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WO/2024/077267A1 |
The present disclosure provides compositions and methods useful in the treatment of trinucleotide repeat disorders, including Huntington's disease and Friedreich's ataxia. The present disclosure also provides pegRNAs designed to target t...
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WO/2024/077184A2 |
Provided herein are TAGE agents and DNA targeting systems comprising an anti-CD11a antigen-binding polypeptide, a site-directed modifying polypeptide, and an ADAR-specific guide RNAs, and uses thereof.
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WO/2024/074709A1 |
The present invention relates to the field of synthetic evolution, and provides with a virus-assisted evolution platform for hyper-directed evolution of phenotypes in mammalian cells.
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WO/2024/074888A2 |
Synthetic apomixis can be achieved in an F1 hybrid of rice by inducing MiMe mutations and egg cell expression of BBM1 in a single step, while simultaneously providing an increased efficiency of clonal seed production. This can be used to...
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WO/2024/076967A1 |
The present invention provides compositions and methods for reducing the level and/or activity of a TET protein, for treating or preventing a disease and disorder associated with an increased level and/or activity of a TET protein, for r...
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WO/2023/205239A3 |
The present disclosure relates to systems for the expression of antibodies or antigen binding fragments in a subject, as well as methods of treatments of diseases therewith.
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WO/2024/077151A1 |
The present disclosure provides fusion proteins comprises fusion proteins comprising the signal sequence of PduB or PduM and a heterologous protein, as well as constructs for expressing the fusion proteins, and methods of their use. The ...
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WO/2024/007010A3 |
The disclosure provides improved Cas-CLOVER systems for gene editing. In embodiments, the disclosure provides recombinant Clo051 endonucleases, or nuclease domains thereof, comprising one or more amino acid mutations (e.g. one or more am...
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WO/2024/039776A3 |
The present invention provides compositions, systems, and methods for regulating protein expression using iRNA compositions which effect the RNA-induced silencing complex (RISC)- mediated cleavage of a universal RNAi target sequence and ...
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WO/2024/076121A1 |
The present invention relates to immune cells co-expressing IL-15 and a chimeric antigen receptor comprising an OX40 ligand as an intracellular signaling domain, and to a composition for preventing or treating cancer comprising same as a...
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WO/2024/075022A2 |
Disclosed herein are RNA polynucleotides comprising a 5' Cap, a 5' UTR comprising a cap proximal sequence disclosed herein, and a sequence encoding a payload. Also disclosed herein are compositions and medical preparations comprising the...
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WO/2024/050498A3 |
The present invention relates to a vesicular stomatitis virus vaccine vector encoding a MARV glycoprotein (rVSVΔG-MARV-GP). Vaccination with as little as 200 plaque-forming units was 100% efficacious against MARV lethality and prevented...
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WO/2024/075828A1 |
A data collection method for determining the likelihood of a subject developing Alzheimer's disease, the method comprising measuring, in a biological specimen of the subject, the proportion of methylated DNA in a CpG island including a c...
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WO/2023/215302A3 |
Methods of treating cancer in subjects in need thereof by administering a combination therapy including (a) a T cell expressing a T cell antigen coupler (TAC) polypeptide having (i) an antigen-binding domain that binds a target antigen, ...
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WO/2024/073848A1 |
The present disclosure relates to pharmaceutical compositions and methods for the preparation, manufacture and therapeutic use of nucleic acid vaccines comprising lipid nanoparticles (LNP) wherein the LNPs comprise cationic lipids, non-c...
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WO/2024/077287A2 |
Methods and compositions for actively loading of therapeutic compositions into yeast-generated extracellular vesicles (EVs) and their methods of use in treating one or more disease or conditions.
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WO/2022/187719A9 |
The present disclosure relates to molecular barcodes comprising a set of one or more identimers. Methods of using the molecular barcodes, such as to classify a material, are disclosed. Methods of making the molecular barcodes and systems...
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WO/2024/076710A1 |
The invention described herein provides an improved system and method for rAAV production. The system of the invention includes a recombinant DNA vector that comprises both the gene of interest (GOI) to be delivered to a target cell, as ...
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WO/2024/028775A3 |
The present disclosure relates to a double stranded short interfering nucleic acid (siNA) agent for use in medicine, in particular for use in the treatment of ophthalmic diseases comprising an antisense ribonucleotide sequence arranged t...
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WO/2024/076750A2 |
The present disclosure relates to pharmaceutical compositions comprising engineered macrophages and methods of use thereof. In some aspects, the present disclosure relates to engineered macrophages derived from pluripotent stem cells. In...
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WO/2024/075841A1 |
It is an object of the present invention to provide an RNA aptamer capable of binding to ASP7967 or an analogue thereof. The RNA aptamer of the present invention is an RNA aptamer that binds to ASP7967 or an analogue thereof, comprising ...
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WO/2023/153845A3 |
The present invention relates to a novel target nucleic acid editing system including a miniaturized nucleic acid editing protein and an engineered guide RNA, uses of homology-directed repair (HDR) in a target gene thereof, and the like....
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WO/2024/076934A2 |
In some aspects, provided herein are compositions, methods, and kits relating to an agent modulates expression of a CFTR protein. An agent provide herein can modify the CFTR gene or modulate process of the CFTR pre-mRNA. In some embodime...
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WO/2024/074274A1 |
The invention relates to a method for preparing an anthropogenic target substance, said method comprising: - producing an emulsion or suspension of the target substance, the emulsion or suspension containing particles and/or particulates...
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WO/2024/074713A1 |
The present invention relates to the adoptive therapy using notably CAR-T cells. Here the inventors used a lentiviral vector approach to silence RINF expression in a shRNA- dependent manner and evaluate the consequences of RINF silencing...
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WO/2024/076500A2 |
In some embodiments, a smart cell drug delivery (SmaCD) system is provided. The SmaCD system can comprise an engineered mammalian regulatory immune cell comprising: (a) a chimeric antigen receptor (CAR) comprising a first binding domain ...
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WO/2024/077263A2 |
Described in several exemplary embodiments herein are polyhedrin promoter sequences from a shrimp nudivirus, vectors containing the same, and uses thereof.
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WO/2024/030970A3 |
Several embodiments of the methods and compositions disclosed herein relate to immune cells that are genetically edited, for example, using Crispr/Cas, to modulate, reduce or otherwise eliminate expression of one or more endogenous genes...
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WO/2024/077140A1 |
In the various aspects and embodiments, the present disclosure provides cell populations or cell "banks" thereof (e.g., cell collections) to provide immune compatible, allogeneic cell therapies covering global, ethnic, and disease-specif...
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WO/2024/074540A2 |
The invention relates to a genetically engineered cell and compositions comprising said cell, wherein said cell is engineered to be especially sensitive towards two or more environmental factors, to enhance the biosafety profile of said ...
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WO/2024/074102A1 |
The present invention pertains to the field of biopharmaceuticals and provides a keratin YK93-6, a nucleic acid molecule encoding same, an expression vector comprising the nucleic acid molecule, a host cell that contains the expression v...
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WO/2024/075729A1 |
This protein manufacturing method comprises a manufacturing step for causing a plant, having introduced therein a nucleic acid fragment encoding a target protein, to produce the target protein. The plant has the autophagy system thereof ...
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WO/2024/077146A2 |
The present disclosure, in various aspects and embodiments, provides methods for generating hematopoietic lineages for cell therapy, including erythroid progenitor cells, progenitor erythroblasts, granulocyte-macrophage progenitor cells ...
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WO/2024/075764A1 |
The present invention addresses the problem of providing an enzyme that can efficiently conjugate a human sugar chain that has been modified with a payload to an antibody having a sugar chain that has been made uniform, and providing a n...
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WO/2024/076978A1 |
An engineered host cell comprising a genetic construct comprising a reporter gene under the control of a uric acid-responsive promoter, and a method for using such a cell to detect uric acid concentrations in a subject. An engineered hos...
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WO/2023/056350A9 |
Aspects of the disclosure relate to biosynthesis of cannabinoids and cannabinoid precursors in recombinant cells and in vitro.
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WO/2024/076109A1 |
A transformation cartridge, according to the present invention, comprises: a main body part in which a cell flow path, a material flow path, and a resultant flow path meeting each other are formed; and an electric field forming part whic...
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WO/2024/074107A1 |
The present invention belongs to the field of biopharmaceuticals. Provided are a keratin YK93-2, a nucleic acid molecule encoding the same, an expression vector comprising the nucleic acid molecule, a host cell comprising the expression ...
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WO/2023/034940A3 |
Provided are recombinant adeno-associated virus (r.AAV) vectors comprising a transgene to express galactose-1 -phosphate uridylyl transferase (GALT); virions comprising said vectors (r.AAV virions); methods of their production; methods o...
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WO/2024/074111A1 |
The present invention pertains to the field of biopharmaceuticals and provides a keratin YK93-9, a nucleic acid molecule encoding same, an expression vector comprising the nucleic acid molecule, a host cell that contains the expression v...
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WO/2024/075662A1 |
One purpose of the present disclosure is to provide a method for testing for the presence/absence of a stroke. The method for testing for the presence/absence of a stroke includes a step of measuring the presence/absence of at least on...
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WO/2024/010841A3 |
In the various aspects and embodiments, the present disclosure provides compositions and methods for treating pancreatic cancer (e.g., pancreatic ductal adenocarcinoma, or PDAC). In accordance with aspects of the disclosure, the composit...
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WO/2024/074668A1 |
The invention relates to the fields of medicine and immunology. In particular, it relates to novel antisense oligonucleotides that may be used in the treatment, prevention and/or delay of an USH2A related disease or condition.
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WO/2024/076990A1 |
Described herein are multi-specific antigen binding proteins that bind human fibrin or fibrinogen yC domain and vascular endothelial growth factor, and methods of use thereof. In certain aspects, described herein are pharmaceutical compo...
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WO/2024/077270A1 |
Described herein are immunoresponsive cells engineered to express cytokines and chimeric receptors. Also described herein are nucleic acids, cells, and methods directed to the same.
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WO/2024/076688A2 |
Certain embodiments of the invention provide a synthetic genomic safe harbor in the genome of a cell. Certain embodiments provide a method of creating a synthetic genomic safe harbor in a genome. Certain embodiments of the invention prov...
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WO/2024/074110A1 |
The present invention relates to the field of biological pharmacy, provides keratin YK93-5, a nucleic acid molecule encoding same, an expression vector containing the nucleic acid molecule, a host cell containing the expression vector or...
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WO/2024/077130A1 |
The present disclosure relates to compositions and methods for treating bladder cancer. In some embodiments the compositions and methods involve using antisense (AS) nucleic acids directed against Insulin-like Growth Factor 1 Receptor (I...
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WO/2024/077109A1 |
The present disclosure relates in some aspects to compositions of UNCI 3A antisense oligonucleotides (ASOs) and methods of using ASOs to alter UNC13A cryptic exon splicing and increase UNC13A protein expression. Such ASOs may be used to ...
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WO/2024/075368A1 |
The present invention includes: a generation step for generating ultrafine bubbles in a liquid so as to generate an ultrafine bubble-containing liquid; and an expression step for applying, to a plant, the ultrafine bubble-containing liqu...
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