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NUCLEIC ACID SEQUENCE(S) AND CONSTRUCT(S) FOR TREATING METHYLMALONIC ACIDEMIA AND METHOD(S) THEREOF

Resumen de: WO2025126242A1

The present disclosure provides a nucleic acid sequence encoding MMUT for the treatment of Methylmalonic acidemia (MMA). Said nucleic acid is codon optimized to improve various parameters and allows high expression in the cells it is expressed in. The present disclosure also provides nucleic acid construct(s) comprising the nucleic acid sequence encoding MMUT along with one or more regulatory elements. Modes of delivery of the said nucleic acid sequence include delivery by way of encapsulation in a lipid nanoparticle when the nucleic acid is an mRNA and by way of a recombinant AAV vector when the nucleic acid is a DNA sequence. The present disclosure also relates to a composition and kit comprising the nucleic acid sequence, constructs or delivery vehicles as well as their applications in treating MMA.

PREPARATION AND USE OF ALBUMIN-PACLITAXEL TWIN DRUG NANOPARTICLE

Resumen de: WO2025124615A2

The present invention belongs to the technical field of pharmaceutical chemistry and biopharmaceuticals. Disclosed in the present invention are the preparation and use of an albumin-paclitaxel twin drug nanoparticle. The preparation particularly relates to mixing albumin with water to prepare an albumin solution; mixing paclitaxel or a paclitaxel twin drug compound with an organic solvent to obtain a drug solution; and mixing the albumin solution with the drug solution, and performing ultrasonic dispersion, and then rotary evaporation and ultrafiltration to prepare an albumin-binding paclitaxel or albumin-paclitaxel twin drug nanoparticle. With regard to the albumin-paclitaxel twin drug prepared in the present invention, drug accumulation in tumors can be increased by means of using the special GP60-caveolin-SPARC transport mechanism of albumin. Both in-vivo and in-vitro experiments prove that the albumin-paclitaxel twin drug nanoparticles of the present invention have a good anti-tumor effect.

COMPOSITION AND COMPOUND FOR DELIVERING THERAPEUTIC AGENT

Resumen de: WO2025124560A1

A lipid nanoparticle composition for delivering a therapeutic agent, comprising a compound of formula I or compounds of formula I and formula II, or a salt thereof.

PROTEIN CARRIER-BASED ACTIVE INGREDIENT TRANSDERMAL DELIVERY SYSTEM, PREPARATION METHOD THEREFOR, AND USE THEREOF

Resumen de: WO2025123597A1

A protein carrier-based active ingredient transdermal delivery system, which is a small molecule active ingredient-protein-polymer composite structure and comprises a small molecule active ingredient, a protein loaded with the small molecule active ingredient, and a polymer coating a surface of the protein. The protein is modified with a polymerizable double-bond compound. After the loading of the small molecule active ingredient, a polymerization reaction is initiated on the surface of the protein in situ, so that the surface of the protein is coated with a polymer layer. The polymer layer is biologically friendly, and the surface properties of the polymer layer can be regulated and controlled according to the transdermal depth requirement, thereby achieving the targeted transdermal delivery of proteins and active small molecules while breaking through the skin barrier, and significantly overcoming the defect of poor delivery efficiency of previous protein-based delivery systems. The transdermal delivery system has a simple preparation method with a high yield, and thus can be industrially produced on a large scale.

NANOPARTICLE LOADED WITH PHOTOSENSITIZER CE6 AND TARGETING MITOCHONDRIA OF BREAST CANCER CELLS, AND PREPARATION METHOD THEREFOR AND USE THEREOF

Resumen de: WO2025123478A1

The present invention provides a nanoparticle loaded with a photosensitizer Ce6 and targeting mitochondria of breast cancer cells, and a preparation method therefor and a use thereof. The nanoparticle uses hollow mesoporous silica as a carrier; a photosensitizer Ce6 is loaded at the hollow position of the carrier; a red cell membrane is encapsulated outside the carrier; and one side of the carrier after being encapsulated by the red cell membrane is modified with a Pt nanocluster, and the other side of the carrier is modified with an LXL-1 aptamer and TPP. The present invention also provides a preparation method for the nanoparticle and a use of the nanoparticle in preparation of a drug for treating breast cancer.

SUPRAMOLECULAR IONIZABLE LIPID MOLECULES WITH HETEROATOMIC TUNING FOR NUCLEIC ACID DELIVERY

Resumen de: WO2025123134A1

The present application relates to ionizable lipids that include ester functional groups. The present application further relates to compositions and uses thereof for the delivery of agents such as nucleic acids and drugs.

BREAST-CANCER-TARGETED MILK-DERIVED EXOSOME DRUG DELIVERY SYSTEM, AND PREPARATION METHOD THEREFOR AND USE THEREOF

Resumen de: WO2025123664A1

Disclosed are a breast-cancer-targeted milk-derived exosome drug delivery system, and a preparation method therefor and the use thereof, which belong to the technical field of pharmaceuticals. The method for preparing the breast-cancer-targeted milk-derived exosome drug delivery system involves: adding sodium citrate to animal milk to remove casein, performing differential centrifugation under a low-temperature condition by using a centrifuge, separating and enriching milk-derived exosomes, performing ultrasonic drug loading on the drug solution and the milk-derived exosome suspension at 37°C, and performing freeze drying to obtain drug-loaded exosomes. In the preparation method, the exosomes are obtained by taking animal milk as a source. The source of animal milk is abundant and the operation of the method is simple, which reduces the production cost. Moreover, natural ligands on the milk-derived exosomes can specifically target and bind to breast-cancer cells, so that uptake by cancer cells is enhanced, the cytotoxicity of the drug to normal cells is reduced, and the effect of specifically treating breast cancer is achieved. The present application is widely used in the field of targeted delivery of drugs for breast cancer treatment.

LIPID NANOPARTICLES LYOPHILIZATION METHODS AND COMPOSITIONS

Resumen de: AU2023393613A1

The invention provides a lyophilized nucleic acid lipid nanoparticle (NALNP) comprising (a) a lipid nanoparticle comprising a nucleic acid, and (b) a lyophilization buffer comprising a sugar, a lyophilization reagent, and a pharmaceutically acceptable diluent, as well as a method of preparing same.

Polymer Nanoaggregate Pharmaceutical Composition and Use Thereof

Resumen de: US2025195443A1

This disclosure is directed to a pharmaceutical composition for treating or preventing a disease. The pharmaceutical composition can comprise a polymer-drug nanoaggregate having a polymer and at least one bioactive agent that is water insoluble or poorly water soluble. The polymer is water soluble and comprises at least one first terminal group modified with H or a hydrophobic moiety and a second terminal group modified with a hydrophilic moiety and can be a modified symmetrically or asymmetrically branched polymers. This disclosure is also directed to a method for treating or preventing a disease including one or more immune disorders, infectious diseases and cancers using the pharmaceutical composition disclosed herein. The pharmaceutical composition can be a vaccine or an adjuvant for a vaccine.

ANTIBACTERIAL DRUG-ELUTING COMPOSITIONS AND METHODS

Resumen de: US2025195446A1

The present disclosure describes compositions that can be used in the treatment of an ocular injury or disease in a subject in need thereof. Methods of preparing the compositions are also described. The compositions can include one or more nanoparticles encapsulating a hydrophilic therapeutic agent; gelatin methacryloyl (GelMA); hyaluronic acid-glycidyl methacrylate (HAGM); and a visible light-activated photoinitiator.

CELL TYPE SELECTIVE DELIVERY OF EXOSOME CARGO USING NOTCH LIGAND-RECEPTOR SYSTEM

Resumen de: US2025195448A1

Provided herein are engineered exosomes and uses thereof for cell-type selective delivery of cargo. In particular, provided herein are engineered exosomes expressing a Notch ligand binding domain and uses thereof for targeted delivery of therapeutic cargo to cells expressing Notch ligand, such as neurons.

MELT PROCESSED VIRAL NANOPARTICLE CONSTRUCTS

Resumen de: US2025195449A1

A melt processed viral nanoparticle construct for delivery of virus or virus-like particles to a site of interest includes a degradable polymer matrix and a plurality of virus or virus-like particles encapsulated within the degradable polymer matrix. The nanoparticle construct upon administration to the site of interest providing a sustained release of the virus or virus-like particles and/or nanoparticles upon degradation of the polymer matrix.

Oral Rapamycin Nanoparticle Preparations and Use

Resumen de: US2025195435A1

Oral preparations of microcapsules and nanoparticles including an inhibitor of the mammalian target of rapamycin. The preparations are intended to assist with the treatment and prevention of cancer, neurocognitive dysfunction, genetically predisposed disorders, and age-related disorders. The embodiments discussed address the present need for alternative preparations or manufacturing processes that ensure efficacy while improving other performance characteristics such as storage stability, biodistribution, dosage cost, etc.

LIPID NANOPARTICLES FOR TARGETED DELIVERY OF MRNA

Resumen de: US2025195431A1

Disclosed are compositions including a pharmaceutical agent and a lipid composition; wherein the pharmaceutical agent is assembled with the lipid composition; and the lipid composition comprises a lipidoid having structural Formula (I):or a pharmaceutically acceptable salt thereof, wherein Ra, Rb1, Rb2, Rb3, Rb4, n1 and n2 are as described herein.

LNCRNA TRANSCRIPTS IN MELANOMAGENESIS

Resumen de: US2025197864A1

The invention provides compositions and methods for treatment of melanoma and other cancers. In particular, the invention provides a single or double-stranded nucleic acid that inhibits a certain group of long non-coding RNAs (lncRNAs) that have been discovered to be associated with melanoma. Inhibition of these lncRNAs in melanoma cells and xenograft mouse models leads to inhibition of cell proliferation, induction of apoptosis, and reduced cancer cell growth. The invention also relates to a method of inhibiting cancer cell growth with specific kinase inhibitors that have been found to show similar inhibition effects as the nucleic acids targeting the lncRNAs. The single or double-stranded nucleic acid and the specific kinase inhibitors constitute a novel therapeutic strategy in the treatment of melanoma and other cancers.

Anti-Peripheral Lymph Node Addressin Antibodies and Uses Thereof

Resumen de: US2025197508A1

The present disclosure provides, inter alia, anti-peripheral lymph node address in antibodies and antigen binding fragments thereof. The present disclosure also provides compositions comprising drug-containing polymeric particles that mimic lymphocyte migration in vivo and can specifically deliver immunosuppressive or immunoregulatory drugs to lymphoid tissues and sites of chronic inflammation where T-cell activation and T-cell mediated injury are occurring; such compositions comprise the antibodies or antigen-binding fragments thereof described in the disclosure. The present disclosure also comprises antibody-drug conjugates and compositions comprising the antibody-drug conjugates. Methods of preparing and using these antibodies, antigen-binding fragments thereof, and compositions thereof are also provided.

TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR

Resumen de: US2025197463A1

The present invention provides, among other things, an improved method of treating cystic fibrosis (CF) in a human subject. The method comprises administering a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein at a concentration of 0.5 mg/mL or greater to a human subject via nebulization. The composition is aerosolized using a nebulizer and a nominal dose of the mRNA is administered to the human subject via the nebulizer over a period of time, typically at least 30 minutes, at a suitable nebulization rate, e.g., at least 0.2 mL/minute.

Acid Degradable Solid Lipid Nanoparticles

Resumen de: US2025197348A1

Acid degradable solid lipid nanoparticles comprise PEG conjugated to cholesterol via an acid degradable linkage comprising an azide-benzaldehyde acetal.

GLYCOMIMETIC LIGANDS

Resumen de: US2025195689A1

Described herein are glycan compounds and particles comprising glycan compounds. The compounds and particles described herein are useful in methods of treating immune and inflammatory-related diseases. or a pharmaceutically acceptable salt thereof

GAS NANO/MICRO-BUBBLE COMPOSITIONS AND USES THEREOF

Resumen de: US2025195698A1

Provided herein are gas bubble populations optimized for certain imaging and therapeutic ultrasound uses, methods of preparing such populations, and methods of use thereof.

Multiplexed Analysis of Materials for Tissue Delivery

Resumen de: US2025195688A1

Disclosed herein are compositions and methods for identifying materials suitable for functional delivery of a bioactive agent to a target tissue. These compositions and methods have the advantage of simultaneously screening a library of materials for the ability to deliver a bioactive agent to a cell, tissue, or organ. The compositions and methods can also be used to confirm that the agent is delivered in a manner sufficient for function of the agent.

HUMANIZED ANTI-CD8 ANTIBODIES AND USES THEREOF

Resumen de: US2025195685A1

The present disclosure provides humanized antibodies and antigen binding domains thereof that bind CD8α and other antibody formats comprising these antigen binding domains, their use as a targeting moiety on lipid nanoparticles (tLNP) to deliver a therapeutic payload (such as a nucleic acid molecule) or other types of payloads. The present disclosure further relates to pharmaceutical compositions comprising the humanized anti-CD8α antibodies and CD8-targeted tLNP encapsulating a payload.

COMPOSITIONS AND METHODS OF TREATING MUSCLE ATROPHY AND MYOTONIC DYSTROPHY

Resumen de: US2025195676A1

Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscle atrophy or myotonic dystrophy.

METFORMIN NANOFORMULATIONS AND METHODS OF USE THEREOF

Resumen de: US2025195670A1

The present invention provides metformin nanoparticles and methods of use thereof.

SYNTHETIC PLATELETS

Nº publicación: US2025195621A1 19/06/2025

Solicitante:

CASE WESTERN RESERVE UNIV [US]
CASE WESTERN RESERVE UNIVERSITY

Resumen de: US2025195621A1

A synthetic platelet including a biocompatible flexible nanoparticle, the nanoparticle having an outer surface and a plurality of site targeted peptides conjugated to the surface, the synthetic platelet also including a therapeutic agent, wherein the therapeutic agent is encapsulated by the nanoparticle, wherein the synthetic platelet adheres to the site targeted and promotes delivery of the therapeutic agent onto sites of the synthetic platelet adhesion, and wherein the therapeutic agent is released at the site targeted via a site-relevant enzyme.