Absstract of: WO2025006963A1
Provided herein are combinations comprising CRISPR/Cas systems, CtBP-interacting protein, and inhibitor of 53BP1 for use in enhancing homology-directed repair of CRISPR/Cas-mediated cleavage of a target DNA by an exogenous donor nucleic acid. Also provided are methods of using such combinations to make a targeted genetic modification in a cell by homology-directed repair of CRISPR/Cas-mediated cleavage at a target genomic locus in the cell.
Absstract of: WO2024228993A2
Suspension formulations of nanoparticles of clobetasol propionate are described. The suspensions can be used therapeutically to treat skin and ocular burns; to enhance wound healing; to prevent or reduce hypertrophic scarring/keloids; to treat allergic rhinitis/sinusitis, asthma, inner ear disorders including hearing loss, tinnitus, or vertigo, tenosynovitis, tendinitis, enthesitis or arthritis.
Absstract of: WO2024220742A1
Provided herein are lipid conjugated polyethyleneimine (PEI) compounds of Formula (I), which are useful to form delivery systems, such as lipid nanoparticles, for delivery of agents (e.g., nucleic acid molecules) to cells (e.g., endothelial cells). Also provided herein are methods for treating atherosclerosis, a cardiovascular disease, a pulmonary hypertension (e.g, pulmonary arterial hypertension or secondary pulmonary hypertension), a lung cancer, a lung metastasis, chronic obstructive pulmonary disease (COPD), bronchiolitis obliterans, bronchiolitis obliterans organizing pneumonia (BOOP), pulmonary fibrosis, or a fibrotic lung disease, comprising administering to a subject a pharmaceutical composition comprising lipid conjugated polyethyleneimine (PEI) compounds of Formula (I), an agent, and a pharmaceutically acceptable excipient.
Absstract of: US20260191799A1
The present disclosure relates to polymer nanoparticles of metabolites, a cell activity promotion method using same, and a cell activity promotion composition including same. In particular, the present disclosure relates to a method and a composition, for promoting, by the permeation of polymer nanoparticles of metabolites into cells, the activities of cells, for example, adhesion between cells or between tissues, hemostasis, wound healing promotion, hair root regeneration activity, and antibacterial activity.
Absstract of: US20260193172A1
0000 A cycloalkane-based lipid compound of Formula 1 and a pharmaceutically acceptable salt thereof are disclosed. The cycloalkane-based lipid compound is a compound having a form in which carbonyl-based substituents are bonded to the central structure of a cycloalkane. The cycloalkane-based lipid compound is used as an ionizable lipid, which is a component of a lipid nanoparticle for delivering a nucleic acid.
0000
Absstract of: US20260191798A1
A gene delivery system and applications thereof in the technical field of biological medicines that is particularly useful in the preparation of drugs for treatment of tumors are disclosed. The gene delivery system and applications relate to technology for inducing the differentiation of malignant tumor cells into mature cells, in which regulating the expression of HNF4 alpha protein in the malignant tumor cells using messenger ribonucleic acid, the malignant phenotype of the malignant solid tumor cells is inhibited, and the effective treatment of the malignant solid tumors is achieved. The gene delivery system and applications are therefore applicable to a method of preparing a drug for treating malignant solid tumors and to a method of treating a patient having a malignant solid tumor.
Absstract of: AU2025207205A1
The present application relates to nanoparticles comprising (a) an amphiphilic polymer; (b) a first lipid comprising a polar head group and a non-polar tail group; and (c) a peptide comprising a T cell epitope. Also disclosed are populations of said nanoparticles, processes for manufacturing said nanoparticles, precursors for manufacturing said nanoparticles, pharmaceutical compositions of said nanoparticles, and methods of treating a disease or disorder comprising administering said nanoparticles to a subject.
Absstract of: US20260193175A1
0000 The present disclosure discloses an alkanolamine multi-tailed lipid, a preparation method therefor, and a use thereof. The structural formulas of the lipid such as compound of Formula I, compound of Formula II or their stereoisomers, their tautomers, or their pharmaceutically acceptable salts:
0000
0000 The ionizable lipid compound of the present disclosure maintains nanoparticle stability and delivery efficiency while simplifying LNP composition, offering advantages of high efficacy and low toxicity. Even under neutral conditions, the ionizable lipid compound can still adsorb mRNA through hydrogen bonding and van der Waals interactions. The synthesis method for the diethanolamine multi-tailed ionizable lipid of the present disclosure is straightforward. The ionizable lipid can be prepared on a large scale via a few addition reaction steps, which is convenient for high-throughput material screening.
Absstract of: US20260191938A1
One aspect according to the present invention relates to: a gold nanozyme including glycol chitosan and gold particles; and a pharmaceutical composition for preventing or treating inflammatory bowel disease, the composition comprising the gold nanozyme. The gold nanozyme and the pharmaceutical composition comprising same according to one aspect of the present invention was found to inhibit the expression of inflammatory factors, inhibit the generation of reactive oxygen species (ROS) and reactive nitrogen species (RNS) inside cells, and reduce the production of nitric oxide (NO) inside cells. In addition, the gold nanozyme was found to inhibit the secretion of Hight Mobility Group Box 1 (HMGB1) when further including glycyrrhizin. In addition, the gold nanozyme and the composition comprising same were found to enable the recovery of the length, weight, etc., of damaged intestines, and thus can be used in the inflammatory bowel disease prevention and/or treatment industry/market.
Absstract of: US20260191937A1
0000 Disclosed are agents, compositions and methods for treating, reducing or inhibiting, or slowing the progression of, unwanted or undesirable immune responses including pro-inflammatory responses. More particularly, the present disclosure relates to a therapeutic agent, therapeutic combinations and their use in compositions and methods for inhibiting or reducing pro-inflammatory activity of immune cells such as antigen-presenting cells (APC), wherein the therapeutic agent comprises D-ribulose-5-phosphate (RL5P), a RL5P analog, a RL5P analog-producing agent and/or a RL5P-producing agent, such as one selected from 6-phosphogluconate dehydrogenase (6PGD) and a nucleic acid molecule from which 6PGD is producible, and wherein the therapeutic combination comprises the therapeutic agent and an HDAC7-producing agent.
Absstract of: US20260191956A1
The present disclosure provides compositions comprising modified immune cells or precursors thereof (e.g. T cells) comprising chimeric antigen receptors (CARs) along with modified cells comprising lipid nanoparticles (LNPs) comprising a nucleic acids encoding a truncated target antigens. Methods of treating disease (e.g. cancer) by administering said compositions to a subject in need thereof, are also provided.
Absstract of: US20260191929A1
The present invention relates to CD117 targeted LNP molecules for delivery of pro-apoptotic mRNA to hematopoietic stem cells (HSCs) and methods of use thereof for preconditioning a subject for hematopoietic stem cell therapy or to deplete the subject's HSC population for the treatment of a disease or disorder.
Absstract of: AU2024409393A1
The present disclosure provides a method for delivering a nucleic acid cargo to the liver of a subject, the method comprising administering to the subject a lipid nanoparticle encapsulating the nucleic acid and comprising a non-polar lipid. The lipid nanoparticle may exhibit a blebbed morphology, despite the absence or low levels of phospholipid. The disclosure further provides compositions for delivering nucleic acid cargo to the liver in order to treat various diseases, disorders and conditions in the subject.
Absstract of: AU2024406406A1
The present disclosure provides nitrogen-containing silicon ether ionizable lipid compounds and lipid nanoparticles including the ionizable lipid. The disclosure further relates to lipid nanoparticles including a provided ionizable lipid compound together with a phospholipid, e.g., a phospholipid that includes at least one unsaturated tail and a head group having a positively charged nitrogen. The provided materials are particularly beneficial in applications involving delivery of a nucleic acid. The disclosure also provides pharmaceutical compositions and methods including the provided ionizable lipids and/or lipid nanoparticles.
Absstract of: AU2024397320A1
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.
Absstract of: US20260191990A1
The present invention relates to compositions for effective delivery of gene editing agents to a target cell, as well as methods of use thereof for the treatment of a disease or disorder.
Absstract of: US20260191778A1
0000 Lipid nanoparticles (LNPs) containing particular cationic ionizable lipids with a biologically active polynucleotides (e.g., RNAs) are provided. In some aspects, the LNP complexes are provided as aerosols and/or dry powders, such as for delivery to the lungs. Methods of making and using such compositions are provided.
Absstract of: AU2024385239A1
The present disclosure relates to lipids and compositions thereof. In various aspects of the invention, the compositions are lipid nanoparticle compositions used to deliver various DNA molecules and/or therapeutic agents to selected targets, such as cells for gene delivery, and/or to prevent or treat diseases or disorders in a subject in need thereof.
Absstract of: AU2024384650A1
Provided herein are emulsion composition comprising a medium chain triglyceride (MCT), preferably tricaprilin. Also, described here are additional ingredients that make these emulsion compositions more suitable for oral intake, such as one or more emulsifiers, buffers such as phosphate buffers, as well as optionally glycerol and a sweetener and/or a flavoring agent. Described herein are methods of making and using these emulsions. The emulsions described may be used for oral administration of tricaprilin to treat and/or prevent various diseases or disorders.
Absstract of: AU2024390885A1
The present invention provides a composite molecule, a nucleic acid, a vector, a host cell, and a pharmaceutical composition, and uses of the composite molecule, the nucleic acid, the vector, the host cell, and the pharmaceutical composition in preparation of drugs for treating cancers. The composite molecule comprises an anti-tumor antibody domain, a linker, and pro-IL-15; the anti-tumor antibody domain is linked to the pro-IL-15 by means of the linker; the anti-tumor antibody domain is a complete antibody against an immune checkpoint molecule, a tumor antigen molecule or an immune activation molecule, or a nano antibody or an antigen binding fragment thereof; the linker is a polypeptide linker or a non-peptide linker; the pro-IL-15 is a fusion protein comprising IL-15, an IL-15Rα sushi domain, and a linker peptide, and optionally comprising an IL-15Rβ extracellular domain; and the IL-15Rβ extracellular domain, the IL-15, and the IL-15Rα sushi domain are linked by means of the linker peptide.
Absstract of: US20260191902A1
Zinc-based nanoclusters having: a zinc-based metal core covered over its entire surface with a mixed layer including histidine, acetate ions, and ascorbate ions, the metal core composed of zinc or a mixture of 80 to 99.99 wt % zinc and 0.01 to 20 wt % selenium; a spherical shape; a hydrodynamic diameter of 0.6 to 2.0 nm; a metal core diameter of 0.5 to 1.5 nm; a stability of 5 to 20 weeks in liquid form stored at 4° C.; a stability of at least 12 months in dry form stored at 4° C. under nitrogen; a shoulder in the UV-visible spectrum at 300±15 nm and a fluorescence spectrum with excitation wavelengths of 364±15 nm and emission wavelengths of 415±15 nm. Also, a method of preparing the zinc-based nanoclusters and their use for combating zinc deficiencies or zinc and selenium deficiencies.
Absstract of: US20260191793A1
Disclosed are exosomes comprising a Na+ taurocholate co-transporting polypeptide (NTCP) binding motif, such as HBV preS1 peptide (PSIP). Disclosed are methods of making exosomes that have been engineered to express a heterologous binding motif on the surface of the exosome, wherein the heterologous binding motif is a NTCP binding motif. Disclosed are methods of treating a subject infected with hepatitis B virus (HBV) comprising administering to the subject a therapeutically effective amount of an exosome, wherein the exosome comprises a NTCP binding motif and a therapeutic agent, thereby treating the HBV infection or any other liver diseases including liver cancer in the subject.
Absstract of: WO2026146172A1
The disclosure features a method of lyophilizing lipid nanoparticles including mixing a lyophilization buffer with a lipid nanoparticle formulation to form a lyophilization formulation and lyophilizing the lyophilization formulation to form lyophilized lipid nanoparticles, where the method is free of a buffer exchange process.
Absstract of: WO2026146887A1
The present invention relates to a polymeric nanoparticle for oral administration of physiologically active substances and a preparation method therefor and, more specifically, to a carrier or polymeric hydrogel for delivery of a physiologically active substance and a preparation method therefor, wherein the carrier or polymeric hydrogel comprises: polymeric micelles including a PEG-based block copolymer prepared by a reversible addition–fragmentation chain transfer (RAFT) radical polymerization method using a polymer that has a specific functional group at the chain end thereof and a boronic acid group introduced into the backbone thereof; and a physiologically active substance that can be encapsulated in the polymeric micelles, including a type 2 diabetes drug, insulin, an anti-obesity agent, a DPP-4 inhibitor, or an anticancer agent.
Nº publicación: WO2026147893A2 09/07/2026
Applicant:
SAPU NANO LTD [US]
SAPU NANO LIMITED
Absstract of: WO2026147893A2
This invention relates to compositions, and uses and methods thereof, with therapeutic deciparticles. Described herein are deciparticle (nanoparticle) compositions composed of an amphiphilic compound complexed with an auxiliary compound such as a biologically active molecule. Deciparticles of this invention are active drugs having potency for anti-tumor effects and for immunosuppression. This invention is also directed to amphiphilic compounds useful for making deciparticle complexes.