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ステルス安定化硫黄含有リポポリアミノ酸

NºPublicación:  JP2026531746A 24/09/2026
Solicitante: 
ポリペプチドセラピューティックソリューションズ,エス.エル.
JP_2026531746_A

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.

CATIONIC POLY(OXAZOLINE) LIPID CONJUGATES AND LIPID NANOPARTICLES AND POLYPLEXES INCLUDING SAME

NºPublicación:  US20260283956A1 24/09/2026
Solicitante: 
SERINA THERAPEUTICS AL INC [US]
Serina Therapeutics (AL), Inc.
US_20260283956_A1

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.

CORRECTIVE EDITING OF THE SERPINA1 Z ALLELE FOR TREATMENT OF ALPHA-1 ANTITRYPSIN DEFICIENCY AND GENE EDITING SYSTEMS

NºPublicación:  WO2026198558A1 24/09/2026
Solicitante: 
VERVE THERAPEUTICS INC [US]
VERVE THERAPEUTICS, INC.
WO_2026198558_A1

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.

MULTI-COPY NUMBER SUPPRESSOR TRNA CONSTRUCT

NºPublicación:  WO2026198635A1 24/09/2026
Solicitante: 
ARTAN BIO [US]
ARTAN BIO
WO_2026198635_A1

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.

GENOME EDITING COMPOSITIONS AND METHODS FOR TREATMENT OF ALPHA-1-ANTITRYPSIN DEFICIENCY

NºPublicación:  WO2026198578A1 24/09/2026
Solicitante: 
PRIME MEDICINE INC [US]
PRIME MEDICINE, INC.
WO_2026198578_A1

Resumen de: WO2026198578A1

Provided herein are prime editing methods and compositions for treatment of genetic disorders such as alpha-1 antitrypsin deficiency.

MODIFIED AND NON-MODIFIED LONG NON-CODING RNA AND METHODS OF USE THEREOF

NºPublicación:  WO2026193603A1 24/09/2026
Solicitante: 
THE GOVERNING COUNCIL OF THE UNIV OF TORONTO [CA]
THE GOVERNING COUNCIL OF THE UNIVERSITY OF TORONTO
WO_2026193603_A1

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.

IONIZABLE LIPIDOID COMPOSITIONS AND THERAPEUTIC USES THEREOF

NºPublicación:  WO2026198639A1 24/09/2026
Solicitante: 
FLAGSHIP PIONEERING INNOVATIONS VII LLC [US]
FLAGSHIP PIONEERING INNOVATIONS VII, LLC
WO_2026198639_A1

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.

LIPID COMPLEX

NºPublicación:  US20260284185A1 24/09/2026
Solicitante: 
EISAI R&D MAN CO LTD [JP]
NATIONAL UNIV CORPORATION KUMAMOTO UNIV [JP]
EISAI R&D MANAGEMENT CO., LTD.
NATIONAL UNIVERSITY CORPORATION KUMAMOTO UNIVERSITY
US_20260284185_A1

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.

COMPOSITION AND METHOD FOR PREVENTING OR TREATING CARDIOVASCULAR DISEASE

NºPublicación:  WO2026194982A1 24/09/2026
Solicitante: 
ACCUREDIT THERAPEUTICS SUZHOU CO LTD [CN]
\u9510\u6B63\u57FA\u56E0\uFF08\u82CF\u5DDE\uFF09\u6709\u9650\u516C\u53F8
WO_2026194982_A1

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.

METHODS AND COMPOSITIONS FOR IMPROVING CELL-FREE (CFDNA) DETECTION

NºPublicación:  WO2026198450A1 24/09/2026
Solicitante: 
THE BROAD INST INC [US]
MASSACHUSETTS INST OF TECHNOLOGY [US]
THE GENERAL HOSPITAL CORP [US]
THE BROAD INSTITUTE, INC.
MASSACHUSETTS INSTITUTE OF TECHNOLOGY
THE GENERAL HOSPITAL CORPORATION
WO_2026198450_A1

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.

METHOD FOR PRODUCING MILK LIKE PRODUCTS

NºPublicación:  US20260284114A1 24/09/2026
Solicitante: 
SOC DES PRODUITS NESTLE S A [CH]
SOCI\u00C9T\u00C9 DES PRODUITS NESTL\u00C9 S.A.
US_20260284114_A1

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.

COMPOSITIONS AND METHODS FOR EPIGENETIC REGULATION OF PCSK9 EXPRESSION

NºPublicación:  US20260284228A1 24/09/2026
Solicitante: 
NCHROMA BIO INC [US]
nChroma Bio, Inc.
US_20260284228_A1

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.

INFLUENZA VIRUS VACCINES

NºPublicación:  US20260284174A1 24/09/2026
Solicitante: 
GLAXOSMITHKLINE BIOLOGICALS SA [BE]
GLAXOSMITHKLINE BIOLOGICALS SA
US_20260284174_A1

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.

DELIVERY OF NUCLEIC ACIDS USING A TARGETED PEPTIDE CARRIER SYSTEM

NºPublicación:  US20260284225A1 24/09/2026
Solicitante: 
PURDUE RES FOUNDATION [US]
Purdue Research Foundation
US_20260284225_A1

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.

PROTEIN PARTICLES AND METHODS OF MAKING AND USING THE SAME

NºPublicación:  US20260283966A1 24/09/2026
Solicitante: 
DAIRY MAN INC [US]
CORNELL UNIV [US]
Dairy Management Inc.
Cornell University
US_20260283966_A1

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.

RNA FOR PREVENTING OR TREATING TUBERCULOSIS

NºPublicación:  US20260284168A1 24/09/2026
Solicitante: 
BIONTECH SE [DE]
NATIONAL UNIV OF SINGAPORE [SG]
THE REGENTS OF THE UNIV OF CALIFORNIA [US]
BIONTECH SE
NATIONAL UNIVERSITY OF SINGAPORE
THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
US_20260284168_A1

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.

HYALURONIC ACID COMPOSITIONS WITH LOW EXTENTS OF ESTER LINKAGES AND METHODS OF USE THEREOF

NºPublicación:  US20260284271A1 24/09/2026
Solicitante: 
PROHIBIX LLC [US]
Prohibix LLC
US_20260284271_A1

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.

METHODS AND COMPOSITIONS FOR TARGETED DELIVERY BY POLYMERSOMES

NºPublicación:  US20260284198A1 24/09/2026
Solicitante: 
ROCK BIOMEDICAL INC [TW]
Rock BioMedical, Inc.
US_20260284198_A1

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.

PEPTIDE-BASED VACCINES AND METHODS FOR TREATMENT

NºPublicación:  US20260284163A1 24/09/2026
Solicitante: 
THE JOHNS HOPKINS UNIV [US]
The Johns Hopkins University
US_20260284163_A1

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.

NANOPARTICLES OF MYCOPHENOLIC ACID PRODRUG MOLECULES AND THERAPEUTIC USE THEREOF

NºPublicación:  US20260284001A1 24/09/2026
Solicitante: 
A D A S R L [IT]
A.D.A. S.R.L.
US_20260284001_A1

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.

METHODS OF LYOPHILIZING LIPID NANOPARTICLES

NºPublicación:  US20260283974A1 24/09/2026
Solicitante: 
TAKEDA PHARMACEUTICAL CO LIMITED [JP]
TAKEDA PHARMACEUTICAL COMPANY LIMITED
US_20260283974_A1

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.

FLUIDIC MIXER UNIT DEVICE FOR NANOPARTICLE PRODUCTION

NºPublicación:  US20260284615A1 24/09/2026
Solicitante: 
ASTRAZENECA AB [SE]
AstraZeneca AB
US_20260284615_A1

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.

HIGHLY WATER-DISPERSIBLE COMPOSITION HAVING HIGH CERAMIDE CONTENT

NºPublicación:  US20260283987A1 24/09/2026
Solicitante: 
MOOGENE MEDI CO LTD [KR]
MOOGENE MEDI CO., LTD.
US_20260283987_A1

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.

POLYPEPTIDE ADJUVANTS AND USES THEREOF

NºPublicación:  US20260284209A1 24/09/2026
Solicitante: 
BOARD OF REGENTS THE UNIV OF TEXAS SYSTEM [US]
BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM
US_20260284209_A1

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.

NANOPARTICLE COMPOSITIONS COMPRISING ONE OR MORE VARIANTS OR POLY (D,L-LACTIC-CO-GLYCOLIC ACID)

Nº publicación: US20260283975A1 24/09/2026

Solicitante:

KOEBENHAVNS UNIV [DK]
K\u00D8BENHAVNS UNIVERSITET

US_20260283975_A1

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.

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