Resumen de: US20260355120A1
The present invention provides, in part, asymmetric “good” buffer-based cationic lipids of Formula (I′), and sub-formulas thereof:or a pharmaceutically acceptable salt thereof. The compounds provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those caused by viral or bacterial infection.
Resumen de: US20260355117A1
The present disclosure provides a particulate delivery system comprising an extracted yeast cell wall comprising beta-glucan and a pro-payload molecule comprising a payload scaffolding molecule operably and reversibly linked to a payload molecule through a cleavable linker. The disclosure further provides methods of making and methods of using the particulate delivery system.
Resumen de: AU2025292967A1
Pharmaceutical compositions of Compound (I) including solid dispersions of Compound (I) and nanocrystalline powder forms of Compound (I). Related unit dosage forms, and methods of making and using the same are also provided.
Resumen de: AU2025284498A1
The present invention relates to a lipid composition for lipid nanoparticles (LNP), which comprises: (a) one or more cationic and/or cationically ionizable lipids; (b) a sterol; (c) one or more helper lipids; and (d) a lipid obtainable by a method comprising (i) subjecting a mixture comprising one or more monomers of the following formula (I), (I) wherein R1 represents an ethyl group, R3 and R4, which may be the same or different, independently represent a hydrogen atom, or a C1-2 alkyl group, to cationic ring opening polymerization in the presence of a polymerization initiator for providing a linear polymer; and (ii) introducing a terminal group R2 of the following formula (II): (II) wherein k is an integer of 1 so that a spacer exists between the polyoxazoline chain and Z, whereby the structure of the spacer is determined by the repeating units y and z; y is 0 or an integer of 1 to 3, and z is 0 or an integer of 1 to 3; a which may be the same or different when more than one is present, represents an integer of 1 to 6; b represents 0 or an integer of 1 to 6; X1 which may be the same or different when more than one is present, independently represents an oxygen atom, a group -O-CO-, -N-CO-, or -NR' wherein R' is a hydrogen atom or an alkyl group; X2 which may be the same or different when more than one is present, represents a single bond or a group -CO-; X3 which may be the same or different when more than one is present, independently represents a single bond, an oxygen at
Resumen de: AU2025376352A1
The present disclosure provides dual gRNAs for allele-selective or specific gene editing, nucleic acids encoding such gRNAs, vectors comprising such nucleic acids, RNPs comprising such gRNAs and Cas protein, pharmaceutical compositions comprising any of the foregoing, and kits comprising any of the foregoing. The present disclosure further provides methods related to such gRNAs, including methods of manufacturing, methods of effecting allele-selective or specific gene editing, and methods of preventing, treating, or ameliorating a symptom of a target disease, disorder, or condition, containing. The present disclosure further provides lipid-based TCVs.
Resumen de: US20260355345A1
An antibacterial nanoparticle comprising an inner silica core; a gold (Au) nanosphere surrounding the inner silica core; a surface layer comprising silver (Ag) surrounding the gold nanosphere or the silver is alloyed into the gold nanosphere; and an antimicrobial peptide conjugated to the surface layer, wherein the antimicrobial peptide is selected from the group consisting of a cysteine terminated Syn71 peptide, a cysteine terminated MC1-2 peptide, and a cysteine terminated Syn20 peptide.
Resumen de: AU2025378053A1
The present disclosure provides gRNAs for allele-selective gene editing, nucleic acids encoding such a gRNA, vectors comprising such a nucleic acid, RNPs comprising such a gRNA and a Cas protein, pharmaceutical compositions comprising any of the foregoing, and kits comprising any of the foregoing. The present disclosure further provides methods related to such gRNAs, including methods of manufacturing, methods of effecting allele-selective gene editing, and methods of preventing, treating, or ameliorating a symptom of a target disease, disorder, or condition using such gRNAs. The present disclosure further provides lipid-based TCVs.
Resumen de: AU2025265441A1
The present disclosure relates generally to nucleic acid-lipid particles, in particular functionalised nucleic acid-lipid particles, methods for producing them, and to pharmaceutical compositions containing them and their uses in medicine.
Resumen de: US20260355297A1
The present disclosure relates to methods of treating Type 1 diabetes (T1D) using tolerizing immune modifying nanoparticles encapsulating T1D associated antigens.
Resumen de: US20260355341A1
The invention relates to a lipid-based nanoparticle comprising an antigen-binding domain capable of specifically binding to a target expressed on activated immune cells surface and one or several mRNA molecule(s) encoding an activity-enhancing protein of said activated immune cells, and to uses thereof.
Resumen de: US20260356808A1
A novel ionizable lipid compound, lipid nanoparticles including the ionizable lipid compound, and uses thereof are disclosed. The novel ionizable lipid compound is represented by formula (I) or a salt thereof. Lipid nanoparticles including a novel ionizable lipid compound have excellent nucleic acid encapsulation efficiency and high cell delivery efficiency of nucleic acids. (Terminal area 1)j-connection1-headk-connection 2-(Terminal area 2)m Formula (I).
Resumen de: US20260355349A1
The invention relates to a lipid-based nanoparticle comprising an antigen-binding domain capable of specifically binding to a target expressed on activated immune cells surface and one or several mRNA molecule(s) encoding an activity-inhibiting protein of said activated immune cells, and to uses thereof.
Resumen de: US20260355339A1
The present invention relates to solid microbeads having an anti-CD28 monoclonal antibody immobilized thereto for use in the local treatment of malignant melanoma, wherein the anti-CD28 monoclonal antibody binds to an epitope on CD28 outside the C″ D loop. The present invention further relates to methods for the local treatment of malignant melanoma comprising the administration of said microbeads.
Resumen de: AU2025243613A1
The present invention relates to formulations, devices and methods for coating surfaces of microprojections on microprojection arrays with nucleic acids. The present invention also relates to formulations and methods for coating surfaces of microprojections with nucleic acids where the nucleic acids are associated with lipid nanoparticles (LNP), in particular where the nucleic acid is mRNA.
Resumen de: US20260355308A1
A composition for an oral vaccine comprises nanoparticles containing an antigen and fibroin.
Resumen de: US20260356882A1
Provided herein are methods for functionalizing nanoparticles with functional proteins, and methods of using the functionalized nanoparticles. Compositions for preparing the functionalized nanoparticles are also described herein.
Resumen de: US20260355124A1
The present disclosure relates to a method for preparing extracellular vesicles with reduced non-specific binding/endocytosis. By means of performing knockout and deglycosylation operations on extracellular vesicle-associated adhesion molecules, extracellular vesicles with reduced non-specific binding/endocytosis are obtained, and the circulation time thereof in the blood is pro-longed. Furthermore, an IL-12 combined antibody is loaded onto the extracellular vesicles with reduced non-specific binding/endocytosis to achieve enhanced efficacy of IL-12 in a “multimer” mode, which further achieves targeting by means of the antibody, reducing an initial concentration of IL-12 and improving safety while also achieving efficient inhibition of tumor growth at a certain level of safety.
Resumen de: US20260355111A1
The present disclosure relates to a protocol for the formulation and lyophilization of hydroxyl-terminated poly(ethylene glycol) (PEG) based block copolymer micelles without the use of excipients (cryo-protectants). The micelles are formulated using the equilibrium nanoprecipitation (ENP) method, which allows for control over the aggregation number of the micelles and thus the grafting density of the PEG micelle corona chains. By using the ENP method to produce micelles with high dimensionless PEG grafting densities (> about 7), the micelles are stabilized against aggregation during lyophilization due to the shielding of the micelle core domains by the dense PEG corona chains. By preventing aggregation during the lyophilization without using excipients, the original desired surface mechanical properties of the micelles are preserved after reconstitution. Therefore, the protocol described herein can be applied to effectively storing PEG-based block copolymer micelles without using excipients while still preserving their original surface mechanical properties.
Resumen de: US20260356793A1
The present invention discloses a novel cationic lipid and a method for preparing the same, a lipid composition containing said cationic lipid and an LNP-drug composition and a formulation thereof containing the lipid composition, in particular, the LNP-pharmaceutical composition and the formulation thereof containing the cationic lipid, which is characterized by high delivery efficiency and high biocompatibility, thereby improving the therapeutic and preventive effects of the drug. The structure of the cationic lipid is shown in the general formula (1), and the definition of each symbol in the formula is described in the text.
Resumen de: US20260356906A1
The invention described herein provides an engineered insulin polypeptide comprising two cleavable peptides interposed between the insulin B-chain and the C-peptide, and between the C-peptide and the insulin A-chain to secrete active insulin from muscle cells. Also provides are polynucleotide encoding the engineered insulin for intramuscular delivery through Adeno Associated Virus (AAV) vectors, and the use thereof to treat diseases such as diabetes, hyperglycemia and other hyperglycemia-related diseases.
Resumen de: US20260355114A1
Hyaluronic acid polymers may be converted to derivatives thereof by reaction with divinyl sulfone to provide vinyl sulfone substituted polymers, where the polymers may additionally be further derivatized, including crosslinked, and the crosslinked and non-crosslinked derivatives may be used in biomedical and other applications.
Resumen de: US20260355125A1
Described herein are electrospun biodegradable flexible nanofiber membranes that retain therapeutic-loaded nanoparticles within fibers, providing localized drug delivery suitable for post-operative treatment of solid tumors. Poly(lactic-co-glycolic acid) fibers retain doxorubicin-loaded mesoporous silicon dioxide nanoparticles to form a drug-in-nanoparticle-in-fiber construct that provides a dual-barrier release architecture governed by nanoparticle pore diffusion and polymer-matrix diffusion and degradation to suppress burst release and provide sustained delivery. The flexible nanofiber membrane comprises coaxial or triaxial fibers that compartmentalize different payloads to provide staged time-release profiles, including a delayed-start release mediated by acidic pH lower than physiological pH, using a pH-responsive layer that increases porosity or permeability under acidic conditions. Representative methods include surfactant-templated synthesis of mesoporous silica nanoparticles, drug loading, dispersion into polymer solutions, electrospinning to form flexible nanofiber membranes, and in vitro characterization of morphology, chemical composition, release behavior, and cellular uptake in a non-small cell lung cancer cell model.
Resumen de: US20260355234A1
Microbiome-ceria nanocompositions and methods for application of these nanocompositions to benefit human health. The nanocomposition formulations include cerium oxide nanoparticles combined with proteins and/or other bioactive agents isolated from commensal microbes such as Lactobacillus acidophilus (L. acidophilus).
Resumen de: US20260355346A1
A hybrid biomimetic nanostructure platform for oral delivery of therapeutics to the central nervous system across dual biological barriers comprises a protective outer shell encapsulating a structured inner core that carries a therapeutic payload and a blood-brain barrier transcytosis moiety. The outer shell bears at least one microfold-cell targeting moiety on its exterior and resists gastrointestinal degradation. Following oral administration the microfold-cell targeting moiety directs transcytosis across the intestinal epithelium; the blood-brain barrier transcytosis moiety thereafter directs receptor-mediated transcytosis across brain capillary endothelium; and a stimulus-responsive element triggers payload release in central nervous system tissue. The platform enables oral administration of small molecule, peptide, protein, nucleic acid, and gene-editing payloads for treatment of neurological, neurodegenerative, neuroinflammatory, neuropsychiatric, neurodevelopmental, and neuro-oncologic conditions. A closed-loop system couples the composition to biomarker-driven dosing adjustment with clinician override.
Nº publicación: US20260355123A1 12/11/2026
Solicitante:
RESEARCH FOUNDATION OF THE CITY UNIV OF NEW YORK [US]
Research Foundation of the City University of New York
Resumen de: US20260355123A1
A drug carrier nanoparticle has been synthesized that can specifically target the proximal tubules of the kidneys. The nanoparticles accumulate in the kidneys to a greater extent than other organs (e.g., up to 3 or more times greater in the kidney than any other organ). They can encapsulate many classes of drug molecules. The nanoparticles are biodegradable and release the drug as they degrade. The particles can sustainably release a drug within the kidneys for up to two months. The nanoparticles are useful for the treatment of diseases that affect the proximal tubules, such as heart failure, liver cirrhosis, hypertension, and renal failure; the study of relative blood flow to the renal cortex and medulla; and delivery of agents to treat gout.