Resumen de: WO2025244940A1
Disclosed herein are compositions and methods for the treatment of hepatitis B infection, including chronic hepatitis B (CHB).
Resumen de: WO2025243932A1
The present invention provides a polyion complex comprising: a block copolymer that has a hydrophilic polymer segment and a cationic poly(amino acid) segment; and a nucleic acid. The cationic poly(amino acid) segment includes an amino acid residue A that is selected from an amino acid residue having an achiral carbon atom as a carbon atom constituting the main chain and a polar non-charged amino acid residue, and also includes an amino acid residue B having a cationic group in a side chain.
Resumen de: WO2025242237A1
An engineered cell membrane nanovesicle and a preparation method therefor and use thereof. A use of normal fibroblasts in the preparation of a drug or tumor diagnostic reagent or nano-drug delivery system or engineered vesicle for treating tumors. The vesicle prepared using the normal fibroblasts has high specificity and the ability to quickly target various types of tumor tissues, and can achieve tumor targeted diagnostic imaging or drug delivery; also, the accumulation in normal organs and non-targeted tissues is greatly reduced, the toxic side effects in vivo are minimized, and safety and effectiveness are greatly improved.
Resumen de: WO2025242194A1
A latanoprost ophthalmic gel, comprising the following raw materials: 0.001-0.01% of latanoprost, 0.01-0.1% of a gel matrix, 0.1-5.0% of a nonionic surfactant, 0.1-5.0% of an osmotic pressure regulator, 0.01-1.0% of a pH regulator, and the balance of water for injection. The raw materials do not comprise a bacteriostatic agent. Not adding the bacteriostatic agent can fundamentally eliminate toxic side effects caused by the bacteriostatic agent, avoid adverse reactions to users, and improve product safety; and the non-ionic surfactant can form nanomicelles during the preparation process of the gel to exert a solubilizing effect, so as to overcome the problems of reduced solubility and poor stability of latanoprost caused by the absence of the bacteriostatic agent.
Resumen de: WO2025242219A1
Disclosed are a chloroquine structure-containing compound, a composition containing same, and an application thereof. The present invention provides a compound as represented by formula (I) or a pharmaceutically acceptable salt thereof. The chloroquine structure-containing compound provided by the invention is a nano material obtained from the synthesis of chloroquine, a derivative thereof, and a lipid tail. The nano material can be applied to gene therapy, drug delivery, and the like. A lipid nanoparticle (LNP) formed from the nano material and mRNA has a novel nanoscale spatial structure different from that of a traditional LNP, and compared to a traditional LNP, the nano material has better pharmaceutical properties. The LNP exhibits an immunosuppressive function, avoids triggering an excessive inflammatory response, and has a higher safety profile.
Resumen de: WO2025242083A1
Provided in the present invention is a functional material capable of self-assembling into nanoscale dimensions, with an amino acid or polypeptide as a core backbone, a hydrophobic segment at one end and a hydrophilic segment at the other end. The material can be used in fields such as drug delivery through self-assembly into nanoscale forms. Formula (I).
Resumen de: WO2025241798A1
The present invention relates to the technical field of pharmaceutical formulations, and provides a dual-targeted nanoparticle, a preparation method therefor, and a use thereof. The dual-targeted particle comprises a browning inducer, i.e., Chiglitazar sodium, a photothermal reagent, i.e., indocyanine green, an adipocyte homing peptide, a cell penetrating peptide, a DSPE-PEG polymer, and a lipid matrix. The browning inducer and the photothermal reagent are encapsulated into a nanoparticle having excellent biocompatibility, and the surface of said nanoparticle is coated with the functionalized adipose homing peptide and the cell penetrating peptide to construct the dual-targeted nanoparticle. The combination of the dual-targeted nanoparticle and photothermal-drug therapy can effectively promote "browning" of white adipose, reduce weight and enhance glycolipid metabolism, and lay a foundation for developing safe and effective drugs against obesity and related metabolic disorders.
Resumen de: US2025360203A1
Disclosed is an mRNA composition containing: an mRNA sequence comprising, in order from 5′ to 3′, a 5′ cap region, a 5′ UTR region, a start codon region, a 3′ UTR region, and a poly (A) tail region; and an antisense oligonucleotide containing a region complementary to the 5′ cap region. The mRNA composition containing the mRNA sequence and the antisense oligonucleotide containing a region complementary to the 5′ cap region of the mRNA sequence may regulate the translation rate of the mRNA, enables selective protein expression based on the type of nucleotide modification and DNA repair mechanism, and may improve the stability of the mRNA against RNA-degrading proteins, thereby improving the stability and efficiency of mRNA vaccines or therapeutics.
Resumen de: US2025361511A1
Provided here are nanoparticle compositions containing siRNA to disrupt the signal regulatory protein-α (SIRPα) signaling pathway. Embodiments include methods for treating a subject diagnosed as having ovarian cancer by administering to the subject the nanoparticle composition containing SIRPα siRNA. Other methods include administering to the subject the nanoparticle composition containing SIRPα siRNA in addition to a platinum-based chemotherapeutic agent.
Resumen de: US2025361208A1
Provided herein are lipids having the Formula (I):and pharmaceutically acceptable salts thereof, wherein R1, R2, a, and b are as defined herein. Also provided herein are lipid nanoparticle (LNP) compositions comprising lipid having the Formula (I) and a capsid-free, non-viral vector (e.g., ceDNA). In one aspect of any of the aspects or embodiments herein, these LNPs can be used to deliver a capsid-free, non-viral DNA vector to a target site of interest (e.g., cell, tissue, organ, and the like).
Resumen de: US2025361270A1
Disclosed are peptides and peptidomimetics that in some embodiments include the amino acid sequence KRGARST or (SEQ ID NO: 1), AKRGARSTA or (SEQ ID NO: 2), or CKRGARSTC (SEQ ID NO: 3). Also disclosed are conjugates and compositions that include the peptides and/or peptidomimetics, methods for directing a moiety to tumor lymphatic vasculature, methods for imaging tumor lymphatic vasculature, methods for reducing or inhibiting tumor metastasis, methods for reducing the number of tumor lymphatic vessels, methods for treating cancer, methods for treating a disease or disorder associated with a gC1q/p32 receptor biological activity, methods for detecting the presence of a gC1q/p32 receptor, methods for detecting interactions between gC1q/p32 receptors and the presently disclosed conjugates and compositions, methods for delivering the presently disclosed conjugates and compositions to gC1q/p32 receptors, methods for assessing gC1q/p32 receptor levels in cells, methods for identifying subjects having diseases associated with gC1q/p32 receptor biological activities, and methods for screening for compounds that interact with gC1q/p32 receptors.
Resumen de: US2025360197A1
The Human Immunodeficiency Virus (HIV) infection and recurrent infection prevention mRNA vaccine comprising epitopes of HIV-1 and HIV-2 viruses and their corresponding Nef proteins.
Resumen de: US2025360199A1
This disclosure generally relates to methods and compositions for eliciting broad and robust immune responses to a protein of interest. The methods employ both DNA and RNA-based vaccines that encode at least a portion of the protein of interest.
Resumen de: US2025360140A1
The disclosure provides the use of particular substituted heterocycle fused gamma-carboline compounds as pharmaceuticals for the treatment of residual symptoms of psychosis or schizophrenia. The disclosure also provides novel long acting injectable formulations of particular substituted heterocycle fused gamma-carboline compounds and use of such long acting injectable formulations for the treatment of residual symptoms of psychosis or schizophrenia.
Resumen de: US2025360176A1
A medicinal chewing gum has an inner core containing a first gum base and a first cannabinoid in a lipophilic nanosized form and an outer layer containing a second gum base and a second cannabinoid in a hydrophilic nanosized form, thereby providing quick release of the second cannabinoid in the outer layer and sustained release of the first cannabinoid in the inner layer. At least one of the inner core and the outer layer contains a synergistic compound having a synergistic effect with at least one of the first and second cannabinoids in the treatment of a medical condition. At least one of the first cannabinoid and the second cannabinoid are a cannabinoid other than cannabidiol (CBD).
Resumen de: US2025360213A1
Materials and methods for enhancing the effectiveness of proton radiation therapy (e.g., high linear energy transfer (LET) proton radiation therapy) against tumor cells are provided herein.
Resumen de: US2025360089A1
Aspects of the disclosure relate to particle formulations, e.g., nanoparticle formulations, methods of making particle formulations, and methods for delivery of oligonucleotides and/or synthetic RNA, e.g., for increasing gene expression in a targeted manner. In some embodiments, compositions and methods are provided that are useful for posttranscriptionally altering protein and/or RNA levels in a targeted manner. Aspects of the disclosure described herein provide compositions and methods that are useful for protecting RNAs from degradation (e.g., exonuclease mediated degradation).
Resumen de: US2025360087A1
Drug delivery systems are needed to assist in improving the therapeutic characteristics of pharmaceutical agents. Provided is a dry composition, comprising a polysaccharide, such as inulin, and lipid droplets, wherein the lipid droplets are encapsulated within polymeric chains of the polysaccharide, and wherein the polysaccharide is not in a nano-particulate form. The use of such compositions enables efficient delivery of agents, such as poorly-water soluble drugs and antibiotics.
Resumen de: US2025360086A1
Provided herein is a device for producing an aqueous-core polymeric-shell particle as described herein. Also provided are methods of preparing such particles, as well as the particles themselves. The particles are useful in medicine, particular in the context of vaccines.
Resumen de: US2025360083A1
Disclosed herein are lipid nanoparticles comprising plurality of lipids, a targeting moiety for an HIV-1 chemokine receptor, and a CRISPR nucleic acid complementary to an HIV-1 gene, pharmaceutical compositions and methods of use thereof.
Nº publicación: AU2025263792A1 27/11/2025
Solicitante:
AKOUOS INC
Akouos, Inc
Resumen de: AU2025263792A1
COMPOSITIONS AND METHODS FOR TREATING NON-AGE- ASSOCIATED HEARING IMPAIRMENT IN A HUMAN SUBJECT Provided herein are compositions that include at least two different nucleic acid vectors, where each of the at least two different vectors includes a coding sequence that encodes a different portion of an otoferlin protein, and the use of these compositions to treat hearing loss in a subject. COMPOSITIONS AND METHODS FOR TREATING NON-AGE- ASSOCIATED HEARING IMPAIRMENT IN A HUMAN SUBJECT Provided herein are compositions that include at least two different nucleic acid vectors, where each of the at least two different vectors includes a coding sequence that encodes a different portion of an otoferlin protein, and the use of these compositions to treat hearing loss in a subject. ov - - o v