucleic acid encoding Cas-CLOVER fusion protein, and methods of using the compositions for treating PKU.
Resumen de: EP4527927A1
The present invention relates to the field of oligonucleotide-based antiviral therapies. In particular, the invention discloses oligonucleotide molecules that may either be provided as RNA molecules capable of inducing RNAi-mediated immunity against human parainfluenza virus or as antisense oligonucleotides (ASOs), e.g., gapmers, targeting RNAs derived from human parainfluenza virus as well as oligonucleotide-based therapeutics utilizing said oligonucleotide molecules. In further aspects, the invention provides methods for screening and preparing an oligonucleotide-based therapeutic that can be used to treat an infection with a respiratory virus of interest. Such therapeutics can deliver the claimed oligonucleotides, e.g., via LNPs (lipid nanoparticles) or lipid anchors, or via lentiviral vectors or polymers such as, e.g., polyethylenimine (PEI vectors). Finally, the present invention provides a composition for use in treating a respiratory disease which comprises a lentiviral vector pseudotyped with a vesiculoviral envelope glycoprotein and at least one surface protein of influenza A virus and/or of respiratory syncytial virus.
Resumen de: WO2025056466A1
The invention provides stabilizing agents, compositions, methods of manufacturing, and uses thereof. In one aspect, a lipid nanoparticle composition includes: (a) an ionizable lipid; (b) two or more lipids; and (c) a stabilizing agent of formula (I) and a method for preparing the stabilizing agent and the lipid nanoparticle are provided.
Resumen de: WO2025059498A2
Provided herein are recombinant polypeptides comprising an engineered ectodomain of a viral protein from enveloped viruses. Also provided herein ar two-component protein nanostructures and compositions for use in vaccinating, generating an immune response, or treating or preventing a viral infection.
Resumen de: TR2025004339A1
Buluş, beyin felci sonrası oluşan nörolojik hasarı azaltan akıllı ilaç sistemleri ile ilgilidir. Buluş özellikle; Sodyum selenit (Na2SeO3)?den bitkisel bir biyomolekül olan epigallokateşin gallat (EGCG) kullanılarak EGCG@Se NPs sentezi yapılması ve STING (stimulator of interferon genes) yolak modülatörleri olan CMA (agonist) ve C176 (antagonist), EGCG@Se NPs ile elektrostatik etkileşimler aracılığıyla kendi kendine konjuge edilmesi;CMA-EGCG@Se NPs ve C176-EGCG@Se NPs yapılarının, hedeflenmiş ilaç taşıma sistemleri haline getirmek amacıyla CD71 antikoru (OX26) ile kaplanması; Sentezlenen CMA-EGCG@Se NPs ve C176-EGCG@Se NPs yapılarının, mikroskobik ve spektroskobik yöntemler kullanılarak karakterize edilmesi;OX26-CMA-EGCG@Se NPs ve OX26-C176-EGCG@Se NPs yapılarının yüzeyinde hareketsizleştirilmiş OX26 antikor moleküllerinin miktarını belirlenmesi işlem adımlarını içeren OX26 Kaplı STING modülatörlü nanopartikül sistemleri ve serebral iskemi tedavisinde kullanımları ile ilgilidir.
Resumen de: WO2025132122A1
Disclosed herein are methods for delivering therapeutic agents using lipid nanoparticles (LNPs) containing at least one compound of Formula (I) or a pharmaceutically acceptable salt, tautomer, hydrate, solvate, stereoisomer, or isotope thereof, wherein R1, R2, R3, m, and n are as defined herein. Also disclosed are methods for treating mucosal disorders with LNPs including the compound of Formula (I).
Resumen de: EP4825376A1
The present disclosure belongs to the technical field of molecules, and particularly relates to a polypeptide-encapsulated quercetin molecular capsule and a preparation method therefor. The method includes steps of: (1) mixing amino acid A, quercetin, and water in a ratio, followed by ultrasonic enzymolysis to prepare a precursor; (2) mixing the precursor with amino acid B in a ratio to prepare a molecular capsule precursor; and (3) adding the molecular capsule precursor into a crystallizer for recrystallization of an outer structure, and performing filtering and high-pressure drying to prepare the polypeptide-encapsulated quercetin molecular capsule. The present disclosure can effectively improve the bioavailability of the quercetin in the constructed molecular capsule by optimizing the powder properties of the drug crystal, and implement controllable sustained release through a specific particle size, thus better controlling the release rate and release amount of the quercetin.
Resumen de: WO2025128958A1
Aspects of the disclosure relate to compositions and methods for improving the stability of lipid nanoparticles (LNPs). In some embodiments, the LNPs comprise one or more active pharmaceutical ingredients (API) encapsulated therein. The disclosure is based, in part, on compositions that directly or indirectly reduce the degradation (e.g., oxidation, hydrolysis, etc.) of one or more lipids components of the lipid nanoparticle. In some embodiments, the compositions comprise a citrate drug product matrix, EDTA drug product matrix, methionine drug product matrix, and/or a tryptophan drug product matrix. The disclosure also provides methods for storing compositions contemplated herein as well as methods for improving the stability of the API.
Nº publicación: EP4823566A1 21/10/2026
Solicitante:
UNIV PENNSYLVANIA [US]
The Trustees of The University of Pennsylvania
Resumen de: WO2025128913A1
Composition and methods for preventing or reducing inflammation resulting from nanoparticle administration to a subject are provided. In one embodiment a method of delivering an agent to a target organ of a subject, comprises administering to the subject a galectin inhibitor and a nanoparticle comprising the agent, wherein inflammation resulting from administration of the nanoparticle is reduced or prevented.