Resumen de: US2025367234A1
Described herein are cell-free therapeutic compositions derived from blood or plasma and uses thereof for the treatment of selected diseases and disorders.
Resumen de: US2025367319A1
This disclosure relates to compositions and methods for treating lung disorders, including, for example, pulmonary fibrosis, and other fibrotic disorders. Thioredoxin domain-containing 5 (TXNDC5) is significantly increased in fibrotic lungs from human PF patients. Therefore, a targeted nanoparticle comprising an inhibitor of TXNDC5 was developed, which may have potential to treat pulmonary fibrosis.
Resumen de: US2025367320A1
The present disclosure relates to RNA particles for delivery of RNA to target tissues after administration, in particular after parenteral administration such as intravenous, intramuscular, subcutaneous or intratumoral administration, and compositions comprising such RNA particles. The RNA particles in one embodiment comprise single-stranded RNA such as mRNA which encodes a peptide or protein of interest, such as a pharmaceutically active peptide or protein. The RNA is taken up by cells of a target tissue and the RNA is translated into the encoded peptide or protein, which may exhibit its physiological activity.
Resumen de: US2025367322A1
The disclosure provides nucleic acid-containing lipid nanoparticle (LNP) compositions and methods relating to the delivery of TnpB nucleobase editing systems comprising TnpB polypeptides, engineered TnpB ncRNAs, and optionally one or more additional accessory functionalities (e.g., a deaminase, reverse transcriptase, recombinase, nuclease, a donor template, or combinations thereof) for use in applications such as precision gene editing.
Resumen de: US2025367300A1
A self-assembled nanomaterial includes a Janus base nanotube and a biologically active molecule covalently or non-covalently adhered thereto, wherein the Janus base nanotube includes at least one compound represented by disclosed Formulas I, II, III, IV. V. VI, VII, VIII, or IX, or a pharmaceutically acceptable salt thereof.
Resumen de: US2025367274A1
The present application is directed, in general, to tolerizing immune mediated particles comprising gene therapy vector antigens for use in combination with gene therapy regimens in order to reduce immunogenicity to the gene therapy vector antigens and/or transgene protein products expressed by the vectors.
Resumen de: US2025367275A1
The present disclosure provides methods for treating an individual with pancreatic cancer with an individualized cancer vaccine and a PD-1 axis antagonist.
Resumen de: US2025367133A1
The disclosure relates to block copolymer nanoparticles for therapeutic delivery of nucleotides, and methods therefor. More particularly, the invention relates to polymer nanoparticles, such as reversible addition-fragmentation chain transfer (RAFT) polymer compositions, for delivering miRNAs.
Resumen de: AU2024281393A1
Compositions and methods for enhancing payload-based gene therapy by increasing the percentage of payload delivered to a non-liver target in a subject by blocking binding of LDL to LDL receptors (LDLR) in the liver, and then administering a payload-based therapy targeting a non-liver tissue.
Resumen de: AU2024271802A1
The present disclosure relates generally to lipid nanoparticles (LNPs) and compositions comprising the same, and their use in delivery of agents, such as nucleic acid-based therapeutics, in particular to transfection recalcitrant cells and/or to lung tissue.
Resumen de: AU2024269259A1
In various aspects and embodiments provided are compositions containing extracellular vesicles (including modified extracellular vesicles), methods of making preparations of extracellular vesicles, methods of modifying extracellular vesicles and methods of using modified and/or unmodified extracellular vesicles.
Resumen de: WO2025250730A1
The present disclosure provides lipid compounds and compositions (e.g., lipid nanoparticle (LNP) compositions) comprising lipid compounds of the present disclosure. The present disclosure provides methods of delivering an active agent (e.g., polynucleotide) to a cell or tissue in a subject, preferably an extrahepatic cell or tissue, comprising administering to the subject an effective amount of a lipid nanoparticle of the present disclosure, wherein the lipid nanoparticle comprises lipid compounds of the present disclosure and the active agent (e.g., polynucleotide).
Resumen de: WO2025248434A1
A composition for topical use in the treatment of androgenetic alopecia comprises a nanoemulsion, said nanoemulsion comprising a mixture of oils, at least one surfactant, finasteride and minoxidil. The mixture of oils comprises sandalwood oil, rosemary oil and sweet almond oil.
Resumen de: WO2025247930A1
Nanoparticles (1) for administering at least one active ingredient (4), said nanoparticles comprising a magnetizable core (2), the core (2) having a shell (3) made of a polyphosphate and said shell (3) completely enclosing the core (2), characterized in that the shell (3) is mixed with an active ingredient (4).
Resumen de: WO2025247992A1
The invention relates to antigen-functionalized dextran-based nanoparticles capable of activating, functionally modifying, re-programming, and stimulating expansion of T cells, A pharmaceutical comprising the nanoparticles find use in the treatment of a medical condition requiring the activation and/or expansion of T-cells and/or in the production of receptor engineered-T cells used in such treatments.
Resumen de: WO2025247999A1
Method for the production of extracellular vesicles, EVs, loaded with a molecule of interest in a bioreactor containing a porous three-dimensional, 3D, scaffold, wherein the method comprises a seeding of the scaffold with a starting number of adherent immortalized cells resuspended after detachment, an attachment of the cells on said scaffold, an expansion of the cells on said scaffold up to a second number of adherent cells, defining a multiplication factor of at least 5 for a single expansion step, a production of EVs by said cells, a phase of loading said EVs with said molecule of interest, a harvest of EVs.
Resumen de: WO2025247843A1
The present invention relates to a pharmaceutical, dental and/or cosmetic composition comprising purified and/or isolated matrix derivative (EMD) proteins and a suitable pharmaceutical carrier, characterized in that the purified and/or isolated enamel matrix derivative (EMD) proteins comprised in said composition have a predominant MW of between 10-13kDa and an aggregation particle size of between 20-200 nm at Room Temperature (RT) and a pH of between 60-7.5. Said pharmaceutical, dental and/or cosmetic composition is disclosed for use in healing, restoration, enhancement and/or promotion of soft tissue in the oral cavity and/or craniomaxillofacial complex (CMF), in particular for use in treating patients suffering from a gingival deficiency and/or disorder. Preferably, the composition of the invention is administered via injection into the soft tissue in the oral cavity and/or the craniomaxillofacial complex (CMF) of the patient.
Resumen de: AU2024251515A1
The present application relates to lipid nanoparticles containing a steroid compound, and the preparation and a use of the lipid nanoparticles. The lipid nanoparticles can be used to deliver effective loads (such as a nucleic acid) into non-liver organs such as the spleen for the treatment or prevention of certain diseases or conditions, particularly spleen-associated diseases.
Resumen de: US2025367135A1
The present invention relates to a dendritic cell-mimicked nanostructure and a fabrication method therefor and, more specifically, to a nanostructure in which a shell including a cell membrane of dendritic cell-derived lipid molecules is introduced to a nanoparticle core in order to take advantage of the surface antigen-presenting ability of dendritic cells and which enables targeting without disappearance in vivo, thereby providing an effect of inducing an effective immune response.
Resumen de: US2025367131A1
The invention encompasses novel ionizable lipids compounds and their use in lipid nanoparticles delivery systems that are useful in the delivery of nucleic acids to a mammalian subject that can be included for use, for example, as cancer vaccines, gene editing therapeutics, delivery of nucleic acid (e.g., mRNA) encoding antibodies, vaccines for infectious disease, and protein replacement therapeutics. Additionally, the invention encompasses compositions and therapeutics comprising the ionzable lipids in the lipid nanoparticles and the use of the composition and therapeutics for the preparation of a pharmaceutical composition, especially a vaccine, (e.g., for use in the prophylaxis or treatment of infectious diseases, tumor or cancer diseases, rare diseases, allergies, or autoimmune diseases). The invention encompasses methods of treatment or prophylaxis of the aforementioned diseases.
Resumen de: US2025367134A1
The technology includes selective modulation of only a peripheral CB1R by using a CB1R antagonist contained in a peripherally restricted delivery system.
Resumen de: US2025368757A1
Disclosed are methods for producing matrix-encapsulated proteins, including matrix-encapsulated hemoglobin. Also provided are pharmaceutical compositions comprising a matrix-encapsulated hemoglobin, as well as methods of using thereof to treat hypoxia, cyanide poisoning, hydrogen sulfide poisoning, and/or azide poisoning.
Resumen de: US2025368958A1
Chimeric antigen receptor (CAR)-expressing neutrophils loaded with nanoparticles comprising a drug; and a method of treating cancer or other disorders in a subject comprising administering to the subject a therapeutically effective amount of the CAR-expressing neutrophils.
Resumen de: WO2025250940A2
The present disclosure provides a nanobody platform having a high affinity purification on protein A, wherein the nanobodies may incorporate diverse bioactive peptides, lipid or glycoside in its CDR1 and/or CDR3 regions. The present disclosure also provides a peptide-modified nanobody comprising non-toxic bioactive peptides and that exhibit potent anti-tumor activity. Moreover, the peptide-modified nanobody is able to be combined with other technologies, such as but not limited to bispecific antibodies and antibody-drug conjugates. Further, the present disclosure also provides production processes of the nanobodies and their use as a treatment and diagnostic agents.
Nº publicación: WO2025250344A1 04/12/2025
Solicitante:
MUSC FOUNDATION FOR RES DEVELOPMENT [US]
MUSC FOUNDATION FOR RESEARCH DEVELOPMENT
Resumen de: WO2025250344A1
A C-terminal fragment of insulin-like growth factor binding protein 2 (IGFBP2) containing 3 cysteine residues has been shown to spontaneously self-assemble into nanotube (NT) structures. These NTs can encapsulate drugs during the assembly process and then deliver their cargo intracellularly following binding to interns and endocytosis. The integrin-dependence of this process is mediated by the presence of a naturally occurring RGD motif within the peptide sequence. A particular advantage of such NTs is that by encapsulating small molecule drugs, tissues are protected from their adverse effects. Here, the use of NTs to deliver small drugs and biologics across the blood-brain barrier (BBB) by penetrating the CNS and delivering their cargo is described.