Absstract of: WO2025143163A1
The present inventors have found a carbamoyl lipid having a cyclic group in a side chain, which can form lipid nanoparticles, and have revealed that the lipid nanoparticles containing, as a constituent, the carbamoyl lipid having a cyclic group in a side chain according to the present invention can express a protein in astrocytes or hepatocytes. The lipid nanoparticles containing, as a constituent, the carbamoyl lipid having a cyclic group in a side chain according to the present invention contain a nucleic acid inside thereof and are expected as a component of a pharmaceutical composition useful for prevention and/or treatment of an astrocyte-related disease.
Absstract of: WO2025140634A1
Provided are an anti-CD276 nanobody, and a preparation method therefor and the use thereof. Specifically, further provided are a corresponding anti-CD276 antibody, a chimeric antigen receptor, a fusion protein, a recombinant protein, an antibody-drug conjugate, and an encoding nucleic acid, an expression vector, a host cell, etc. thereof. The anti-CD276 nanobody is high in affinity, simple in structure and easy to prepare, and the anti-CD276 nanobody-drug conjugate has a significant killing activity, thereby having application prospects in the fields such as tumor treatment and immunodetection.
Absstract of: WO2025144782A1
The present disclosure relates, in one aspect, to lipid nanoparticle (LNP) compositions comprising an agent that reduces or inhibits secretion of small extracellular vesicles (sEVs) and at least one mRNA. The present disclosure further relates to methods for treating, preventing, and/or ameliorating cancer in a subject. The present disclosure further relates to delivering a therapeutic agent to a tumor cell in a subject.
Absstract of: WO2025139283A1
A nano-composite hydrogel for radiosensitization after breast-conserving surgery for breast cancer and a preparation method therefor. The nano-composite hydrogel comprises a hydrogel loaded with mesoporus gold nanoparticles M@AuNPs. The preparation method is as follows: The starting materials include a modified chitosan solution, a modified hyaluronic acid solution, and M@AuNPs. M@AuNPs are dispersed in the modified hyaluronic acid solution and the modified chitosan solution is isovolumetrically mixed with the modified hyaluronic acid solution to give the hydrogel. An injectable hydrogel encapsulating nanoparticles. The injectable hydrogel has a structure uniformly encapsulating outer-layer modified gold nanoparticles, and is capable of implementing immunotherapy by means of the targeted sustained-release of therapeutic drugs and meanwhile achieving ray energy accumulation due to the higher atomic number of the gold nanoparticles, thereby effectively killing tumor cells at the tumor bed position. The strategy effectively reduces the radiation dose in the whole breast radiotherapy, and is beneficial to protecting normal tissues of the breast outside the tumor bed.
Absstract of: WO2025143892A1
The present invention relates to a composition for drug delivery, and a preparation method therefor, and, more specifically, to a composition for drug delivery, and a preparation method therefor, the composition having a form such that a drug is encapsulated inside a nanoparticle structure formed by a polymer and a cationic lipid with a specific structure.
Absstract of: WO2025143871A1
The present invention relates to a composition for drug delivery and a preparation method thereof and, more specifically, to: a composition for drug delivery in which a drug is encapsulated inside a nanoparticle structure formed by a specific polymer and a cationic compound; and a preparation method thereof.
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: WO2025140618A1
Provided are a lipid nanoparticle composition for delivering a nucleic acid drug, a preparation method therefor, and use thereof. The lipid nanoparticle composition comprises three lipid components: a steroid-cationic lipid compound, a neutral phospholipid, and a polyethylene glycol lipid, and the composition has relatively good stability and transfection efficiency. The lipid nanoparticle is used for delivering nucleic acid, such as mRNA, can efficiently and stably deliver the nucleic acid drug to a target cell or organ, and can induce a relatively high specific antibody response in an experimental animal, and the compound has a better safety profile.
Absstract of: CN119894503A
The present disclosure generally relates to lipid nanoparticle compositions comprising a nucleic acid, an ionizable polymer, a cationic lipid, a phospholipid, a sterol, and a PEG-lipid. Furthermore, the present disclosure relates generally to methods of treating or preventing a disease comprising administering to a subject in need thereof the lipid nanoparticle compositions described herein.
Absstract of: AU2023329918A1
Disclosed herein are lipid nanoparticles for the delivery of active agents, including nucleic acids, as well as methods of making using thereof.
Absstract of: WO2024044636A2
Compositions, methods, and kits are provided for treating infections and cancer with metallic nanoclusters. In particular, metallic nanoclusters having a size of less than 10 nm that are conjugated to adenosine triphosphate (ATP) or an analogue thereof can be used to eradicate a cell in a growth arrest phase such as infectious bacterial or fungal cells. Such nanoclusters can also induce endoplasmic reticulum stress and inhibit growth of cancerous cells. Additionally, such metallic nanoclusters can be used to inhibit a purinergic P2X7 receptor and FtsH protease.
Absstract of: AU2023329397A1
The present disclosure relates to PEGylated lipid compounds and pharmaceutically acceptable salts thereof. Such compounds are useful, for example, as constituent parts of lipid nanoparticle (LNP) formulations for delivery of various active agents. The present disclosure further provides LNPs comprising a disclosed compound. Also provided herein are methods of preparing such PEGylated lipid compounds, as well as pharmaceutical compositions comprising an LNP and an active agent; and methods of use thereof.
Absstract of: AU2023375263A1
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: CN119816512A
The present invention relates to an engineered disulfide bond linked ferritin assembly comprising at least one modified ferritin subunit wherein the at least one modified ferritin subunit comprises the amino acid sequence as set forth in SEQ ID NO: 1 and comprises; i) a F116H substitution, and zero or more amino acid substitutions at one or more positions selected from the group comprising E65, E128, E131 and D138 of SEQ ID NO: 1, and ii) a Cys substitution at two or more positions selected from the group comprising G37, L53, R66, G67, A74, A117 and A152 of SEQ ID NO: 1. The invention also relates to the use and preparation thereof.
Absstract of: EP4578448A1
An object is to provide a technique that improves the poor solubility of a compound of formula (1) in water and further suppresses the cytokine excessive release action and bone marrow toxicity of the compound of formula (1). This object is achieved by a complex comprising a modified polysaccharide containing a hydrophobic group, and a compound represented by formula (1).
Absstract of: WO2024042236A1
Provided are stabilized lipid nanoparticle (LNP)/lipidoid nanoparticle (LiNP) formulations and LNP/LiNP suspensions, uses thereof and uses in method of treatment based on the findings that the addition of the surfactant to the formulations or suspensions avoids aggregation, allowing a surprisingly long shelf life and extended stability to shaking. Said reduction of aggregation according to the invention results in a reduction of side effects of the formulations and suspension of the invention, specifically a reduction of side effects caused by vaccines formulations and anticancer formulations comprising LNPs and/or LiNPs.
Absstract of: AU2023330867A1
The present disclosure relates to the field of vaccines and binding molecules, as well as preparations and methods of their use in the treatment and/or prevention of disease. Described are vaccines and binding molecules, compositions containing the same, and uses thereof for treating or preventing coronavirus infections, including multivalent mRNA and nanoparticle vaccines.
Absstract of: WO2024044147A1
Provided herein are methods for purifying an ionizable amino lipid (IAL) composition comprising impurities that may react with polynucleotides, such as mRNA. Methods for purifying IAL compositions comprise one or more scavenging-removal steps to selectively remove reactive impurities.
Absstract of: US2024173262A1
Provided herein are pharmaceutical dosage forms for delivering peptide therapeutics to a mucosal surface. The pharmaceutical dosage forms comprise a mucoadhesive layer; a peptide loading layer; and a water impermeable layer, wherein the peptide loading layer is between the mucoadhesive layer and the water impermeable layer, such that the water impermeable layer facilitates unidirectional movement of the encapsulated peptide through the mucoadhesive layer and to the target mucosal surface. The pharmaceutical dosage form is suitable for delivery of the encapsulated peptide therapeutic to buccal mucosa; sublingual mucosa; palate mucosa; and tongue mucosa. The peptide therapeutic may be an insulin; an insulin derivative; an insulin analog; a pre-insulin; a pro-drug of insulin; a glucagon-like peptide 1 (GLP-1); a GLP-1 analog; a pre- GLP-1; a pro-drug of GLP-1; or a combination thereof and may be suitable for the treatment of diabetes.
Absstract of: WO2024041744A1
Combination therapies for use in the treatment of brain tumours, particularly glioma and embryonal brain tumours. Disclosed herein are pharmaceutical compositions comprising a complex of Panobinostat and a cyclodextrin, a second pharmaceutical agent, and a pharmaceutically acceptable excipient, diluent or carrier, in which the second pharmaceutical agent is Niclosamide, Dasatinib or Duvelisib. Associated methods of treatment and use are further disclosed.
Absstract of: AU2023406947A1
The present application discloses modified single-stranded DNA molecules, as well as their cell-free methods of synthesis and their use as therapeutic agents.
Absstract of: AU2023374051A1
The present disclosure features a method of preparing a population of payload-associated cell complexes (PACCs), as well as related methods of use thereof.
Absstract of: WO2024100137A1
Non-human animal-derived cell composition comprising a plurality of non-human animal- derived cells, wherein the cells comprise at least one exogenous supplement comprised in a carrier selected from the group consisting of nanocarriers, nanoparticles, micelles, liposomes or vesicles is described. The cell composition is characterized by having improved organoleptic properties, such as an improved flavour, colour, texture or mouthfeel and/or having a meat like flavour. Methods of producing the same and comestibles comprising the same are also described.
Absstract of: AU2023378958A1
The present invention provides delivery system compositions comprising self- assembling lipid nanoparticles for targeted delivery of therapeutic or diagnostic agents to target cells. The particles are non-covalently attached to a lipidated antibody or antibody fragment which comprises an antibody or antibody fragment attached, via a peptide linker, to a lipidated peptide portion, wherein the antibody or antibody fragment is at the distal end from the nanoparticle.
Nº publicación: IL320672A 01/07/2025
Applicant:
REGENERON PHARMACEUTICALS INC [US]
HAINES JEFFERY [US]
CROSBY KEITH [US]
YANG YAN [US]
DALY CHRISTOPHER [US]
OLSON WILLIAM [US]
HUANG TAMMY T [US]
DUDGEON DREW [US]
BABB ROBERT [US]
REGENERON PHARMACEUTICALS INC,
HAINES Jeffery,
CROSBY Keith,
YANG Yan,
DALY Christopher,
OLSON William,
HUANG Tammy T,
DUDGEON Drew,
BABB Robert
Absstract of: AU2023379457A1
The present invention provides, in part, protein-drug conjugates comprising an anti-fibroblast growth factor receptor 3 (FGFR3) (e.g., human FGFR3) antigen-binding protein (e.g., scFv, Fab) conjugated to a molecular cargo (e.g., polynucleotides, polypeptides, liposomes or lipid nanoparticles) for delivery of the molecular cargo to a targeted tissue (e.g., brain). Methods for treating various diseases or disorders, such as neurological diseases, with the conjugates are provided.