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WO/2024/079132A1 |
The invention relates to an adeno-associated virus (AAV) capsid fusion protein comprising comprises an insert comprising a binding domain for a cell surface molecule, in particular, a variable immunoglobulin domain of a heavy chain antib...
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WO/2024/081970A2 |
The present invention relates to a series of antisense oligonucleotides containing morpholine 3- thiophosphoramidate nucleotides or morpholine 3-thiophosphoramidate and DNA/RNA (modified) nucleotides having phosphorothioates (pS) linkers...
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WO/2024/078626A1 |
Provided is a method for enhancing gene knock-in efficiency mediated by a CRISPR gene editing system in cells, comprising placing the cells in contact with a DNA-dependent protein kinase (DNA-PK) inhibitor and a cell division cyclin 7 (C...
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WO/2024/079530A2 |
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 the muscle. The application further relates to methods em...
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WO/2024/077702A1 |
An alkaline protease mutant having an improved enzyme activity obtained by means of iterative saturation mutagenesis using overlapping PCR technology, and a preparation therefor and the use thereof. The alkaline protease gene apr from Ba...
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WO/2024/081915A1 |
The present disclosure provides capture proteins for detection of antibodies specific for Kawasaki disease, methods of using the antibodies, and kits comprising the same.
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WO/2024/080275A1 |
The present invention addresses the problem of providing a method whereby environmental DNA or environmental RNA can be captured using a carrier having a high holding force, and the captured environmental DNA or environmental RNA can sub...
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WO/2024/081156A1 |
The present disclosure relates to compositions of matter and assay methods used to detect one or more non-nucleic acid targets of interest in a sample. The compositions and methods provide signal boost upon detection of non-nucleic acid ...
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WO/2024/080682A1 |
The present invention provides a light chain mutant of botulinum toxin obtained by substituting a specific lysine with arginine in the light chain of botulinum toxin. The light chain mutant of botulinum toxin according to the present inv...
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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/2024/079249A1 |
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/2024/081892A1 |
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/2024/079352A1 |
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/2024/079064A1 |
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/2024/077774A1 |
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/2024/081937A2 |
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/2024/080305A1 |
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/2024/081927A1 |
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/2024/079331A1 |
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/2024/077672A1 |
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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WO/2024/081756A2 |
Disclosed herein are genetically engineered cells for AAV production. The genetically engineered cells comprise molecular systems for temporal control of expression of genes required for AAV production. Also disclosed herein are methods ...
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WO/2024/081383A2 |
Provided herein are nucleic acids useful as guide nucleic acids (gNAs), e.g., guide ribonucleic acids (gRNAs), in a CRISPR system wherein the guide nucleic acids contain one or more modifications to one or more nucleotides, use of such g...
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WO/2024/078368A1 |
A method for preparing a halogenated indole glucoside and Tyrian purple. The method comprises: a halogenation step, involving halogenating tryptophan to obtain halogenated tryptophan; and a glucoside preparation step, involving mixing th...
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