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    PRODRUGS FOR USE IN THE TREATMENT OF TISSUE DAMAGE
    2.
    发明申请
    PRODRUGS FOR USE IN THE TREATMENT OF TISSUE DAMAGE 有权

    公开(公告)号：US20250154143A1

    公开(公告)日：2025-05-15

    申请号：US18683412

    申请日：2022-08-18

    Applicant: UCL BUSINESS LTD

    Inventor： Mark Brian PEPYS ,  Melanie Susanne GLOSSOP ,  Christopher SWAIN ,  Graham Walter TAYLOR ,  Stephen Paul WOOD

    IPC: C07D453/02

    Abstract: An agent for use in medicine, wherein the agent comprises a compound of Formula (I): wherein: Ar is an aryl linker group, and R1 and R2 are independently selected from groups of formula —OH; —OR3; —O(CO)R3; —NR3R3′; wherein: R3 and R3′ are independently selected from H, C1-C12 alkyl, C1-C12 alkylene, C3-C8 (hetero)cycloalkyl, or C5-C12 (hetero)aryl, or R3 and R3′ together form a C3-C8 heterocyclic ring with the nitrogen, each optionally substituted with one, two or three groups selected from halogen, —CF3, —OR6, —NR6R6, —COR6, and —CO2R6, C3-C8 (hetero)cycloalkyl, or C5-C12 (hetero)aryl; R4 and R5 are independently selected from H or C1-C6 alkyl; and R6 and R6 are independently C1-C6 alkyl, or together form a C3-C8 heterocyclic ring with the nitrogen, provided that R1 and R2 are not both —OH, including individual pharmaceutically acceptable salts, solvates, or derivatives thereof.

    METHOD
    4.
    发明申请
    METHOD 有权

    公开(公告)号：US20240424023A1

    公开(公告)日：2024-12-26

    申请号：US18690333

    申请日：2022-09-09

    Applicant: Pietro Genovese ,  UCL BUSINESS LTD

    Inventor： Pietro Genovese ,  Thomas Fox ,  Emma Morris ,  Claire Booth ,  Ben Houghton

    IPC: A61K35/17 ,  A61K39/00 ,  A61K48/00 ,  A61P35/00 ,  C12N5/0783 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

    Abstract: The present invention provides a method of engineering a cell, comprising the steps of introducing into the cell: i) a site-directed nuclease which is capable of cleaving a target nucleotide sequence at the 3′-end of Intron 1 of a Cytotoxic T-Lymphocyte Associated Protein 4 gene (CTLA4); and ii) a nucleic acid construct which comprises a nucleic acid sequence comprising one or more of exon 2, exon 3 and exon 4 of CTLA4, or a sequence with at least 70% identity to exon 2, exon 3 and/or exon 4 of the CTLA4 gene, and 5′- and 3′-homology arms, wherein each of the 5′ and '3 homology arms is essentially complementary to a sequence flanking the target nucleotide sequence at the 3′-end of Intron 1 of CTLA4.

    CASRX/CAS13D SYSTEMS TARGETING C9ORF72
    7.
    发明申请
    CASRX/CAS13D SYSTEMS TARGETING C9ORF72 有权

    公开(公告)号：US20240374757A1

    公开(公告)日：2024-11-14

    申请号：US18554454

    申请日：2022-04-19

    Applicant: UCL Business Ltd

    Inventor： Liam KEMPTHORNE ,  Adrian ISAACS

    IPC: A61K48/00 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

    Abstract: Provided herein is a composition comprising (i) a nucleic acid sequence encoding a CasRx/Cas13d polypeptide; and (ii) a guide RNA that binds specifically to a target sequence in C9orf72 RNA. Also provided are associated pharmaceutical compositions, guide RNAs, complexes, vectors and cells, and uses of the compositions to neurodegenerative disorders.

    Capsid
    8.
    发明授权
    Capsid 有权

    公开(公告)号：US12138318B2

    公开(公告)日：2024-11-12

    申请号：US16541516

    申请日：2019-08-15

    Applicant: UCL Business LTD

    Inventor： Amit Nathwani ,  Allison Dane

    IPC: A61K48/00 ,  C07K14/005 ,  C12N15/86

    Abstract: There is described an AAV capsid protein having an amino acid sequence which has at least 98% identity to the sequence of SEQ ID NO: 3 or at least 94% identity to the sequence of SEQ ID NO: 4. Also described is a pharmaceutical composition, an AAV capsid and a viral particle comprising the capsid protein, a recombinant AAV vector comprising a nucleotide sequence which encodes for the capsid protein, and a host cell and a transgenic animal comprising the capsid protein or the vector. In addition, there is described a method of transferring a nucleic acid of interest into a mammal comprising introducing a recombinant AAV vector into the mammal, wherein the recombinant AAV vector comprises a gene of interest which is encapsidated into a capsid comprising the capsid protein.

    METHODS AND REAGENTS FOR THE DETECTION OF ANALYTES
    9.
    发明公开
    METHODS AND REAGENTS FOR THE DETECTION OF ANALYTES 审中-公开

    公开(公告)号：US20240345086A1

    公开(公告)日：2024-10-17

    申请号：US18579635

    申请日：2022-07-20

    Applicant: UCL Business LTD

    Inventor： Kevin Mills ,  Wendy Heywood ,  Ivan Doykov ,  James Moon

    IPC: G01N33/569 ,  G01N33/68

    CPC classification number: G01N33/56983 ,  G01N33/6848 ,  G01N2333/165 ,  G01N2469/20

    Abstract: The present invention relates to a high throughput targeted proteomics-based method for identifying and quantifying one or more binding molecules in a biological fluid of an individual. The present invention involves the use of a plurality of bait molecules to capture the binding molecules. The invention uses proteomics techniques, such as mass spectrometry, for the identification and quantification of the binding molecules. The methods of the invention can be used to identify and quantify antibodies produced by an individual, and proteins expressed by an individual, following exposure to antigens, such as viral antigens, particularly coronavirus antigens and more particularly SARS-CoV-2. Exposure may be via vaccination or following natural infection. The methods of the invention can be used to identify and quantify molecules which bind to a range of proteins of interest, such as autoantigens and neoantigens, as well as to viral vectors.

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