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218
results.
Updated on
23/12/2025 [06:52:00]
Results 25 to 50 of 218
Absstract of: CN121154559A
本发明公开了一种载核酸脂质纳米颗粒的冻干制剂的制备方法，涉及生物医药技术领域。本发明至少包括以下步骤：S1：配制含有载核酸脂质纳米颗粒和冻干保护剂的液体组合物；S2：降温进行预冻；S3：在真空条件下升温进行干燥，制备得到包含载核酸脂质纳米颗粒的冻干制剂。本发明制备方法能使载核酸脂质纳米颗粒的冻干制剂在2℃‑8℃条件下可以稳定储存至少24个月，极大提高了载核酸脂质纳米颗粒冻干制剂的稳定性，安全性更高，更具有市场竞争力。
Absstract of: US2025382349A1
The present invention includes compositions and methods for retrieving tumor-related antibodies and antigens. In one aspect, the invention includes a method for Sequential Tumor-related Antibody and antigen Retrieving (STAR) which directly and efficiently identifies potent antibodies that can specifically bind to tumor-related antigens on the tumor cell surface. In another aspect, the invention includes a CAR comprising a nanobody, a transmembrane domain, and an intracellular domain, wherein the nanobody is retrieved by a STAR method. In another aspect, the invention includes a CAR T system that targets CD13 and treats acute myeloid leukemia. In another aspect, the invention includes a CAR T system and ADC that targets CDH17 and treats NETs and other types of tumors expressing this antigen, with tolerable toxicities.
Absstract of: WO2025257084A1
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
Absstract of: US2025382376A1
A novel nanobody (Nb) and a nanobody-drug conjugate (NDC) targeting CD73, a method for preparing same, and use thereof are provided. The monoclonal nanobody and the corresponding NDC can efficiently bind to isolated CD73, various tumor cells and CD73 on the surface of an immune cell with high specificity and block the catalytic activity of CD73 enzymes, exhibiting high affinity, low immunogenicity, and a significant anti-tumor effect.
Absstract of: US2025381280A1
The present invention relates to a nanoplatform for targeting an inflammatory macrophage and a composition for preventing or treating inflammatory diseases using the same. The nanoplatform for targeting the inflammatory macrophage according to the present invention can selectively target the inflammatory macrophage with a conjugated glucosyl group as a transmitter. Furthermore, it can suppress inflammatory responses in damaged cells or tissues, restore mitochondrial function, and inhibit cell death, making it an effective composition for preventing or treating inflammatory diseases, particularly kidney diseases.
Absstract of: US2025381300A1
The present disclosure relates to compositions and methods for treating phenylketonuria (PKU). In particular, the present disclosure relates to AAV-piggyBac transposon polynucleotide vectors and LNP compositions comprising a nucleic acid encoding a transposase, and methods of using the compositions for treating PKU.
Absstract of: US2025381228A1
Disclosed herein are methods for treating osteoarthritis may be a one-step arthroscopic procedure and may include detaching synovial mesenchymal stem cells (MSCs) from the synovium using a brush device: covering articular cartilage in an affected joint with a scaffold, and placing concentrated MSC exosomes into the affected joint to stimulate differentiation of synovial MSCs into articular cartilage cells.
Absstract of: US2025381150A1
Provided are lipid nanoparticles, compositions, and methods of making and using the same. The lipid nanoparticles contain ionizable lipids, structural lipids, PEG lipids and specific amounts of helper lipids. The lipid nanoparticles may further contain a cell targeting group coupled to a PEG lipid. The lipid nanoparticles may carry a cargo, e g., a mRNA. The lipid nanoparticles may be used for transfection of cells, e.g., immune cells or hematopoietic stem cells.
Absstract of: US2025382633A1
Disclosed herein is an adenoviral vector system utilizing DARPin adapters. The system is highly effective, safe and able to deliver DNA in a cell-specific manner. It is demonstrated that the system is unexpectedly versatile, and can be used in conjunction with protein scaffolds, bioactive peptides and small molecules. This makes the system useful for numerous purposes, including the use of the system for therapeutic and diagnostic purposes.
Absstract of: US2025382341A1
Compositions comprising at least one nanoparticle containing a nucleoside RNA molecule encoding FOXP3 and an optional second agent are described herein. In some cases, the RNA molecule is circular and contains one or more IRES. Methods for treating or preventing inflammation are also described herein.
Absstract of: US2025381139A1
A multi-tail type ionizable lipid, a preparation method therefor and the use thereof are disclosed. The structural formula of the multi-tail type ionizable lipid of the present invention is as follows,wherein R1 and R2 are the same or different, and each is hydrogen or an alkyl chain or an alkyl ring consisting of 1 to 6 carbons, or R1 and R2 together form a nitrogen-containing alkyl ring; L1 and L2 are the same or different, and each is an alkyl chain or an unsaturated hydrocarbyl group consisting of 1 to 6 carbons in length; and R is an alkyl group, an alkyl ring, an unsaturated hydrocarbyl group, or a heterohydrocarbyl group; and n=1 to 6, m1=1 to 15, m2=1 to 15, and x=0 to 5.
Absstract of: US2025381138A1
The present nanoemulsions are a delivery system for virtually any non-opioid active agent for pain, and contain: a hydrocarbon lipid; a perfluorocarbon; water and/or buffered saline; a nonionic surfactant; and optionally a quaternary ammonium compound and/or an additional lipid. Droplets of the non-opioid analgesic formulations have a diameter of 150 nm or less, and preferably range from 90 nm to about 120 nm and have shelf stability for at least twelve months. The nanoemulsions are suitable for a wide variety of routes of administration, including but not limited to IV and parenteral, the latter having particular battlefield and war zone suitability.
Absstract of: US2025381144A1
The present disclosure provides, in part, peptide self-assemblies that are made into tablet form and methods of making and using the same. In some embodiments, the disclosure provides methods and formulations for a tabletized form of a vaccine, particularly a vaccine comprising self-assembling peptide-polymer nanofibers, an excipient and an adjuvant. Methods of making and using the tablet formulation are also provided.
Absstract of: US2025381140A1
The present invention is directed to lipid nanoparticles using cationic cholesterol for topical delivery for nucleic acid delivery, and when administered locally, side effects caused by systemic drug delivery can be minimized and protein expression can be confined to the site of administration. In addition, the duration of protein expression at the site of administration can be increased, and thus the lipid nanoparticles can be useful in the technical field related to nucleic acid therapeutics.
Absstract of: US2025381149A1
The present invention relates to a method and compositions for optimized cytosolic delivery of active agents, in particular nucleic acids, using a specific class of cationic amphiphilic compounds. The method and compositions of the invention enhance intracellular release of the agents and can be used for the treatment of various disorders.
Absstract of: US2025381173A1
The present invention is within the technical field of pain-relief and relates to a pharmaceutical composition comprising at least one anesthetic agent selected from the group consisting of ropivacaine, bupivacaine, etidocaine, levobupivacaine, lidocaine, lignocaine, mepivacaine, articaine, dibucaine, levobupivacaine, prilocaine, benzocaine, chloroprocaine, cocaine, procaine, proparacaine, tetracaine and any pharmaceutically acceptable salt, hydrate, solvate or prodrug thereof; at least one alkanolamine selected from the group consisting of triethanolamine, tripropanolamine and trimethanolamine; water; and optionally a pharmaceutically acceptable diluent, carrier and/or excipient. The present disclosure furthermore relates to the use of the composition for providing pain-relief, a method of treatment, a method of producing said pharmaceutical composition as well as a carbon quantum dot formed from components of the pharmaceutical composition.
Absstract of: US2025385912A1
Improved methods and reactants for the chemical synthesis of therapeutic nanoparticles are provided. The nanoparticles comprise a polymeric core, to which is attached one or more homing molecules and one or more therapeutic agents. Improvements in speed, yield and purity are attained using the methods disclosed herein.
Absstract of: US2025381141A1
Formulation of nanoparticles of calcium phosphate, preferably hydroxyapatite, said nanoparticles being modified with lecithin, preferably phosphatidylcholine, said formulation having an enhanced cellular uptake and being a carrier for bisphosphonate, characterised in that bisphosphonate is selected from the group of bisphosphonate drugs approved for medical use, the group comprising alendronate and zoledronate, bisphosphonate is encapsulated in calcium phosphate nanoparticles in an amount up to 40% by mass, and the nanoparticles are less than 200 mn in size. Method of obtaining said formulation, comprising the steps: a. dissolving Ca(N03)2. 4H2O in a lecithin solution, b. dissolving (NH4)2HP04 in a bisphosphonate solution, c. adjusting the pH of the solution resulting from step a. and of the solution resulting from step b. to the value of 10, d. mixing the solutions from step c. in a reactor to obtain a suspension, e. centrifuging the suspension from step d. to obtain precipitate, f. purifying the precipitate from step e. by rinsing it four times with ultrapure water and centrifuging, g. drying the precipitate from step f. at 50° C. for 12-24 h, h. grinding the precipitate from step g. in a ball mill for 10 minutes at a speed of 150 rpm, wherein step d. is carried out in a continuous or batch reactor.
Absstract of: US2025381213A1
This disclosure relates to methods treating cancer comprising administering to a subject in need thereof a lipid nanoparticle comprising a therapeutically effective amount of a CRISPR/Cas system comprising one or more nucleic acid sequences encoding one or more guide RNAs (gRNAs) that are complementary to one or more target sequences in a cancer gene of a cancer cell and a nucleic acid sequence encoding a CRISPR-associated endonuclease.
Absstract of: US2025382640A1
The present disclosure relates to compositions comprising lipid nanoparticles for delivering nucleic acid molecules into cells. Also included are methods for producing and using such compositions.
Absstract of: US2024156905A1
Fas-associated factor 1 (FAF1) protein-loaded exosomes and a cancer treatment using FAF1 protein-loaded exosomes are disclosed. FAF1 protein-loaded exosomes are isolated from HEK 293 cells in which FAF1 having a known tumor-suppressive function is overexpressed and administered the FAF1 protein-loaded exosomes to various tumor models in which pancreatic cancer cells (MIA PaCa-2), lung cancer cells (A549), colon cancer cells (HCT 116), liver cancer cells (Hep3B), breast cancer cells (MDA-MB-231), kidney cancer cells (Caki-1) and cervical cancer cell (HeLa cell) are transplanted into nude mice through intratumoral injection. The administration exhibited tumor growth inhibitory effects remarkably greater than those of a control group not treated with FAF1.
Absstract of: WO2024130106A1
A transcutaneous composition for treating an oxidative skin disorder of a mammal in need is disclosed containing an effective oxidative skin disorder treating amount of covalently-substituted oxidi zed activated charcoal (OACs) nanoparticles dissolved or dispersed in an aqueous composition containing a thickening agent providing a viscosity of about 1000 to about 20,000 cps, and about 5 to about 20 wt % of a skin permeation enhancer. A substituent of the substituted OAC comprises an average of about 2 to about 5 polyethylene glycol ( PEG) chains covalently linked to each OAC, or an average of about 2 to about 5 metal ion chelating groups covalently linked to each OAC, or an average of about 2 to about 5 PEG chains and an average of about 2 to about 5 metal ion chelating groups linked to each OAC. A method of treating an oxidative skin disorder of a mammal is also disclosed.
Absstract of: US2025302899A1
The present disclosure provides for an oncolytic virus comprising an exogenous nucleic acid encoding for a polypeptide that acts as a CD47-SIRP-alpha immune checkpoint inhibitor. Oncolytic viruses optionally comprise a mutation or deletion of the gene expressing IFN-gamma. Compositions described herein are further described for use in the treatment of cancer.
Absstract of: WO2025259188A1
There is provided a compound comprising a structure represented by general formula (1) or an ionized form thereof for preparing lipid nanoparticles encapsulating a therapeutic, prophylactic and/or biological agent wherein R3 is optionally substituted alkylene, optionally substituted alkenylene, or optionally substituted alkynylene; R1, R2, R10, and R11 are each independently optionally substituted alkylene, optionally substituted alkenylene, optionally substituted alkynylene, or a single bond; R4, R5, R6, R7, R8, and R9 are each independently H, optionally substituted alkyl, optionally substituted alkenyl, or optionally substituted alkynyl; A1 and A2 are each independently a hydrophobic group or contains at least one of the groups defined above for R4 to R9, with the proviso that both A1 and A2 are not H at the same time; B1, B2, B3, and B4 are each independently -H, -ORa, -NRaRb, -SRa, - C(ORa)(Rb)(Rc), -C(SRa)(Rb)(Rc), or -C(NRaRb)(Rc)(Rd), where each of Ra, Rb, Rc, and Rd is independently H, optionally substituted alkyl, optionally substituted alkenyl, or optionally substituted alkynyl; w ≥ 0; x ≥ 1; and y ≥ 1.
Nº publicación: WO2025259802A2 18/12/2025
Applicant:
UNIV OF MASSACHUSETTS [US]
UNIVERSITY OF MASSACHUSETTS
Absstract of: WO2025259802A2
The present disclosure relates to compositions comprising a population of immunomodulatory lipid nanoparticles, as well as methods of using thereof to treat and/or prevent cancer (e.g., via local administration to the lymph nodes) and methods of using thereof to treat and/or prevent cancer metastasis in a subject.
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