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200
results.
Updated on
14/09/2026 [06:52:00]
Results 150 to 175 of 200
Absstract of: WO2026178657A1
Described herein are pH-responsive synthetic peptide shuttle agent having increased cargo transduction activity at acidic pH than at neutral pH. The pH-responsive synthetic peptide shuttle agent generally comprises an amphipathic alpha-helical motif when in acidic pH, the amphipathic alpha-helical motif having solvent-exposed surface comprising a discrete hydrophilic cationic face and a discrete hydrophobic face. The pH-responsive synthetic peptide shuttle agent described herein may be further conjugated to a cargo for intracellular delivery, and/or a targeting ligand for tissue-specific delivery. Also described herein are lipid nanoparticles (LNPs) incorporating synthetic peptide shuttle agents or pH- responsive synthetic peptide shuttle agents for improved payload delivery to the cytosol.
Absstract of: WO2026179355A1
The present application provides a branched cationic lipid containing an ester bond, the structure of which is represented by formula (1), wherein the definition of each symbol is consistent with that described herein. The branched cationic lipid containing an ester bond has a plurality of hydrophobic hydrocarbon tail chains. The cross-sectional area of the hydrophobic end increases, forming a conical geometric configuration, which is conducive to promoting membrane fusion and content release; moreover, a charged head group can interact with oppositely charged molecules on the cell membrane, thereby enabling the multi-tailed lipid to pass through the cell membrane more easily and exert its endosome-disrupting effect inside the cell, and thus improving drug release efficiency. The branched cationic lipid containing an ester bond of the present application comprises one or more biodegradable groups between a branching center and the hydrophobic tails. The presence of the degradable groups enables a lipid nanoparticle (LNP) prepared from the branched cationic lipid to be degraded timely within endosomes, such that the endosomal escape of drug molecules (such as nucleic acids) is promoted, thereby solving the problem that drugs delivered into the cell fail to exert their effects.
Absstract of: WO2026180422A1
The invention is directed to a compound for cell activation having a mean diameter between 500 nm and 10 µm and provided with one or more antigen recognizing moieties capable of activating cells characterized in comprising - first particles with a mean diameter of less than 500 nm which are provided on the surface with one or more first oligonucleotides having a length of 5 to 50 nucleotides - second particles with a mean diameter of less than 500 nm which are provided on the surface with one or more second oligonucleotides having a length of 5 to 50 nucleotides and with one or more antigen recognizing moieties wherein the first and second oligonucleotides have complementary sequences capable of hybridizing to each other, thereby binding the first and second particles to each other.
Absstract of: WO2025049928A1
A gene editing system comprising (a) a fusion polypeptide comprising a CRISPR nuclease and a reverse transcriptase, or a nucleic acid encoding the fusion polypeptide, and (b) an RNA molecule comprising a guide RNA and a reverse transcription donor RNA, or a nucleic acid encoding the RNA molecule. Also provided herein are methods of using the gene editing system for modifying target genes of interest.
Absstract of: WO2026183563A2
Compositions and methods for targeted delivery of a lipid nanoparticle (LNP) are described herein. In some embodiments, the compositions comprise (a) a hepatoavoidant lipid nanoparticle (LNP) associated with an RNA polynucleotide that comprises an expression sequence, and (b) a multi-specific targeting moiety, wherein the multi-specific targeting moiety binds (i) at least one target in or on the hepatoavoidant LNP and (ii) at least one cell-specific target in or on a cell. In some embodiments, the compositions comprise a multi-specific targeting moiety or an RNA polynucleotide comprising an expression sequence encoding a multi-specific targeting moiety, where the multi-specific targeting moiety binds (a) at least one target in or on an LNP, and (b) at least one cell-specific target. When expressed, the multi-specific targeting moiety allows for delivery of the LNP to the cells expressing the cell-specific target, by bringing the LNP in close proximity to the cells. In some embodiments, the LNP comprises an expression sequence encoding a therapeutic molecule, and the methods allow for targeted delivery for treating or preventing a disease or disorder.
Absstract of: WO2026183200A1
The present disclosure provides, among other things, targeted lipid nanoparticles and methods of targeted making lipid nanoparticles.
Absstract of: US20260256708A1
Provided are: oral nanoparticles for enabling oral administration of a bioactive compound which is typically administered by injection due to having low bioavailability attributable to issues with solubility, disintegration in the digestive tract, and intestinal permeability; a method of formulating the oral nanoparticles; and a use of the oral nanoparticles. In addition, provided are: oral nanoparticles capable of maintaining or protecting the balance in the gut microbiome by minimizing the exposure of the gut microbiome to a bioactive compound; a formulation; and a use of same.
Absstract of: WO2026180833A1
The subject of the invention is a core–shell structured nanocomposite composition comprising a core containing glibenclamide and soybean lecithin having phosphatidylcholine as a main component, and a shell containing a Poloxamer polymer. The invention further relates to the use of the nanocomposite composition in human medicine and/or veterinary medicine.
Absstract of: WO2026183439A1
The present disclosure provides compositions of lipid nanoparticles including lipid components and a cell-penetrating peptide conjugated to the lipid components useful for the delivery of nucleic acids including mRNAs, and methods thereof. These compositions may be used to treat a disease or disorder for which the delivery of a nucleic acid is therapeutically effective.
Absstract of: WO2026181030A1
It forms an object of the present invention chitosan piezoelectric nanoparticles (ChNPs), methods to prepare them and their use.
Absstract of: WO2026181880A1
The present invention provides a pH-responsive lipid derivative represented by formula (1): R1-L1-R2 (in the formula, R1 represents a lipid moiety, L1 represents NH or O, and R2 represents a polymer moiety having a repeating unit represented by formula (2) and a repeating unit represented by formula (3)), wherein the amount of a repeating unit (B) based on the total amount of a repeating unit (A) and the repeating unit (B) is 6 to 50 mol% (the symbols in formulae (2) and (3) are as defined in the description).
Absstract of: WO2026182546A1
The present invention relates to an oral gene delivery carrier and a gene delivery composition comprising same. The present invention comprises lipid nanoparticles surface-modified with a bile acid-based polymer (GCGA), and provides improved stability in the gastrointestinal (GI) environment, enhanced mucosal adhesion and cellular uptake, and significantly increased gene delivery efficiency in intestinal epithelial cells, thereby being useful for the development of an oral gene therapeutic agent.
Absstract of: WO2026178739A1
The present invention relates to an ionizable lipid molecule comprising a tocopherol structure, a lipid nanoparticle comprising same, and use thereof. Specifically, provided are an ionizable lipid molecule comprising a structure of a tocopherol and a derivative thereof as represented by formula (1), a lipid nanoparticle comprising same, a preparation method therefor, and use thereof. Compared with ionizable lipid molecules conventionally used in the art, the lipid nanoparticle prepared from the ionizable lipid molecule represented by formula (1) of the present invention can significantly improve nucleic acid delivery efficiency and expression.
Absstract of: WO2026179939A1
Lipid nanoparticles (LNPs) targeting liver sinusoidal endothelial cells and the use thereof. The LNPs consist of a cationic ionized lipid, an auxiliary lipid, a ligand-modified PEGylated lipid, and cholesterol. The auxiliary lipid and the ligand-modified PEGylated lipid target surface receptors of the liver sinusoidal endothelial cells (LSECs). The LNPs enhance, by means of a multivalent targeting design, specific uptake by LSECs, and encapsulate an RNA drug for treating an allergic disease or an autoimmune disease. The RNA drug can encode at least one epitope of an antigen causing an allergic or autoimmune disease. The LNPs can efficiently target LSECs to induce immune tolerance, providing a new antigen-specific immunomodulatory strategy for the treatment of allergic diseases or autoimmune diseases.
Absstract of: US20260256709A1
0000 Described herein are methods, systems and compositions for the production and use of a class of highly branched, nanostructured particles, generally referred to herein as dendritic particles (DPs), synthesized from biodegradable materials that are specifically adapted for drug release in vivo.
Absstract of: US20260258413A1
Compositions and methods for making and using engineered phagocytic cells that express a chimeric antigen receptor having an enhanced phagocytic activity for stable and durable expression are described and can be suitably used for immunotherapy in cancer or infection.
Absstract of: WO2025090138A1
The present disclosure provides stealth lipid nanoparticle (LNP) compositions engineered to target specific tissues or cell-types, e.g., T cells, B cells, natural killer cells, hematopoietic stem cells, to genetically modify the cells with therapeutic nucleic acid encapsulated in the LNP. The present disclosure also provides compositions and methods of making the LNPs and treatment using the same.
Absstract of: WO2025085950A1
The present invention is directed to lipid-based nanoparticles (e.g. lipid nanoparticles), formulations containing lipid-based nanoparticles and methods of treating diseases or conditions with said lipid-based nanoparticles and formulations thereof. The present invention provides a lipid-based nanoparticle comprising (a) an active agent, and (b) a plurality of capture binding domains displayed on the outer surface of the nanoparticle, wherein each capture binding domain is linked to the lipid-based nanoparticle through a site-specific linkage such that each capture binding domain is displayed in substantially the same orientation and capable of capturing a targeting moiety in an orientation that allows the targeting moiety to interact with its target. The present invention also provides a lipid-based nanoparticle comprising (a) an active agent, and (b) a plurality of targeting molecules displayed on the outer surface of the nanoparticle, wherein each targeting molecule is linked to the lipid-based nanoparticle through a site-specific linkage such that each targeting molecule is displayed in substantially the same orientation and capable of binding to a target on a cell surface.
Absstract of: WO2025090417A1
The present disclosure provides bispecific stealth lipid nanoparticle (LNP) compositions engineered to target specific tissues or cell-types, e.g., hematopoietic stem cells, to modify the cells with therapeutic nucleic acid encapsulated in the LNP. The present disclosure also provides compositions and methods of making the LNPs and treatment using the same.
Absstract of: EP4799601A1
0001 The present invention relates to the field of medicinal tissue mineralisation, in particular, i.e. tooth remineralisation and bone regeneration with self-assembling peptides. Use of self-assembling peptides, such as P11-4, also designated Oligopeptide-104, in these processes leads to generation of hydroxyapatite, which is also present in natural enamel, dentin and bone. The inventors have discovered that this can be significantly accelerated by combining a self-assembling peptide (SAP) such as P11-4, calcium ions, phosphate ions, and a polymer in the form of particles encapsulating a cargo selected from at least one of the SAP, the calcium ions and the phosphate ions into a composition or kit. The invention also provides medical use of said composition or kit, in particular, in the tooth, for remineralisation of caries lesions, such as subsurface caries lesions, mineralisation of pits and fissures, treatment of sensitive teeth, pulp capping, and for bone regeneration.
Absstract of: WO2025090891A1
In some embodiments, the invention relates to a composition for targeted delivery of intact mitochondrial DNA (mtDNA) to a target cell, the composition comprising a mtDNA molecule that is complexed to cell-penetrating peptides (CPPs) which promote cellular entry, wherein the complex forms a nanoparticle with the CPPs bound to the mtDNA molecule. In some embodiments, the invention relates to a method of treating a disease or disorder associated with mitochondrial dysfunction in a subject in need thereof.
Absstract of: WO2025088609A1
Disclosed are biocompatible, biodegradable, and low immunogenic alga(e) Nanoparticles (aNPs) that can adhere and deliver active ingredient(s) to mucosal epithelium tissue, as well as compositions comprising the aNPs, methods of preparing the aNPs, and uses thereof in methods of treatment.
Absstract of: US20260176252A1
0000 The present disclosure describes compositions, preparations, nanoparticles (such as lipid nanoparticles), and/or nanomaterials and methods of their use such as a compound of Formula (A) or a pharmaceutically acceptable salt thereof.
0000
Absstract of: EP4799614A1
0001 The present invention relates to a composition for drug delivery and a preparation method therefor and, more specifically, to: a composition for drug delivery which is formed such that a drug is encapsulated inside a nanoparticle structure formed by a polymer and a cationic lipid having a specific structure; and a preparation method therefor.
Nº publicación: EP4799694A2 02/09/2026
Applicant:
CHILDRENS MEDICAL CENTER [US]
OSPEDALE SAN RAFFAELE SRL [IT]
MILANO POLITECNICO [IT]
FOND TELETHON [IT]
CHILDREN'S MEDICAL CENTER CORPORATION
Ospedale San Raffaele S.r.l.
Politecnico di Milano
Fondazione Telethon
Absstract of: EP4799694A2
The present invention provides compositions and methods for the treatment or prevention of a neurological disease or disorder of the central nervous system (e.g., a storage disorder, lysosomal storage disorder, neurodegenerative disease, etc.) by reconstitution of brain myeloid cell and microglia upon transplantation of hematopoietic cells enriched in microglia reconstitution potential. The invention also provides compositions and methods for ablating and reconstituting microglia.
BOPI
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