Resumen de: US2025352575A1
A method of manufacture and compositions and uses of a multifunctional bioinorganic theranostic nanoconstruct are disclosed. Nanoconstructs described herein contain manganese dioxide in a biocompatible matrix and are useful for, e.g., MRI contrast imaging of tumor environments and enhancement of radiation therapy in cancer. Some nanoconstructs described herein incorporate targeting agents for specific targeting of cancer cells.
Resumen de: US2025352666A1
The present disclosure relates to pharmaceutical compositions comprising a lipid nanoparticle (LNP), a therapeutic nucleic acid (TNA) and at least one pharmaceutically acceptable excipient, wherein the LNP comprises at least one lipid, and wherein the LNP is capable of delivering the TNA to a retinal cell. Methods of treating ocular diseases and disorders are also provided.
Resumen de: US2025352637A1
The invention relates to a method of treating a disease or disorder in a patient in need thereof that includes providing an active pharmaceutical ingredient (API) to the patient by administering more than one split-dose of the API over a pre-determined period of time. In embodiments of the invention, the API is an mRNA encoding an antigen. The attractiveness of mRNA as a vaccine modality is supported by several advantages. As a non-infectious agent that does not require incorporation into the host's genome to confer activity along with its well-defined chemical composition, mRNA is regarded as a relatively safe vaccine modality.
Resumen de: US2025352664A1
Methods of administering lipid nanoparticle compositions including poly(ethyloxazoline)-lipid conjugates (PEOZ-lipid LNPs) without triggering a PEOZ-associated immune response. In some aspects, the PEOZ-lipid LNPs trigger a protective response based on the encapsulated payload but do not trigger an IgM or IgG response. also provides an absent or. In other aspects, the PEOZ-lipid LNPs trigger a protective response based on the encapsulated payload but trigger an IgM and IgG response that is markedly reduced (as compared to a comparable PEG-lipid currently used in LNP vaccine delivery systems). PEOZ-DMA LNPs incorporating payloads such as oligonucleotides payloads mRNA, DNA, and siRNA for delivery into living cells is also contemplated.
Resumen de: US2025352663A1
Compositions, methods, and kits are provided for treating infections and cancer with metallic nanoclusters. In particular, metallic nanoclusters having a size of less than 10 nm that are conjugated to adenosine triphosphate (ATP) or an analogue thereof can be used to eradicate a cell in a growth arrest phase such as infectious bacterial or fungal cells. Such nanoclusters can also induce endoplasmic reticulum stress and inhibit growth of cancerous cells. Additionally, such metallic nanoclusters can be used to inhibit a purinergic P2X7 receptor and FtsH protease.
Resumen de: US2025352487A1
Provided herein are compositions and methods of delivering nucleic acids, such as therapeutic mRNAs and DNA, to the central nervous system by delivery, e.g., intrathecally or intracerebrally, of nucleic acid containing lipid nanoparticles using sphingolipids as helper lipids.
Resumen de: US2025352489A1
A gene delivery system and applications thereof in the technical field of biological medicines that is particularly useful in the preparation of drugs for treatment of tumors are disclosed. The gene delivery system and applications relate to technology for inducing the differentiation of malignant tumor cells into mature cells, in which regulating the expression of HNF4 alpha protein in the malignant tumor cells using messenger ribonucleic acid, the malignant phenotype of the malignant solid tumor cells is inhibited, and the effective treatment of the malignant solid tumors is achieved. The gene delivery system and applications are therefore applicable to a method of preparing a drug for treating malignant solid tumors and to a method of treating a patient having a malignant solid tumor.
Resumen de: US2025352507A1
The present invention relates generally to hybrid polymer (e.g., polyphosphazene) based drug delivery platforms and to methods of producing, evaluating, administering, and treating subjects with the same. More particularly, the present invention provides polyphosphazene based drug delivery platforms comprising one or more polyphosphazenes with controlled molecular weights and/or polydispersities as well as selective methods for associating one or more therapeutic drug (or prodrug) substances to the polyphosphazenes.
Resumen de: US2025352490A1
Particles made of a polymeric matrix having associated therewith a therapeutically active agent usable in treating a medical condition associated with an overexpression of P-selectin in a subject in need thereof and featuring a P-selectin selective targeting moiety represented by Formula I as defined and described in the specification and claims, compositions comprises these particles and uses thereof, are provided.
Resumen de: US2025352491A1
The present disclosure is drawn to nanoparticles, method of using and making thereof. The disclosed nanoparticles comprise nanoparticles, that may be rod-shaped and/or coated on the outer surface with albumin, that are designed to effectively cross lymphatic barriers as an effective strategy to improve the efficacy and translatability of putative immunotherapies. Delivery systems or pharmaceutical compositions comprising the disclosed nanoparticles are also provided.
Resumen de: US2025352488A1
One or more ionizable lipid(s) and lipid nanoparticles comprising same are provided. Pharmaceutical compositions comprising the lipid nanoparticles encapsulating an active agent are also provided.
Resumen de: US2025352543A1
A nanocrystalline preparation and a preparation method therefor, the nanocrystalline preparation containing a ROCK2 inhibitor and a stabilizer. The present invention also relates to use of the nanocrystalline preparation in the prevention, alleviation, and/or treatment of selected diseases and medical conditions, especially diseases such as idiopathic pulmonary fibrosis, fatty liver disease and/or steatohepatitis, post-hematopoietic stem cell transplantation graft versus host disease or viral infection.
Resumen de: US2025352537A1
The present invention relates to solid phase pharmaceutical compositions of 6′-fluoro-N-(4-fluorobenzyl)-4′-oxo-3′,4′-dihydro-1′H-spiropiperidine-4,2′-quinoline-1-carboxamide that is useful as a AKR1C3 dependent KARS inhibitor. The present invention also relates to processes for the preparation of said pharmaceutical compositions of said compound, methods of using said pharmaceutical compositions in the treatment of various diseases and disorders, and their use in diseases and disorders mediated by an AKR1C3 dependent KARS inhibitor.
Resumen de: US2025352473A1
Disclosed herein are compositions comprising nanoliposomes useful for the treatment and prevention of cerebrovascular and aging-related degenerative diseases.
Resumen de: US2025354174A1
Nanoparticle compositions for delivery of nucleic acids to subjects including carriers comprising sugar functionalized nucleic acid carriers, and therapeutic or immunogenic nucleic acid agents enclosed within the delivery molecules are described. Methods for treating or preventing diseases or conditions in a subject by administering the nanoparticle compositions that provide immune responses and synergistic therapeutic or preventive effects are provided.
Resumen de: US2025353931A1
The present disclosure relates to a molecular delivery system that facilitates the internalization of LNPs into a specific target of choice, such as a specific cell type, ex vivo and/or in vivo. The present disclosure also relates to methods, molecules, and compositions for enhancing the targeted delivery of compounds within a living system. In particular, embodiments provided herein relate to methods, molecules, and compositions for the targeted delivery of lipid nanoparticles containing therapeutic molecules into a cell or system of choice, such as a T cell. The present disclosure also relates to methods of administering the enhanced targeting system to a patient or system, compositions for use in such methods, and further methods of use of the targeting system as part of T cell-based immunotherapy.
Resumen de: US2025354140A1
The disclosure is generally related to calcium salted spherical nucleic acids (SNAs). SNAs comprise a nanoparticle core surrounded by a shell of oligonucleotides. In some aspects, the disclosure provides a spherical nucleic acid (SNA) comprising: (a) a nanoparticle core; and (b) a shell of oligonucleotides attached to the external surface of the nanoparticle core, wherein one or more oligonucleotides in the shell of oligonucleotides comprises a phosphate backbone; the SNA comprising Ca2+ ions adsorbed to the phosphate backbone of one or more oligonucleotides in the shell of oligonucleotides. Methods of making and using the SNAs are also provided herein.
Resumen de: US2025354138A1
This disclosure provides compositions and methods for the editing of a SERPINA1 gene with prime editing.
Resumen de: US2025352486A1
The present invention is based on a nanodevice comprising a nanoparticle comprising a therapeutic agent, a molecular drill comprising a compound with catalytic activity, and a self-propulsion system. The molecular drill in combination with the self-propulsion system allows breaking the matrix of biofilms formed by infectious microorganisms with high efficiency and the molecular drill, in turn, allows the therapeutic agent to be released from the nanoparticle under specific conditions, preferably in the acidic environment of an infection. The invention relates to the nanodevice, to the nanodevice used in the treatment of an infection caused by biofilms, and to the use of the nanodevice to disinfect samples in vitro or inert materials ex vivo.
Resumen de: WO2024229321A1
This disclosure relates to delivery vehicles comprising payload molecules, e.g., mRNA for the treatment of cystic fibrosis (CF). Nucleic acid therapeutics (e.g., mRNAs) when administered in vivo encode cystic fibrosis transmembrane conductance regulator (CFTR). Nucleic acid therapeutics (e.g., mRNAs) of the disclosure increase and/or restore deficient levels of CFTR expression and/or activity in subjects.
Resumen de: AU2025260005A1
Disclosed herein are spherical high-density lipoprotein-like nanoparticles (HDL-NP) having a soft material core (e.g., a lipid-conjugated inorganic core). Also disclosed herein are methods for synthesizing the spherical HDL-NPs. Also disclosed herein are methods for treating disorders such as cardiovascular disease, cancer, inflammatory disorders or reducing NF-kB activity with the spherical HDL-NPs. Disclosed herein are spherical high-density lipoprotein-like nanoparticles (HDL-NP) having a soft material core (e.g., a lipid-conjugated inorganic core). Also disclosed herein are methods for synthesizing the spherical HDL-NPs. Also disclosed herein are methods for treating disorders such as cardiovascular disease, cancer, inflammatory disorders or reducing NF-kB activity with the spherical HDL-NPs. ov o v
Resumen de: US2025353809A1
Disclosed are a nitrogen-containing chain compound, a preparation method, a composition containing said compound, and a use thereof. Provided is a nitrogen-containing chain compound represented by formula (I) or a pharmaceutically acceptable salt thereof. The nitrogen-containing chain compound represented by formula (I) may be used for preparing a lipid carrier. The prepared lipid carrier can encapsulate a nucleic acid drug, and may be used for delivering a nucleic acid prophylactic agent and/or therapeutic agent to mammalian cells and organs and producing an effect.
Resumen de: CN120569192A
The invention relates to an iron nanocluster having the following features:-the surface of which is covered with a mixed layer comprising histidine (His), acetate ions (Ac) and ascorbate ions (Asc),-which has a spherical shape,-the hydrodynamic diameter of which is 0.6-2.0 nm, preferably less than 1.0 nm,-the metal core diameter of which is 0.5-1.5 nm, preferably less than 1.0 nm,-the metal core diameter of which is 0.5-1.5 nm, preferably less than 1.0 nm,-the surface of which is coated with a mixed layer comprising histidine (His), acetate ions (Ac) and ascorbate ions (Asc),-which has a spherical shape. The nanocluster has a stability duration of 5 to 20 weeks when the nanocluster is in liquid form and stored at a temperature of 4 DEG C, a stability duration of at least 12 months, preferably 12 to 18 months, when the nanocluster is in dry form and stored at a temperature of 4 DEG C and under nitrogen gas, and exhibits spectrophotometric properties, and the nanocluster has a stability duration of 5 to 20 weeks when the nanocluster is in liquid form and stored at a temperature of 4 DEG C and under nitrogen gas, preferably 12 to 18 months when the nanocluster is in dry form and stored at a temperature of 4 DEG C and under nitrogen gas. The iron nanocluster is characterized in that the shoulder peak in the UV-visible spectrum is located at 300 +/-15 nm, the excitation wavelength of the fluorescence spectrum is 364 +/-15 nm, the emission wavelength is 415 +/-15 nm, and the formul
Resumen de: CA3273638A1
The invention relates to methods and materials for use in treating neurodegenerative diseases associated with pathological protein aggregates and provides fusion proteins comprising: (i) a first portion comprising a sequence of a protein which aggregates pathologically in neurodegenerative disease, such as tau protein, and (ii) a second portion comprising a RING-type E3 ubiquitin ligase, a component of a RING-type E3 ubiquitin ligase complex, or a domain of either. These fusion proteins have utility in selectively or preferentially targeting pathogenic protein aggregates for degradation.
Nº publicación: JP2025537759A 20/11/2025
Solicitante:
モティジェニックスシンガポールピーティーイー.リミテッド
Resumen de: MX2025005463A
Compounds, compositions, uses, and methods for reducing obesity in a subject, or for preventing or treating obesity, are provided herein. In certain examples, methods for reducing obesity in a subject and/or for preventing or treating obesity in a subject in need thereof are provided which may include a step of treatment with a GDP-bound form of Rab1a (Rab1aGDP), one or more expressible nucleic acids encoding Rab1aGDP, or a combination thereof.