Alerta

RNA SEPARATION AND RELATED TECHNIQUES FOR DETERMINING VIRUSES SUCH AS CORONAVIRUSES

Resumen de: US2025290166A1

Certain aspects of the present disclosure generally relate to systems and methods for determining viruses such as coronaviruses. For instance, some aspects are directed to systems and methods for determining viruses using a partitioning system. Within the partitioning system, free RNA or other nucleic acids may preferentially partition into one phase, while intact viruses may be present in the other phase or in both phases. Accordingly, in some cases, free RNA or other nucleic acids may be preferentially removed, e.g., as compared to intact RNA or other nucleic acids present within a virus. In some cases, the phase containing intact viruses can be determined to determine the infectiousness, e.g., of a sample arising from a subject. This may be useful, for example, for distinguishing subjects who are capable of spreading an infection from those who are not infectious.

HSC-SPECIFIC ANTIBODY CONJUGATED LIPID NANOPARTICLES AND USES THEREOF

Resumen de: AU2024214423A1

Provided are ionizable cationic lipids and lipid nanoparticles for the delivery of nucleic acids to cells (e.g., HSC), and methods of making and using such lipids and targeted lipid. nanoparticles.

NANOPARTICLE FORMULATIONS FOR A NON-OPIOID DRUG

Resumen de: AU2024234362A1

This disclosure is directed to a nanoparticle formulation for N,N-Diethyl-2-(N-(2-(4-hydroxyphenylamino)-2-oxoethyl)sulfamoyl)benzamide (SRP-3D (DA)), an active pharmaceutical ingredient that is insoluble in water and cannot be dissolved in any injection-safe vehicle to a sufficient concentration for dosing. Also encompassed by the disclosure are nanoparticulate suspension comprising a nanoparticulate SRP-3D (DA) (or SRP-3D (DA) nanoparticles), a wetting agent and a cryoprotectant. The nanoparticulate compositions comprise SRP-3D (DA) particles having an average particle size of less than about 500 nm or less than about 300 nm.

MULTI-FUNCTIONAL LIPID NANOPARTICLES AND USES THEREOF

Resumen de: AU2024217077A1

The present disclosure relates to lipid nanoparticles including a lipid layer, a cell-penetrating peptide conjugated to the lipid layer, a collagen-targeting peptide conjugated to the lipid layer and a nucleic acid associated with a vascular disease or condition. The present disclosure also relates to methods of making and using the described lipid nanoparticles for treating vascular disease.

Compositions and methods for organ specific delivery of nucleic acids

Resumen de: AU2025223843A1

The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid. The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid. ug u g h e p r e s e n t 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 w h i c h s h o w n p r e f e r e n t i a l t a r g e t i n g o r d e l i v e r y o f a n u c l e i c a c i d c o m p o s i t i o n t o a p a r t i c u l a r o r g a n n s o m e e m b o d i m e n t s , t h e c o m p o s i t i o n c o m p r i s e s a s t e r o i d o r s t e r o l , a n i o n i z a b l e c a t i o n i c l i p i d , a p h o s p h o l i p i d , a l i p i d , a n d a p e r m a n e n t l y c a t i o n i c l i p i d w h i c h m a y b e u s e d t o d e l i v e r a n u c l e i c a c i d

Compositions and methods for organ specific delivery of nucleic acids

Resumen de: AU2025223878A1

The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid. The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid. ug u g h e p r e s e n t 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 w h i c h s h o w n p r e f e r e n t i a l t a r g e t i n g o r d e l i v e r y o f a n u c l e i c a c i d c o m p o s i t i o n t o a p a r t i c u l a r o r g a n n s o m e e m b o d i m e n t s , t h e c o m p o s i t i o n c o m p r i s e s a s t e r o i d o r s t e r o l , a n i o n i z a b l e c a t i o n i c l i p i d , a p h o s p h o l i p i d , a l i p i d , a n d a p e r m a n e n t l y c a t i o n i c l i p i d w h i c h m a y b e u s e d t o d e l i v e r a n u c l e i c a c i d

Lipid nanoparticle compositions for delivery of mRNA and long nucleic acids

Resumen de: AU2025220887A1

LIPID NANOPARTICLE COMPOSITIONS FOR DELIVERY OF MRNA AND LONG NUCLEIC ACIDS In some aspects, the present disclosure provides compositions of lipid nanoparticles useful for the delivery of large RNAs including mRNAs. These compositions may include a cationic ionizable lipid, a phospholipid, a PEGylated lipid, and a steroid including using less of a cationic ionizable lipid than compositions with shorter nucleic acids. These compositions may be used to treat a disease or disorder for which the delivery of an mRNA is therapeutically effective. LIPID NANOPARTICLE COMPOSITIONS FOR DELIVERY OF MRNA AND LONG NUCLEIC ACIDS In some aspects, the present disclosure provides compositions of lipid nanoparticles useful for the delivery of large RNAs including mRNAs. These compositions may include a cationic ionizable lipid, a phospholipid, a PEGylated lipid, and a steroid including using less of a cationic ionizable lipid than compositions with shorter nucleic acids. These compositions may be used to treat a disease or disorder for which the delivery of an mRNA is therapeutically effective. ug u g n s o m e a s p e c t s , t h e p r e s e n t 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 o f l i p i d n a n o p a r t i c l e s u s e f u l f o r t h e d e l i v e r y o f l a r g e s i n c l u d i n g m s h e s e c o m p o s i t i o n s m a y i n c l u d e a c a t i o n i c i o n i z a b l e l i p i d , a p h o s p h o l i p i d , a y l a t e d l i p i d , a n d a s t e r o i d

ANTIBODY AND SIRNA NANOCARRIERS AND USES THEREOF

Resumen de: WO2025193917A1

The present invention provides a method for suppressing a target gene involved in the Wnt/β-catenin signaling pathway in target cells expressing a receptor capable of binding an extracellular Wnt ligand and an intracellular Wnt effector. The method comprises providing nanocarriers and binding the nanocarriers to the target cells and/or internalizing of the nanocarriers into the target cells, whereby the target gene is suppressed in the target cells. In each nanocarrier, an antibody specific for the receptor, a siRNA specific for the effector, or a combination thereof may be attached to a nanoparticle. The target cells may be in a subject. The method may further comprise reducing disease burden and/or inducing stabilization of a disease in the subject. Also provided are methods for synthesizing the nanocarriers.

新規のイオン化可能な脂質および脂質ナノ粒子ならびにそれらの使用方法

Resumen de: MX2025002413A

Novel ionizable lipids and lipid nanoparticles that can be used in the delivery of therapeutic cargos are disclosed.

ARTIFICIAL AMINO ACID CONTAINING LIPO-OLIGOMERS FOR RIBONUCLEOPROTEIN DELIVERY

Resumen de: WO2024099975A1

The invention relates to nanoparticles for Cas protein/gRNA ribonucleoprotein (RNP) complex, siRNA or PMO delivery comprising one or more Cas protein/gRNA RNP complex(es), siRNA(s) or PMO(s) as cargo; and a carrier comprising a sequence-defined T-shape lipo-oligomer comprising a tyrosine tripeptide and either a single artificial amino acid or two hydrophobic artificial amino acids at either side of a central lysine branching point and a hydrophobic tail comprising two fatty acids, wherein the artificial amino acid is an oligo(alkylamino) acid. The invention further relates to therapeutic and non-therapeutic uses thereof and to an in vitro method for transfecting mammalian cells.

DESIGNER EXTRACELLULAR VESICLES FOR TARGETED DELIVERY TO MUSCLE CELLS

Resumen de: WO2024102965A1

Disclosed herein are designer extracellular vesicles (EVs) that target muscle cells. For example, in some embodiments, the EVs are decorated NHERF1, NHERF2, a fusion protein containing an E8 fragment of laminin and an exosomal or lysosomal transmembrane protein, or a combination thereof. These EVs can in some embodiments, be used to deliver diagnostic and/or therapeutic cargo to muscle cells in a subject in need thereof. In some embodiments, these EVs are loaded with a DUX4 silencing oligonucleotide to treat Facioscapulohumeral muscular dystrophy (FSHD) in a subject. For example, in some embodiments, the therapeutic cargo is a DUX4 silencing oligonucleotide.

SURFACE-MODIFIED LIPID NANOPARTICLES

Resumen de: TW202428247A

The disclosure provides conjugates comprising a targeting moiety, e.g., an antibody, Fab fragment or single chain variable fragment (ScFv), and a lipid nanoparticle (LNP) encapsulating a therapeutic agent (i.e., payload), wherein the targeting moiety, e.g., antibody, Fab fragment or the ScFv, is conjugated to the lipid nanoparticle through a linker, and wherein the linker comprises an enzyme recognition sequence such as a sortase recognition motif or a lipoic acid acceptor peptide. The disclosure further provides methods of preparing such conjugates.

LIPID NANOPARTICLE DRUG CONJUGATES

Resumen de: AU2023375378A1

The disclosure provides conjugates comprising a targeting moiety, e.g., an antibody, Fab fragment or single chain variable fragment (ScFv), and a lipid nanoparticle (LNP) encapsulating a therapeutic agent (i.e., payload), wherein the targeting moiety, e.g., antibody, Fab fragment or the ScFv, is conjugated to the lipid nanoparticle through a linker, and wherein the linker comprises an enzyme recognition sequence and a Click product formed from a Click reaction between a first Click handle on the targeting moiety, e.g., antibody, Fab fragment, or ScFv, and a second Click handle on the LNP.

LIPID NANOPARTICLE DRUG CONJUGATES

Resumen de: AU2023375377A1

The disclosure provides conjugates comprising a targeting moiety, e.g., an antibody, Fab fragment or single chain variable fragment (ScFv), and a lipid nanoparticle (LNP) encapsulating a therapeutic agent (i.e., payload), wherein the targeting moiety, e.g., antibody, Fab fragment or the ScFv, is conjugated to the lipid nanoparticle through a linker, and wherein the linker comprises an enzyme recognition sequence and a Click product formed from a Click reaction between a first Click handle on the targeting moiety, e.g., antibody, Fab fragment, or ScFv, and a second Click handle on the LNP.

PREPARATION OF LIPID NANOPARTICLE (LNP) CONJUGATES

Resumen de: AU2023375378A1

The disclosure provides conjugates comprising a targeting moiety, e.g., an antibody, Fab fragment or single chain variable fragment (ScFv), and a lipid nanoparticle (LNP) encapsulating a therapeutic agent (i.e., payload), wherein the targeting moiety, e.g., antibody, Fab fragment or the ScFv, is conjugated to the lipid nanoparticle through a linker, and wherein the linker comprises an enzyme recognition sequence and a Click product formed from a Click reaction between a first Click handle on the targeting moiety, e.g., antibody, Fab fragment, or ScFv, and a second Click handle on the LNP.

PARTICLE COMPOSITIONS COMPRISING POLYSARCOSINE LIPID CONJUGATES

Resumen de: US2024180844A1

The present disclosure features particle compositions, such as liposomes and lipid nanoparticles, comprising a polysarcosine-lipid conjugate, as well as methods of making and using the same.

SELF-ASSEMBLING LIPID NANOPARTICLES FOR TARGETED DELIVERY OF THERAPEUTIC AGENTS

Resumen de: MX2025005244A

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.

RNA COMPOSITIONS COMPRISING LIPID NANOPARTICLES OR LIPID RECONSTRUCTED NATURAL MESSENGER PACKS

Resumen de: CN120359023A

Disclosed herein are RNA compositions comprising one or more polynucleotides encoding one or more gene editing systems, the one or more polynucleotides being formulated within a lipid reconstituted native messenger packet (LNMP) comprising a native lipid and an ionizable lipid. The disclosure also includes a method for preparing an RNA composition comprising reconstituting a membrane comprising a purified NMP lipid in the presence of an ionizable lipid to produce an LNMP comprising the ionizable lipid, and loading one or more polynucleotides encoding one or more gene editing systems into the LNMP. The present disclosure also includes RNA compositions that can be repeatedly administered.

CIRCULAR RNA COMPOSITIONS

Resumen de: US2025262324A1

Provided herein are circular RNA constructs comprising an IRES, and at least one expression sequence encoding binding molecule, compositions thereof, and methods of treatment, including for cancer and autoimmune disease. In particular, circular RNA comprising an IRES and a CD19 binder, a HER2 binder, or a BCMA binder are provided, optionally formulated with a delivery vehicle. Precursor polynucleotides comprising an IRES, and at least one expression sequence encoding a CAR construct are also described herein.

A PROTEIN-COATED GAS VESICLE

Resumen de: WO2024100320A1

The present invention is directed to a protein-coated gas vesicle comprising a class II type hydrophobin HFBI. According to the present invention, there is also provided a protein-coated gas vesicle comprising class II type hydrophobin HFBI for use in medical applications. According to the present invention, there is also provided an ultrasound contrast agent comprising a plurality of vesicles comprising class II type hydrophobin HFBI.

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

Resumen de: WO2024100217A1

The present invention relates to zinc-based nanoclusters having the following features: - they comprise a zinc-based metal core covered over its entire surface with a mixed layer comprising histidine (His), acetate ions (Ac) and ascorbate ions (Asc), said metal core consisting of zinc (Zn) or of a mixture of zinc and selenium (ZnSe), the amount of zinc within said mixture ranging from 80 to 99.99% and the amount of selenium within said mixture ranging from 0.01 to 20%, the percentages being percentages by weight based on the total weight of the mixture; - they have a spherical shape; - they have a hydrodynamic diameter ranging from 0.6 to 2.0 nm, and preferably less than 1.0 nm; - they have a metal core diameter ranging from 0.5 to 1.5 nm, and preferably less than 1.0 nm; - they have a stability over time ranging from 5 to 20 weeks when the nanoclusters are in liquid form and are stored at a temperature of 4°C; - they have a stability over time of at least 12 months, and preferably from 12 to 18 months, when the nanoclusters are in dry form and are stored at a temperature of 4°C and under nitrogen; - they have spectrophotometric properties, with a shoulder peak on 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, it being possible for said zinc-based nanoclusters to also be denoted by the formula "ZnNC@HisAcAsc" or "ZnSeNC@HisAcAsc" depending on whether the metal core con

CELL COMPOSITIONS

Resumen de: CN120187845A

A cell composition of non-human animal origin is described comprising a plurality of cells of non-human animal origin wherein the cells comprise at least one exogenous supplement contained in a carrier selected from the group consisting of nanocarriers, nanoparticles, micelles, liposomes or vesicles. The cellular compositions are characterized by having improved organoleptic properties, such as improved flavor, color, texture or mouthfeel and/or with a meat-like flavor. Methods of producing them and comestible products comprising them are also described.

IRON NANOCLUSTERS, METHODS FOR OBTAINING SAME AND USES THEREOF FOR COMBATTING IRON DEFICIENCIES

Resumen de: WO2024100213A1

The present invention relates to iron nanoclusters having the following features: - they are covered on their surface by a mixed layer comprising histidine (His), acetate ions (Ac) and ascorbate ions (Asc); - they are spherically shaped; - they have a hydrodynamic diameter ranging from 0.6 to 2.0 nm, and preferably of less than 1.0 nm; - they have a metal core diameter ranging from 0.5 to 1.5 nm, and preferably of less than 1.0 nm; - they have a stability over time ranging from 5 to 20 weeks when the nanoclusters are in liquid form and are stored at a temperature of 4°C; - they have a stability over time of at least 12 months, and preferably 12 to 18 months, when the nanoclusters are in dry form and are stored at a temperature of 4°C and under nitrogen; - they have spectrophotometric properties, with a shoulder peak on 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, it being possible for these iron nanoclusters to be denoted by the formula "FeNC@HisAcAsc". The invention also relates to a method for preparing these iron nanoclusters, and to the uses thereof for combatting iron deficiencies.

BASE EDITING OF TRANSTHYRETIN GENE

Resumen de: AU2023375580A1

Provided herein are compositions for gene modification related to base editor systems, and methods of using the same to treat or prevent conditions associated with the extracellular deposition in various tissues of amyloid fibrils formed by the aggregation of misfolded transthyretin (TTR) proteins. Such conditions include, but are not limited to, polyneuropathy due to hereditary transthyretin amyloidosis (hATTR-PN) and hereditary cardiomyopathy due to transthyretin amyloidosis (hATTR-CM), both associated with autosomal dominant mutations of the TTR gene, and an age-related cardiomyopathy associated with wild-type TTR proteins (ATTRwt), also known as senile cardiac amyloidosis.

基于生物硫醇特异性响应的电荷转移复合物药物制剂及其制备方法

Nº publicación: CN120643703A 16/09/2025

Solicitante:

山西医科大学

Resumen de: CN120643703A

一种基于生物硫醇特异性响应的电荷转移复合物药物制剂，由具有电子供体性质的临床小分子药物和具有电子受体能力的醌式结构有机共轭分子通过电荷转移作用形成D‑A前体，再以可在水溶液中形成胶束的两亲性聚合物包覆形成CTDC纳米粒子。本发明药物制剂首先可利用其光热、光声信号用于疾病诊断；其次电子受体部分能与疾病微环境中生物硫醇物质反应导致CTDC解组装释放电子供体药物发挥药效，且光照会加速药物释放过程，CTDC解组装伴随光声信号消失，用于监测药物释放过程。通过集成实时光声成像、微环境响应药物释放、药物释放监测和疾病治疗，实现了基于临床药物的精准诊疗一体化。