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相同組換え修復を増加させるための方法及び組成物

Publication No.:  JP2026522927A 09/07/2026
Applicant: 
リジェネロン・ファーマシューティカルズ・インコーポレイテッド
JP_2026522927_A

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.

コルチコステロイドナノ懸濁液及びその使用

Publication No.:  JP2026522788A 09/07/2026
Applicant: 
エイマックスセラピューティクス,インコーポレイテッド
JP_2026522788_A

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.

コンジュゲートしたポリエチレンイミン化合物及び組成物、ならびにアテローム性動脈硬化症、心血管疾患、及び肺疾患の処置のためのそれらの使用

Publication No.:  JP2026522770A 09/07/2026
Applicant: 
バソアールエックス,インク.
JP_2026522770_A

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.

POLYMER NANOPARTICLES OF METABOLITES AND USE THEREOF

Publication No.:  US20260191799A1 09/07/2026
Applicant: 
POSTECH RES AND BUSINESS DEVELOPMENT FOUNDATION [KR]
CJ CHEILJEDANG CORP [KR]
POSTECH Research and Business Development Foundation
CJ CHEILJEDANG CORPORATION
US_20260191799_A1

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.

CYCLOALKANE-BASED LIPID COMPOUND FOR DELIVERING NUCLEIC ACIDS, AND LIPID NANOPARTICLES COMPRISING SAME

Publication No.:  US20260193172A1 09/07/2026
Applicant: 
GREEN CROSS CORP [KR]
GREEN CROSS CORPORATION
US_20260193172_A1

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

GENE DELIVERY SYSTEM AND APPLICATION THEREOF IN PREPARATION OF DRUGS FOR TREATMENT OF TUMORS

Publication No.:  US20260191798A1 09/07/2026
Applicant: 
SHANGHAI CELL DIFF MEDICINE LTD [CN]
SHANGHAI CELL DIFF MEDICINE LTD.
US_20260191798_A1

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.

NANOPARTICLES COMPRISING AMPHIPHILIC POLYMERS AND LIPIDS FOR THE TARGETED DELIVERY OF ANTIGENS

Publication No.:  AU2025207205A1 09/07/2026
Applicant: 
TOPAS THERAPEUTICS GMBH
TOPAS THERAPEUTICS GMBH
AU_2025207205_PA

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.

ALKANOLAMINE MULTI-TAILED LIPID, PREPARATION METHOD THEREFOR, AND USE THEREOF

Publication No.:  US20260193175A1 09/07/2026
Applicant: 
SOUTH CHINA UNIV OF TECHNOLOGY [CN]
South China University of Technology
US_20260193175_A1

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.

GOLD NANOZYME COMPRISING GLYCOL CHITOSAN AND GOLD PARTICLES, AND PHARMACEUTICAL COMPOSITION FOR PREVENTING OR TREATING INFLAMMATORY BOWEL DISEASE COMPRISING SAME

Publication No.:  US20260191938A1 09/07/2026
Applicant: 
IUCF HYU INDUSTRY UNIV COOPERATION FOUNDATION HANYANG UNIV [KR]
IUCF-HYU (INDUSTRY-UNIVERSITY COOPERATION FOUNDATION HANYANG UNIVERSITY)
US_20260191938_A1

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.

AGENTS, COMPOSITIONS AND METHODS FOR MODULATING IMMUNITY

Publication No.:  US20260191937A1 09/07/2026
Applicant: 
THE UNIV OF QUEENSLAND [AU]
The University of Queensland
US_20260191937_A1

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.

IN VIVO EXPANSION OF CAR T CELLS AND DELIVERY OF CAR TARGET ANTIGEN TO TUMORS USING MRNA LIPID NANOPARTICLES

Publication No.:  US20260191956A1 09/07/2026
Applicant: 
THE TRUSTEES OF THE UNIV OF PENNSYLVANIA [US]
The Trustees of the University of Pennsylvania
US_20260191956_A1

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.

HSC targeted LNP for delivery of pro-apoptotic mRNA and methods of use thereof

Publication No.:  US20260191929A1 09/07/2026
Applicant: 
THE TRUSTEES OF THE UNIV OF PENNSYLVANIA [US]
THE CHILDRENS HOSPITAL OF PHILADELPHIA [US]
The Trustees of the University of Pennsylvania
The Children's Hospital of Philadelphia
US_20260191929_A1

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.

LIPID NANOPARTICLES HAVING NON-POLAR LIPIDS FOR NUCLEIC ACID DELIVERY TO THE LIVER

Publication No.:  AU2024409393A1 09/07/2026
Applicant: 
NANOVATION THERAPEUTICS INC
NANOVATION THERAPEUTICS INC.
AU_2024409393_A1

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.

IONIZABLE LIPIDS SUITABLE FOR LIPID NANOPARTICLES

Publication No.:  AU2024406406A1 09/07/2026
Applicant: 
GENEVANT SCIENCES GMBH
GENEVANT SCIENCES GMBH
AU_2024406406_PA

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.

STIMULI-RESPONSIVE STING-ACTIVATING POLYMER COMPOSITIONS AND METHODS OF USE

Publication No.:  AU2024397320A1 09/07/2026
Applicant: 
THE BOARD OF REGENTS OF THE UNIV OF TEXAS SYSTEM
THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM
AU_2024397320_PA

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.

Targeted Delivery of Gene Editing Constructs and Methods of use Thereof

Publication No.:  US20260191990A1 09/07/2026
Applicant: 
THE TRUSTEES OF THE UNIV OF PENNSYLVANIA [US]
The Trustees of the University of Pennsylvania
US_20260191990_A1

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.

AEROSOLIZED LIPID NANOPARTICLES AND USES THEREOF

Publication No.:  US20260191778A1 09/07/2026
Applicant: 
BOARD OF REGENTS THE UNIV OF TEXAS SYSTEM [US]
Board of Regents, The University of Texas System
US_20260191778_A1

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.

LIPID NANOPARTICLES AND METHODS OF USE FOR DNA DELIVERY

Publication No.:  AU2024385239A1 09/07/2026
Applicant: 
THE WISTAR INST OF ANATOMY AND BIOLOGY
THE TRUSTEES OF THE UNIV OF PENNSYLVANIA
THE WISTAR INSTITUTE OF ANATOMY AND BIOLOGY
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
AU_2024385239_PA

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.

FORMULATIONS OF LIPID NANO EMULSIONS WITH CONTROLLED PHARMACOKINETIC PROFILES AND METHODS OF MAKING AND USING THE SAME

Publication No.:  AU2024384650A1 09/07/2026
Applicant: 
CERECIN AUSTRALIA PTY LTD
CERECIN AUSTRALIA PTY LIMITED
AU_2024384650_PA

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.

BIFUNCTIONAL COMPOSITE MOLECULE OF ANTI-TUMOR ANTIBODY AND INTERLEUKIN-15 PRECURSOR, AND USE OF BIFUNCTIONAL COMPOSITE MOLECULE

Publication No.:  AU2024390885A1 09/07/2026
Applicant: 
CHANGPING NAT LABORATORY
CHANGPING NATIONAL LABORATORY
AU_2024390885_PA

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.

ZINC-BASED NANOCLUSTERS, METHODS FOR OBTAINING SAME AND USES THEREOF FOR COMBATTING ZINC DEFICIENCIES

Publication No.:  US20260191902A1 09/07/2026
Applicant: 
UNIV DE LORRAINE [FR]
CENTRE HOSPITALIER REGIONAL UNIV DE NANCY [FR]
INSTITUT NATIONAL DE LA SANTE ET DE LA RECH MEDICALE [FR]
Universit\u00E9 de Lorraine
CENTRE HOSPITALIER REGIONAL UNIVERSITAIRE DE NANCY
INSTITUT NATIONAL DE LA SANT\u00C9 ET DE LA RECHERCHE M\u00C9DICALE
US_20260191902_A1

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.

HBV SPECIFIC EXOSOMES AND USES THEREOF

Publication No.:  US20260191793A1 09/07/2026
Applicant: 
THE US GOV AS REPRESENTED BY THE DEPARTMENT OF VETERANS AFFAIRS [US]
EAST TENNESSEE STATE UNIV RESEARCH CORPORATION [US]
The United States Government as represented by the Department of Veterans Affairs
EAST TENNESSEE STATE UNIVERSITY RESEARCH CORPORATION
US_20260191793_A1

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.

LIPID NANOPARTICLE FORMULATIONS AND METHODS OF PREPARING THEREOF

Publication No.:  WO2026146172A1 09/07/2026
Applicant: 
GLOBAL LIFE SCIENCES SOLUTIONS CANADA ULC [CA]
GLOBAL LIFE SCIENCES SOLUTIONS OPERATIONS UK LTD [GB]
GLOBAL LIFE SCIENCES SOLUTIONS CANADA ULC
GLOBAL LIFE SCIENCES SOLUTIONS OPERATIONS UK LIMITED
WO_2026146172_A1

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.

POLYMERIC NANOPARTICLES FOR ORAL ADMINISTRATION AND DELIVERY OF PHYSIOLOGICALLY ACTIVE SUBSTANCES AND PREPARATION METHOD THEREFOR

Publication No.:  WO2026146887A1 09/07/2026
Applicant: 
KANG IN SOO [KR]
\uAC15\uC778\uC218
WO_2026146887_A1

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.

DECIPARTICLE ANTI-CANCER DRUGS

Nº publicación: WO2026147893A2 09/07/2026

Applicant:

SAPU NANO LTD [US]
SAPU NANO LIMITED

WO_2026147893_A2

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.

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