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PREPARATION OF HIGHLY CONCENTRATED MRNA

Resumen de: US2025340586A1

Provided herein are compositions of highly concentrated mRNA and related methods for preparation and use of the compositions as mRNA process intermediates in the synthesis of therapeutic and prophylactic mRNA formulations.

LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS

Resumen de: US2024417370A1

Compounds are provided having the following Structure (I):or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein R1, R1, L1, L2, L2a, L2b, and A are as defined herein. Use of the compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided.

IONIZABLE CATIONIC LIPIDS FOR RNA DELIVERY

Resumen de: AU2024268834A1

The present disclosure describes compounds of Formula (I) and pharmaceutically acceptable salts thereof:

LIPID NANOPARTICLE (LNP) DELIVERY SYSTEMS AND FORMULATIONS

Resumen de: AU2024259356A1

The present disclosure describes compositions, nanoparticles (such as lipid nanoparticles), and/or lipid nanoparticle compositions and methods of their use.

NANOPARTICLES OF MYCOPHENOLIC ACID PRODRUG MOLECULES AND THERAPEUTIC USE THEREOF

Resumen de: AU2024255212A1

A nanoparticle is described, which comprises a plurality of therapeutic molecules arranged in the form of a spherical micelle, suitable for the localized delivery and release of mycophenolic acid, and therefore effective in the treatment of autoimmune diseases, fibrotic diseases and/or organ rejection diseases, in particular of the lungs. A pharmaceutical composition comprising the nanoparticle in a pharmaceutically acceptable vehicle, and a method for manufacturing said nanoparticle are also described.

RNA DELIVERY VEHICLE

Resumen de: AU2024250317A1

The present disclosure relates to an RNA delivery vehicle comprising lipid nanoparticles comprising the RNA therein, and a RNA-binding protein or peptide coated on the exterior surface of the lipid nanoparticle, and uses thereof.

Nanoparticle compositions for sustained therapy

Resumen de: AU2025237926A1

This disclosure provides compositions and methods for promoting the formation, expansion and recruitment of TR1 cells and/or B cells in an antigen-specific manner and treating diseases and disorders in a subject in need thereof. This disclosure provides compositions and methods for promoting the formation, expansion and recruitment of TR1 cells and/or B cells in an antigen-specific manner and treating diseases and disorders in a subject in need thereof. ep h i s d i s c l o s u r e p r o v i d e s c o m p o s i t i o n s a n d m e t h o d s f o r p r o m o t i n g t h e f o r m a t i o n , e x p a n s i o n e p a n d r e c r u i t m e n t o f c e l l s a n d o r c e l l s i n a n a n t i g e n - s p e c i f i c m a n n e r a n d t r e a t i n g d i s e a s e s a n d d i s o r d e r s i n a s u b j e c t i n n e e d t h e r e o f

Long-acting spleen-targeting cationic lipid compound comprising benzene ring structure, composition comprising same, and use thereof

Resumen de: AU2025202600A1

The present disclosure relates to the field of medicine, specifically to a long-acting spleen- targeting cationic lipid compound comprising a benzene ring structure, a composition comprising the same, and a use thereof. More specifically, the present disclosure provides a compound of formula (I), or an N-oxide thereof, a solvate thereof, a pharmaceutically acceptable salt thereof, or a stereoisomer thereof. The present disclosure further provides a composition comprising the aforementioned compound and a use thereof in delivering therapeutic or prophylactic agents. The present disclosure relates to the field of medicine, specifically to a long-acting spleen- targeting cationic lipid compound comprising a benzene ring structure, a composition comprising the same, and a use thereof. More specifically, the present disclosure provides a compound of formula (I), or an N-oxide thereof, a solvate thereof, a pharmaceutically acceptable salt thereof, or a stereoisomer thereof. The present disclosure further provides a composition comprising the aforementioned compound and a use thereof in delivering therapeutic or prophylactic agents. G1 G2-M1-R1NHN R3 G3-M2-R2 pr h e p r e s e n t d i s c l o s u r e r e l a t e s t o t h e f i e l d o f m e d i c i n e , s p e c i f i c a l l y t o a l o n g - a c t i n g s p l e e n - p r t a r g e t i n g c a t i o n i c l i p i d c o m p o u n d c o m p r i s i n g a b e n z e n e r i n g s t r u c t u r e , a c o m p o s i t i o n c o m p r i s

NANOBODY TARGETING BCMA, CHIMERIC ANTIGEN RECEPTOR AND USE THEREOF

Resumen de: EP4644418A1

Provided are a nanobody targeting BCMA and a chimeric antigen receptor thereof. The nanobody comprises VHH01 and VHH02, and has strong specificity and high affinity for BCMA. A constructed CAR-T cell has high specific killing efficiency on a BCMA positive cell line, and can be used for developing an immunotherapeutic drug targeting BCMA.

LIPID COMPOUND AND LIPID NANOPARTICLE COMPOSITION

Resumen de: EP4644379A1

The present application disclosed a compound of formula (I) and a salt and a stereoisomer thereof, wherein each variable is as defined in the specification. The present application also disclosed a nanoparticle composition comprising the compound or a salt or a stereoisomer thereof, and the use of the nanoparticle composition for delivering active agents.

ナノデリバリーシステムならびにその治療および診断上の使用

Resumen de: US2023120902A1

The present invention provides a generic platform for delivering molecules with low blood-brain barrier (BBB) penetration into the brain. The nano-delivery system is based on a core nanoparticle which is conjugated through a first polymeric linker to a brain-internalizing transporter moiety, and is further conjugated to a second polymeric linker bound to an active agent selected rom a biologically active molecule or a labeling molecule. Further provided is a process for preparation of the nano-delivery system. The present invention further provides pharmaceutical compositions comprising the nano-delivery system and its use in therapeutic and/or diagnostic methods.

NUCLEIC ACID DELIVERY CARRIER, PREPARATION METHOD THEREFOR, AND USE THEREOF

Resumen de: EP4643850A1

A lipid nanoparticle composition, a preparation method therefor, and use thereof in nucleic acid delivery. An mRNA drug or vaccine can be prepared on the basis of the lipid nanoparticle composition. The starting materials of the lipid nanoparticle composition comprise an ionizable cationic lipid, an auxiliary phospholipid, a sterol compound, a lipid polyethylene glycol conjugate, and a buffer.

セクレトーム含有組成物の生成並びにそれを使用及び分析する方法

Resumen de: CN120225659A

The present disclosure provides methods for producing and/or purifying secretory groups, extracellular vesicles, and fractions thereof from progenitor cells; also provided are compositions containing such secretion-producing groups, extracellular vesicles, and fractions thereof. The present disclosure also provides methods of analyzing the activity, function, and potency of these secretory groups, extracellular vesicles, and fractions thereof. The present disclosure also relates to therapeutic uses of secretograms, extracellular vesicles and fractions thereof. The present disclosure also relates to scalable culture regimens that comply with good production specifications (GMP) for issuing clinically available secretory groups.

エナンチオ特異的カチオン性脂質と配合された組換えタンパク質組成物およびその使用

Resumen de: AU2023365611A1

Provided herein are vaccine compositions including recombinant protein antigens derived from computationally optimized broadly reactive influenza antigen (COBRA) proteins and an immunomodulator, and methods of use thereof. The vaccine compositions include one or more COBRA proteins, and the immunomodulator is a cationic lipid. The cationic lipid includes R-DOTAP. The methods of use of the vaccine compositions includes methods of inducing a humoral immune response against influenza viruses, methods of inducing polyfunctional CD8+ and CD4+ effector T cells against influenza viruses, methods of inducing memory T cells against influenza viruses, methods of enhancing immunity against influenza viruses, and methods of inducing balanced Th1/Th2 immune response against influenza viruses in a subject.

サルベコウイルスを標的とするマルタボディ構築物、組成物および方法

Resumen de: WO2024082067A1

Described herein is a fusion polypeptide comprising a sarbecovirus binding moiety linked to a nanocage monomer or subunit thereof, wherein the sarbecovirus binding moiety is capable of binding to SARS-CoV-2 and at least one sarbecovirus other than SARS-CoV-2. Also described are methods for treating and/or preventing sarbecovirus infection and/or a sarbecovirus-associated condition.

MODULAR LIPID COMPOUNDS AND TWO- TO THREE-COMPONENT LIPID NANOPARTICLE COMPOSITIONS

Resumen de: US2024261223A1

Novel delivery nanoparticles composes of two-, or three-component lipid compounds. Compositions comprising such lipid compounds, and related methods of their use are disclosed. Nanoparticle compositions include at least one novel modular lipid as well as additional lipids such as ionizable lipids, and phospholipids. Nanoparticle compositions further including biologically active agents, such as siRNA or mRNA, are useful in the delivery of said biologically active agents to subjects in need thereof.

LONG-ACTING SPLEEN-TARGETING CATIONIC LIPID COMPOUND COMPRISING BENZENE RING STRUCTURE, COMPOSITION COMPRISING SAME, AND USE THEREOF

Resumen de: EP4644360A2

The present disclosure relates to the field of medicine, specifically to a long-acting spleentargeting cationic lipid compound comprising a benzene ring structure, a composition comprising the same, and a use thereof. More specifically, the present disclosure provides a compound of formula (I), or an N-oxide thereof, a solvate thereof, a pharmaceutically acceptable salt thereof, or a stereoisomer thereof. The present disclosure further provides a composition comprising the aforementioned compound and a use thereof in delivering therapeutic or prophylactic agents.

NOVEL PORIN BCP34, AND MUTANT THEREOF AND USE THEREOF

Resumen de: EP4644407A1

The present invention provides a novel porin BCP34, and a mutant thereof and a use thereof. Specifically, the wild-type porin BCP34 is a nonamer protein, and has a monomer of which the amino acid sequence is SEQ ID NO: 1; and the mutant of the monomer of the porin BCP34 comprises an amino acid sequence having at least 70%, 75%, 80%, 85%, 90%, 95%, or at least 99% identity to SEQ ID NO:1, and an amino acid sequence having mutations at one or more positions in the sensor region, a transmembrane region, the entrance, barrel inner wall, and the exit of the monomer of the wild-type porin BCP34. The wild-type porin BCP34 of the present invention has the capability of being applied to nanopore sequencing, and the mutant of the porin BCP34 obtained by optimization has higher library capture capability and nanopore sequencing accuracy.

NANOSTRUCTURED AQUEOUS FORMULATION OF PROPOLIS EXTRACT AND THE USES THEREOF

Resumen de: EP4643865A1

The present invention relates to a nanostructured aqueous formulation containing a high content of propolis extract, in particular green or Brazilian red propolis extract, for use in pharmaceutical, food, cosmetic and veterinary formulations. The bioactives present in the propolis extract are responsible for its antioxidant, anti-inflammatory, antitumor and antiviral activities, which are enhanced and protected by the encapsulation of the extract in the lipid nanostructure.

VACCINE COMPOSITION COMPRISING GOLD-NANOPARTICLE-CARRIER HAVING DOUBLE-STRANDED DNA BOUND THERETO

Resumen de: EP4643875A1

The present invention relates to a vaccine composition including double-stranded DNA delivered into cells via gold nanoparticles, and more particularly, to a vaccine composition characterized in that the double-stranded DNA is derived from a viral, bacterial or cancer gene and expresses an antigen to induce an immune response.

LIPID NANOPARTICLE FORMULATION COMPRISING IONIZED LIPIDS WITH BRANCHED STRUCTURE, AND USE THEREOF

Resumen de: EP4644361A1

The present invention relates to: ionized lipids comprising lipids with a branched structure; a lipid nanoparticle formulation using same; and use thereof. The ionized lipids of the present invention are a biodegradable lipid material with a lipid structure in which a heteroamine structure is branched, and the lipid nanoparticles using the ionized lipids can deliver a nucleic acid drug and the like with high efficiency, and thus can be effectively used in related technical fields such as mRNA vaccines and therapeutic agents.

4-AMINOPIPERIDINE COMPOUND, LIPID NANOPARTICLES THEREOF, AND PHARMACEUTICAL COMPOSITION CONTAINING SAME

Resumen de: EP4644371A1

The present invention addresses the problem of providing: a compound that is a 4-aminopiperidine lipid; lipid nanoparticles containing the lipid; and a pharmaceutical composition useful for nucleic acid medicines and the like. The present inventors have discovered a compound that is a 4-aminopiperidine lipid or a salt thereof, examined lipid nanoparticles that have the potential of being formed into various pharmaceutical compositions, and revealed that the lipid that is the compound according to the present invention or a salt thereof can form lipid nanoparticles, and moreover, lipid nanoparticles in which nucleic acid is encapsulated (i.e., nucleic acid lipid nanoparticles). In addition, the nucleic acid lipid nanoparticles containing the lipid according to the present invention are expected to serve as a component of pharmaceutical compositions useful for prevention and/or treatment of astrocyte-related diseases.

LIPID COMPOUND FOR GENE DELIVERY AND LIPID NANOPARTICLE COMPRISING SAME

Resumen de: EP4644377A1

Disclosed herein are a lipid compound comprising an isonitrile or a carboxylic acid terminal group, and a preparation method therefor and the use thereof in the preparation of a lipid compound that can be used for gene delivery. Also disclosed herein are a lipid compound that can be used for gene delivery, and a preparation method therefor and the use thereof in gene delivery. Further disclosed herein are a liposome and a lipid nanoparticle comprising the lipid compound that can be used for gene delivery, and a gene delivery composition comprising the lipid compound, the liposome, or the lipid nanoparticle. The lipid compound, the liposome, the lipid nanoparticle and the gene delivery composition herein that can be used for gene delivery enable efficient compounding, protection, and intracellular and targeted delivery and release of biomolecules, such as nucleic acids in tissues and organs in vitro and in vivo.

POLYNUCLEOTIDE MOLECULE FOR PREVENTING OR TREATING HPV INFECTION-RELATED DISEASES

Resumen de: EP4644552A1

The present application relates to a polynucleotide molecule that can be used for preventing or treatment HPV infection-related diseases, and a pharmaceutical product, a pharmaceutical composition, or an mRNA vaccine comprising said polynucleotide.

一种肿瘤靶向的生物膜包覆纳米自组装体及其应用

Nº publicación: CN120884712A 04/11/2025

Solicitante:

安徽医科大学