Resumen de: WO2025031991A1
It relates to sulfur-containing lipo-polyamino acid conjugates of formula (I), and acceptable salts, stereoisomers and mixtures thereof. It also relates to self-assembled particles comprising these lipo/polyamino acid conjugates and optionally active agents, and to compositions comprising the lipo-polyamino acid conjugates or the self-assembled particles comprising them. It relates as well to the use of the lipo-polyamino acid conjugates, self-assembled particles, or compositions comprising them in medicine, cosmetics and diagnostics, and to the use of the lipo-polyamino acid conjugates of formula (I) as carriers.
Resumen de: US20260283956A1
Poly(oxazoline)-lipid conjugates with pendant cationic groups (cationic POZ-lipid) and lipid nanoparticles (LNPs) including cationic POZ-lipids used to facilitate delivery of an encapsulated payload. LNPs and polyplexes including cationic POZ-lipid and a nucleic acid payload such as, but not limited to, mRNA or modified mRNA are disclosed. Such LNPs have no immunogenicity or reduced immunogenicity as compared to a corresponding LNP containing an ionizable lipid.
Resumen de: WO2026198558A1
Disclosed herein are gene editing systems and components and formulations thereof. Some gene editing systems are described in the context of compositions for correcting the Z allele of the SERPINA1 gene to cure and/or treat alpha-1 antitrypsin deficiency (A1AD) by restoring the normal function of alpha-1 antitrypsin (A1AT) protein via corrective editing. The corrective editing may be implemented using template-based gene editing, may include one or more synonymous edits including at specific locations within and/or outside the operative protospacer region, may include a plurality of different guide nucleic acid molecules, and may be delivered in vivo to a subject. Gene editing systems may include affinity moieties. Gene editing systems may include modified proteins, such as polymerases and nucleases.
Resumen de: WO2026198635A1
The present disclosure provides an expression construct comprising three repeating units, wherein each repeating unit comprises a promoter, a nucleic acid encoding a tRNAArg/Op suppressor, and a termination sequence, wherein the promoter is operably linked to the nucleic acid encoding the tRNAArg/Op suppressor, wherein the tRNAArg/Op suppressor hybridizes to a UGA stop codon and is aminoacylated or is capable of being aminoacylated with arginine, wherein the promoter, nucleic acid encoding the tRNAArg/Op suppressor, and/or the termination sequence in each repeating unit may be the same as or different than the corresponding component in one or more other repeating unit, and wherein the nucleic acid encoding the tRNAArg/Op suppressor comprises a sequence that is at least 80% identical to SEQ ID NO: 6.
Resumen de: WO2026198578A1
Provided herein are prime editing methods and compositions for treatment of genetic disorders such as alpha-1 antitrypsin deficiency.
Resumen de: WO2026193603A1
Described herein are long non-coding (lncRNA), DNAs and vectors that encode them, nanoparticle complexes and pharmaceutical compositions comprising the lncRNA. The lncRNA can be encapsulated by a lipid nanoparticle, optionally where the lncRNA lacks a cap and/or tail and wherein the lncRNA comprises one or more lncRNA. Described herein are also methods of treating inflammatory diseases or diseases or conditions associated with a deficiency of a lncRNA, methods of altering expression of one or more cytokine expression in a subject using said long non-coding (lncRNA), DNAs and vectors that encode them, nanoparticle complexes and pharmaceutical compositions.
Resumen de: WO2026198639A1
Disclosed are lipidoid compounds having the structure of formula (I), (II), or (III), or a salt thereof: wherein the groups are as defined in the application. Also disclosed are nanoparticle compositions comprising a lipidoid of the invention that are capable of delivering a therapeutic agent. The application also discloses pharmaceutical compositions comprising a lipidoid composition of the invention.
Resumen de: US20260284185A1
Problem A composition and a method that can be used to induce an immune response to HTLV-1 are required.Solution A lipid complex comprising at least one nucleic acid selected from: a nucleic acid comprising a polynucleotide that encodes an immunogenic fragment of human T-cell leukemia virus 1 (HTLV-1) antigenic Gag protein; a nucleic acid comprising a polynucleotide that encodes an immunogenic fragment of HTLV-1 antigenic Tax protein; and a nucleic acid comprising a polynucleotide that encodes an immunogenic fragment of HTLV-1 antigenic HBZ protein; wherein the at least one nucleic acid is encapsulated in a lipid.
Resumen de: WO2026194982A1
The present invention provides a composition and method for preventing or treating a cardiovascular disease. The composition can reduce the Lp(a) level by editing an LPA gene, thereby reducing the risk of developing a cardiovascular disease or treating the cardiovascular disease. The composition comprises: (i) a nucleic acid encoding one or more guide RNAs, or a vector comprising the nucleic acid encoding the one or more guide RNAs; and (ii) an RNA-guided DNA binder, a nucleic acid encoding the RNA-guided DNA binder, or a vector comprising the nucleic acid encoding the RNA-guided DNA binder.
Resumen de: WO2026198450A1
Described is a method for substantially increasing the concentration of cell-free DNA (cfDNA) in a subject by administering a priming agent, such as heparin-mimicking polymers. Heparin-mimicking polymers inhibit cfDNA uptake by macrophages, or inhibit deoxyribonucleases prior to collection of a sample of cfDNA, e.g., by way of a liquid biopsy. Also described is a method for substantially increasing the concentration of cell-free DNA (cfDNA) in a subject by administering a targeted nanoparticle. These methods dramatically enhance the quality of detection achieved by downstream cfDNA analytical applications, such as sequencing applications.
Resumen de: US20260284114A1
A method of producing mammalian breast milk exosomes, for example human breast milk exosomes comprising generating lactocytes derived from mammalian mammary epithelial cells, for example human mammary epithelial cells, expressing the mammalian milk like product, for example the human milk like product from lactocytes, and purifying the exosomes from the mammalian milk like product.
Resumen de: US20260284228A1
This application relates to compositions and methods comprising epigenetic editors for epigenetic modification of PCSK9, as well as nucleic acids and vectors encoding the same. Also disclosed are cells epigenetically modified by the epigenetic editors.
Resumen de: US20260284174A1
The present invention is inter alia directed to immunogenic compositions comprising: (a) a first hemagglutinin (HA) antigen or a first nucleic acid, suitably mRNA, encoding the first HA antigen wherein the first HA antigen is derived from a strain of subtype H3 of Influenza A virus; and (b) a second HA antigen or a second nucleic acid, suitably mRNA, encoding the second HA antigen wherein the second HA antigen is derived from a strain of subtype H1 of Influenza A virus, wherein the ratio of (a):(b) is comprised between 1.5:1 and 20:1. The present invention is also directed to vaccines and kits or kits-of-parts comprising such. Immunogenic compositions, vaccines and kits-of-parts provided herein are suitable for use as a medicament, in particular, for use in the treatment or prophylaxis of an infection with an Influenza virus, suitably an Influenza A and/or Influenza B.
Resumen de: US20260284225A1
0000 A layer-by-layer ELP-nucleic acid (NA) nanoparticle (LENN) complex for targeted delivery of a therapeutic NA cargo to tumor cells; a method of formulating a LENN complex using a layer-by-layer deposition process (LbL); LENN complex formulations; pharmaceutical compositions comprising a LENN complex formulations; and methods of treating bladder cancer in a subject via LENN-mediated delivery of a therapeutic NA cargo.
Resumen de: US20260283966A1
Described herein are particles including a protein and optionally an active agent such as a particle comprising whey protein hydrolysate and optionally tryptophan. Also described herein are methods of making and using particles that include a protein and optionally an active agent.
Resumen de: US20260284168A1
The disclosure provides agents and methods for preventing or treating tuberculosis using RNA. The RNA encoding antigens of Mycobacterium tuberculosis, immunogenic variants or fragments thereof is formulated and administered in a way that the antigens, variants or fragments are produced by cells of a subject.
Resumen de: US20260284271A1
Compositions comprised of hyaluronic acid with low extents of ester linkages are disclosed. The compositions may enable longer half-lives of hyaluronic acid delivered in tissues throughout the body to prolong mechanical effects through controlled swelling and solubilization of hyaluronic acid as ester crosslinks between hyaluronic acid polymers hydrolyze. The compositions may also enable longer half-lives of medicaments or drugs in tissues throughout the body as ester linkages between the medicaments or drugs and hyaluronic acid polymers or networks hydrolyze. Compositions may include acrylate modified hyaluronic acid with low degrees of acrylate modification in order to preserve the physical properties of hyaluronic acid in the body.
Resumen de: US20260284198A1
The present disclosure relates to a copolymer and a polymersome for targeted delivery of biomolecules to a living organism. Hie exemplary copolymer comprises an initiator block, a propagator block, and a linkage connecting the initiator block and the propagator block. The initiator block comprises a glycan head configured to provide a targeted delivery, and the propagator block comprises a functional moiety configured to provide desired properties for the polymersome.
Resumen de: US20260284163A1
Provided are, inter alia, immunogenic and vaccine compositions including a lipid vesicle, a first nucleic acid; and a peptide, and methods of treating or preventing pancreatic cancer using the compositions.
Resumen de: US20260284001A1
A nanoparticle has a plurality of therapeutic molecules arranged in the form of a spherical micelle, suitable for localized delivery and release of mycophenolic acid, and effective in treatment of autoimmune diseases, fibrotic diseases and/or organ rejection diseases. A pharmaceutical composition including the nanoparticle in a pharmaceutically acceptable vehicle, and a method for manufacturing the nanoparticle are provided.
Resumen de: US20260283974A1
0000 This disclosure features novel lipid nanoparticle formulations and uses thereof. The lipid nanoparticle (“LNP”) includes an encapsulated therapeutic agent and an aqueous solution comprising a salt and an anionic polymer, wherein the salt and the anionic polymer are dissolved in the aqueous solution, thereby forming polymer coated lipid nanoparticle (“PCLNP”). Lipid nanoparticles of this disclosure are useful in the process of lyophilization or freeze drying and decrease nanoparticle aggregation and maintain efficacy once reconstituted.
Resumen de: US20260284615A1
A device 100 for producing nanoparticles includes a microplate 102 which includes a plurality of fluidic mixing units 104 arranged in an array. Each fluidic mixing unit is configured to produce nanoparticles. The device enables high throughput lipid nanoparticle testing and development, and is configured to generate large numbers of novel or unique nanoparticle formulations.
Resumen de: US20260283987A1
Hydrophobic ceramides are of limited use due to having extremely low solubility in aqueous solutions, and have low cell transduction efficiency when used as a drug. The present invention provides a highly water-dispersible composition having a high ceramide content. A high dose of a hydrophobic ceramide can be easily delivered to a target in the body through the present invention to increase the utility as a highly water-dispersible drug carrier or pharmaceutical composition according to the intended purpose.
Resumen de: US20260284209A1
The present disclosure provides polypeptide-based adjuvants that are capable of activating the innate immune system and are capable of generating targeted and precise immunogenicity. Also provided are compositions, pharmaceutical compositions, and vaccines comprising or using the polypeptide-based adjuvants, as well as methods of treating cancer or including or enhancing an immune response using such polypeptide-based adjuvants.
Nº publicación: US20260283975A1 24/09/2026
Solicitante:
KOEBENHAVNS UNIV [DK]
K\u00D8BENHAVNS UNIVERSITET
Resumen de: US20260283975A1
The present invention has been made within the field of nanoparticles and relates to a nanoparticle composition comprising the polymer poly(D,L-lactic-co-glycolic acid) (PLGA). In particular the present invention relates to a lipid polymer hybrid nanoparticle composition comprising a cationic or cationically ionisable lipid or lipid-like material, a helper lipid, a lipopolymer, and one or more variants of PLGA. The nanoparticle composition is particularly useful for delivery of nucleic acid molecules, specifically mRNA; thereby making them highly suitable for use in vaccines, such as for the prevention and/or treatment of infectious diseases.