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WO/2024/077777A1 |
Disclosed in the present invention are a multifunctional recombinant antibody, and a preparation method and use therefor. Provided is a monoclonal antibody capable of specifically recognizing human CD276 protein, also provided is a human...
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WO/2024/054536A3 |
A system comprised of customized nanoscopic projectiles directed to target cells. Projectiles are metered into a central force accelerator, comprised of hundreds to thousands of concurrent channels. Asperities of the channels' surface, t...
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WO/2024/079249A1 |
The invention relates to a hybrid AAV vector comprising a transgene of interest operably linked to an hepatocyte-specific promoter, which enhances transgene expression in the liver and its use for liver-directed gene therapy.
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WO/2024/031059A3 |
The present disclosure relates to a method of inducing cellular rejuvenation and/or regeneration of a cell or tissue comprising contacting the cell with an effective amount of a protein or nucleic acid encoding a protein that induces cel...
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WO/2024/081892A1 |
Provided herein are nucleic acid molecules encoding an HPV antigen. Also provided are vaccines against human papillomavirus (HPV) comprising the nucleic acids, methods of inducing immune responses, and methods for prophylactically and/or...
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WO/2024/049971A3 |
Embodiments of the disclosure encompass methods and compositions related to modified cells and uses thereof, wherein the cells are engineered with respect to expression of B-cell translocation gene 1 (BTG1). In specific embodiments, the ...
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WO/2024/079352A1 |
The present invention relates to pharmaceutical compositions comprising at least one lipid nanopartide (LNP) specific for targeting Langerhans cells (LC) wherein the LNP encapsulates at least one mRNA, is capable of specifically binding ...
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WO/2024/081954A2 |
This disclosure relates to isolated oligonucleotides comprising duplex regions targeting human complement C3 mRNA, and delivery systems, kits and compositions comprising the same, and methods of using the same for inhibiting or downregul...
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WO/2024/081922A1 |
Described herein are mRNA carrier systems and methods of their use for entering the cytoplasm and expressing carried mRNA, to induce a tumor killing effect. The mRNA carrier systems can comprise DNA or RNA nanostructures. Also described ...
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WO/2024/081934A1 |
Described herein are methods and compositions for treating Alzheimer's Disease (AD), as well as compositions comprising a reelin-derived peptide and methods of use thereof.
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WO/2024/078381A1 |
The present invention belongs to the technical field of tumor treatment, and specifically relates to an antibody or an antigen binding fragment thereof that bind to human CD39, and a preparation method therefor and the use thereof. The a...
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WO/2024/078127A1 |
The present invention relates to an HIF2α gene mutant, a vector containing the mutant, and the use of the mutant in the construction of a non-alcoholic fatty liver disease (NAFLD) mouse model. The genetically mutated mouse constructed b...
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WO/2024/081876A1 |
Extracellular vesicles (EVs) are natural liposome-like vesicles secreted by cells into the extracellular space. Provided herein are techniques to enrich cargo-loaded EVs over non-loaded EVs and contaminants. To achieve EVs were engineere...
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WO/2024/044542A3 |
Described are methods for treating diseases and disorders that can be mediated in part by a reduction in ANGPTL3 gene expression in a human subject in need of treatment, using pharmaceutical compositions that include ANGPTL3 RNAi agents....
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WO/2024/011135A3 |
Aspects of the disclosure relate to oligonucleotides (e.g., RNAi oligonucleotides such as siRNAs) designed to target DUX4 RNAs and targeting complexes for delivering the oligonucleotides to cells (e.g., muscle cells) and uses thereof, pa...
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WO/2024/044741A3 |
Described herein are highly pure, chemically synthesized, stabilized, 5'-capped oligonucleotides. Additionally, described herein are methods for making and using said oligonucleotides.
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WO/2024/079064A1 |
The invention relates to the field of transposases. In particular, it provides a polypeptide comprising or consisting of a Tc1/mariner superfamily transposase, e.g., a Sleeping Beauty transposase, having a substitution in amino acid posi...
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WO/2024/078345A1 |
Provided are an snRNA nucleic acid molecule and an application thereof. The snRNA nucleic acid molecule comprises: a recognition domain, a stem-loop sequence, and an Sm sequence; wherein the number of recognition domains is at least two;...
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WO/2024/077773A1 |
Provided are an anti-human IL-2 monoclonal antibody, an antibody complex and use thereof. Once the anti-human IL-2 monoclonal antibody binds to IL2, binding of IL2 to IL2Rβ/γ (CD122/132) is maintained but binding of IL-2 to IL-2Rα (CD...
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WO/2024/079667A1 |
The application relates to nucleic acid regulatory elements (NAREs) that are able to enhance expression of genes in the central nervous system. The application further relates to methods employing NAREs and uses of the NAREs. Expression ...
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WO/2024/079592A1 |
Methods for treating B-cell malignancies such as relapsed and/or refractory B-cell malignancies with a population of genetically engineered T cells expressing a chimeric antigen receptor (CAR) targeting CD19 and having multiple genetic e...
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WO/2024/081706A1 |
The disclosure provides gene therapy vectors, such as adeno-associated virus (AAV), designed for treatment of an immunoglobulin-μ binding protein 2 (IGHMBP2)-related disorder.
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WO/2024/078420A1 |
An isothermal nucleic acid amplification RAA primer probe combination for detecting Candida auris, and the use thereof. The primer probe combination has a high amplification efficiency, a strong specificity, and no obvious false positive...
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WO/2024/077776A1 |
Provided are a bifunctional molecule formed by connecting an Fc mutated antibody to a cytokine, and use of the bifunctional molecule in the preparation of a therapeutic antibody drug. These Fc mutated monoclonal antibodies or monoclonal ...
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WO/2024/081861A1 |
Disclosed herein are 2 novel long non-coding RNAs (lncRNAs), TROLL-2C and TROLL-3A. It is shown herein that lncRNAs TROLL-2C and TROLL-3A are suitable targets for cancer therapies and can be used to make prognostic determinations about a...
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WO/2024/059635A3 |
The invention relates to methods of generating an immune response for the treatment or prevention of a pathogenic infection. The invention also relates to methods of generating MHC-I, MHC-II, and/or MHC-E restricted CD8+ and/or CD4+ T ce...
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WO/2024/077774A1 |
Provided is a monoclonal antibody capable of specifically recognizing human CD73 protein and a humanized recombinant antibody thereof, and a reconstructed multifunctional antibody capable of recognizing human CD73 and having an IL15 func...
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WO/2024/077775A1 |
Disclosed are a multifunctional recombinant antibody, a preparation method therefor, and use thereof. Provided is a monoclonal antibody able to specifically recognize human HER3 protein. Also provided is a humanized recombinant antibody ...
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WO/2024/079076A1 |
dsRNAi oligonucleotides can be used in methods for the treatment of the advanced fibrotic and/or cirrhotic stages of NASH.
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WO/2024/026455A3 |
The present disclosure relates, in part, to methods and compositions for generating monocytes, macrophages, and/or precursors thereof (such as an induced pluripotent stem cell), comprising (i) a chimeric antigen receptor (CAR); (ii) a po...
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WO/2024/081937A2 |
Disclosed herein are Cas12a fusion proteins. The Cas12a fusion proteins may comprise, for example p300 or SID. Further provided are DNA constructs encoding at least one gRNA, such as an array of multiple gRNAs. The Cas12a fusion proteins...
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WO/2024/081551A1 |
Provided herein are methods for purifying recombinant adeno-associated virus (rAAV) particles using chromatography in a large scale. Further provided include the rAAV particles and the pharmaceutical composition prepared by the method.
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WO/2024/030924A3 |
Provided herein are high affinity dimer CEACAM1 variants. These variants enhance the hCEACAMl mediated modulation of immunity in the treatment of infectious agents, autoimmunity, inflammation and cancer.
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WO/2024/080305A1 |
According to one embodiment of the present invention, a bioactive protein that usually has a low expression level and/or low activity when expressed as a recombinant protein using a host cell such as a CHO cell can be expressed in the fo...
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WO/2024/081381A1 |
Provides herein are multifunctional molecules comprising T cell receptor variable beta-binding moieties and cytokines, and methods of treating conditions or diseases in a subject using the same.
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WO/2024/079071A1 |
dsRNAi oligonucleotides can be used in methods for the treatment of NASH, preferably the advanced fibrotic and/or cirrhotic stages thereof, using dosage regimens comprising a loading phase followed by a maintenance phase.
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WO/2024/079665A1 |
The application relates to nucleic acid regulatory elements that are able to enhance expression of genes constitutively in a variety of tissues, or in particular tissues including liver, muscle, and the CNS. The application further relat...
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WO/2024/030824A3 |
The invention relates to novel regulatory sequences which mediate expression of an operably-linked protein-encoding polynucleotide of interest, wherein the polynucleotide is transcribed in all plant tissues except male reproductive tissu...
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WO/2024/081927A1 |
Provided herein are methods of improving (e.g., increasing) the production of viral particles (e.g., AAV) in mammalian cells (e.g., HEK293 or HEK293T cells) using selective HDAC6 inhibitors (e.g., compounds of Formula I, Formula II, or F...
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WO/2024/077439A1 |
The present application discloses a method for constructing a single-cell transcriptome and chromatin accessibility dual-omics single-cell sequencing library. The method comprises: a) preparing a single-cell suspension; b) obtaining an o...
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WO/2024/044689A3 |
Provided herein, in some embodiments, are nucleic acid constructs encoding therapeutic nucleic acids (e.g., miRNAs) of interest comprising one or more alternatively-spliced exons that regulate the expression of proteins or RNAs of intere...
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WO/2024/079331A1 |
The invention relates to the field of drug delivery for inflammatory diseases, more specifically inflammatory kidney, eye, lung, and intestinal diseases, and kidney, eye, lung and intestinal diseases in general.
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WO/2024/079078A1 |
Disclosed are methods and compositions relating to antisense therapy for treating epilepsy, such as a focal epilepsy and temporal lobe epilepsy, in a subject in need thereof by targeting GRIK2 mRNA. In particular, the disclosure provides...
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WO/2024/081879A1 |
This invention relates to compositions and methods comprising epigenetic editors for epigenetic modification of CD247, as well as nucleic acids and vectors encoding the same. Also disclosed are cells epigenetically modified by the epigen...
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WO/2024/078017A1 |
Provided are a chicken-derived anti-human TK1 recombinant IgY single-chain antibody, a chicken-derived anti-human TK1 recombinant full-length monoclonal IgY antibody, and a use. On the basis of an improved phage display method, an anti-h...
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WO/2024/078124A1 |
Provided in the present invention is the use of a TDGF1 gene in the preparation of a drug for treating aging-related diseases or reversing cell aging. Compared with the prior art, the present invention has the following advantages: the u...
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WO/2024/080866A1 |
The present invention relates to a composition for use in the production of polyomaviral vector particles, the composition comprising a first DNA construct comprising recombinant DNA and a polyomaviral intergenic region, wherein said fir...
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WO/2024/081352A2 |
The present disclosure provides third-generation self-inactivating (SIN) lentiviral vectors comprising a nucleic acid sequence encoding MyoVIIa isoform 1 for treatment of Usher IB syndrome in a subject in need thereof. In some embodiment...
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WO/2024/030930A3 |
Approximately twelve million U.S. residents suffer from vision loss due to an untreatable blinding disorder, compositions and methods for inducing efficient conversion of retinal glia into photoreceptors or retinal ganglion cells. New tr...
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WO/2024/077672A1 |
Provided are cAMP green fluorescent probes G-Flamp2 and G-Flamp2b and the use thereof, and a kit. The cAMP fluorescent probe G-Flamp2 has an amino acid sequence as shown in SEQ ID NO: 1; and the other cAMP fluorescent probe G-Flamp2b has...
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