Alerta

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.

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

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

Kompositpartikel

Resumen de: DE102024112307A1

Die Erfindung betrifft Kompositpartikel, umfassend einen Kern aus einer hydrophoben Phase, welche auch vernetzt sein kann, wobei der Kern an der Oberfläche geladen ist. Auf der Oberfläche des Kerns ist eine Lipiddoppelschicht angeordnet, welche den Kern umschließt. Diese Lipiddoppelschicht umfasst mindestens ein Lipid mit einer funktionellen Gruppe. Entsprechend funktionalisierte Kompositpartikel können zur Aktivierung von T-Zellen eingesetzt werden.

METHOD OF USING LIPID NANOPARTICLES FOR INTRAMUSCULAR DELIVERY

Resumen de: WO2025231114A1

Compounds are provided having the following Formula (I): or a pharmaceutically acceptable salt, tautomer, or stereoisomer thereof, wherein R1, R2, R3, and G1 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.

LIPIDS NANOPARTICLE FORMULATIONS SUITABLE FOR LUNG TARGETING

Resumen de: WO2025228979A1

The present invention provides lipids and lipid nanoparticle formulations comprising these lipids, alone or in combination with other lipids. These lipid nanoparticles may be formulated with nucleic acids to facilitate their intracellular delivery both in vitro and for in vivo therapeutic applications. The present formulation is specifically directed to compositions comprising ionizable lipids and a lipid, which comprises a quaternary ammonium group as a permanently charged lipid.

COMPOSITE PARTICLES

Resumen de: WO2025228859A1

The invention relates to composite particles comprising a core made of a hydrophobic phase, which can also be cross-linked, wherein the core is charged on the surface. A lipid double layer which surrounds the core is located on the surface of the core. Said lipid double layer comprises at least one lipid with a functional group. Correspondingly functionalized composite particles can be used to activate T cells.

IN VIVO GENE EDITING WITH CRISPR SYSTEMS

Resumen de: WO2025231432A1

The present disclosure provides methods and compositions for therapeutic use, where the methods and compositions include the use of the CRISPR complex. The CRISPR complex is used to perform therapeutic genome editing in vivo in somatic cells of an organism.

USE OF AMINOTHIOLESTER COMPOUNDS IN COMBINATION WITH HYPOMETHYLATING AGENTS (HMA) FOR THE PREVENTION AND TREATMENT OF CANCER

Resumen de: WO2025229179A1

The present invention relates to the combination of a compound of formula (I) as described herein with an hypomethylating agent (HMA) for use for the prevention and/ or treatment of cancer, in particular acute myeloid leukemia (AML), and AML-related myeloid diseases. The present invention further relates to a pharmaceutical composition comprising a compound of formula (I) as described herein with an HMA.

PARTICLES, COMPOSITIONS AND METHODS

Resumen de: WO2025228975A1

The present disclosure relates generally to nucleic acid-lipid particles, in particular functionalised nucleic acid-lipid particles, methods for producing them, and to pharmaceutical compositions containing them and their uses in medicine.

METHOD FOR MANUFACTURING LIPID NANOPARTICLES

Resumen de: WO2025228668A1

Disclosed is an impingement method for manufacturing lipid nanoparticles (LNPs) using a jet impingement reactor having a substantially spheroidal reaction chamber and/or non-retracted nozzles, an organic lipid solution having a lipid concentration of at least 50 mg/mL and an aqueous solution, such as a buffered aqueous solution comprising an active ingredient, for example an oligonucleotide or polynucleotide. Also provided is a dispersion of LNPs which is obtainable by the method.

ＲＳＶ－ＦをコードするＲＮＡ分子およびそれを含有するワクチン

Resumen de: JP2025166811A

【課題】肺炎および気管支炎を含む、ＲＳＶ感染誘導性の急性の気道の病気の処置および／または予防のための組成物を提供する。【解決手段】ＲＳＶに対する免疫応答を誘導するための組成物であって、呼吸器合胞体ウイルス（ＲＳＶ）融合タンパク質Ｆ（Ｆ）ポリペプチドをコードする少なくとも１つのオープンリーディングフレームを含むＲＮＡ分子を含む組成物が提供される。本開示は、脂質ナノ粒子中に製剤化されたＲＮＡ分子（ＲＮＡ－ＬＮＰ）を含む組成物にさらに関する。本開示は、肺炎および気管支炎を含む、ＲＳＶ感染誘導性の急性の気道の病気の処置および／または予防のための、ＲＮＡ分子、ＲＮＡ－ＬＮＰ、および組成物の使用にさらに関する。【選択図】なし

SINGLE-AND MULTI-EPITOPE PEPTIDE AND MRNA VACCINES TO GENERATE TOLEROGENIC EFFECTS FOR ALLERGIC AND AUTOIMMUNE DISEASE BY TARGETING LIVER SINUSOIDAL ENDOTHELIAL CELLS

Resumen de: US2025339514A1

In various embodiments tolerogenic nanoparticles are provided that induce immune tolerance to one or more desired antigen(s) and/or that reduce an immune response to those antigen(s). In certain embodiments the tolerogenic nanoparticle comprises a nanoparticle comprising a biocompatible polymer, a cationic lipid, or a combination thereof; an antigen disposed within or attached to said biocompatible polymer or a nucleic acid encoding said antigen, where said antigen comprises an antigen to which immune tolerance is to be induced by administration of said tolerogenic nanoparticle to a mammal; and a targeting moiety that binds to a scavenger receptor in the liver.

MACA-DERIVED NANOPARTICLES AND METHODS OF USE

Resumen de: WO2025231358A1

Isolation and characterization of MNDP, lipid nanoparticles derived from Lepidium meyenii Walp (maca), are disclosed. MDNP sequestered and neutralized multiple pro-inflammatory cytokines and APPs in its protein corona to reveal a highly effective therapeutic strategy for managing severe inflammatory responses. Lipid nanoparticle compositions based on the lipidomic analysis of MNDP are disclosed. Methods for the prevention and treatment for inflammatory diseases such as sepsis are disclosed.

IMMUNOGENIC PROTEINS AND NUCLEIC ACIDS ENCODING THE SAME

Resumen de: WO2025231483A2

The invention relates to proteins and nucleic acids for immunization regimens, in particular, immunogens that can prime rare bnAb precursor B cells and guide their maturation towards bnAbs capable of neutralizing diverse HIV strains, modifications thereof, and/or development of nanoparticles, and/or development of membrane-anchored immunogens, and methods of making and using the same. The invention also encompasses cell surface trimers that bind to the broadly neutralizing antibodies and/or nucleic acids encoding the same.

LIPID NANOPARTICLE (LNP) FORMULATIONS

Nº publicación: WO2025230906A1 06/11/2025

Solicitante:

SALIOGEN THERAPEUTICS INC [US]
SALIOGEN THERAPEUTICS, INC

Resumen de: WO2025230906A1

The present disclosure relates to lipid nanoparticles and uses thereof for, e.g. delivery to the liver and/or treating liver-related disorders or diseases.