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    ASSAY FOR QUANTITATIVE ASSESSMENT OF MRNA CAPPING EFFICIENCY
    1.
    发明公开
    ASSAY FOR QUANTITATIVE ASSESSMENT OF MRNA CAPPING EFFICIENCY 审中-公开

    公开(公告)号：US20240263217A1

    公开(公告)日：2024-08-08

    申请号：US18565849

    申请日：2022-06-03

    申请人： TRANSLATE BIO, INC.

    发明人： Frank DEROSA ,  Anusha DIAS ,  Xiaobo GU

    IPC分类号： C12Q1/6825 ,  C12N15/10

    CPC分类号： C12Q1/6825 ,  C12N15/10

    摘要： The invention relates to a method of quantifying capping efficiency in a sample from an in vitro transcription reaction mixture comprising a plurality of mRNA transcript, characterized by a step of contacting the mRNA transcripts with an oligonucleotide complementary to a sequence of nucleotides in the 5′ untranslated region of the mRNA transcripts to form an mRNA:DNA hybrid between the oligonucleotide and the sequence of nucleotides of the mRNA transcripts in order to release the first five, six, or seven nucleotides of the mRNA transcripts using nuclease (e.g., RNAse H) digestion.

    METHODS AND COMPOSITIONS FOR DELIVERING MRNA CODED ANTIBODIES
    7.
    发明公开
    METHODS AND COMPOSITIONS FOR DELIVERING MRNA CODED ANTIBODIES 审中-公开

    公开(公告)号：US20230355524A1

    公开(公告)日：2023-11-09

    申请号：US18352615

    申请日：2023-07-14

    申请人： TRANSLATE BIO, INC.

    发明人： Lianne BOEGLIN ,  Christopher GUENARD ,  Khang Anh TRAN ,  Anusha DIAS ,  Frank DEROSA

    IPC分类号： A61K9/107 ,  C07K16/28 ,  A61K48/00 ,  A61K9/00

    CPC分类号： A61K9/1075 ,  C07K16/2866 ,  A61K48/0033 ,  A61K9/007 ,  A61K2039/505

    摘要： The present invention provides, among other things, method of treating an immune disease in a subject, the method comprising: administering to a subject in need thereof one or more mRNAs encoding a heavy chain and a light chain of an antibody that binds to a protein target selected from IL-4, IL-5, IL-6, IL-9, IL-13, IL-25, or IL-33, IL-4R, IL-5R, IL-6R, IL-9R, IL-13R, IL-25R or IL-33R, and wherein the one or more mRNAs are encapsulated in a lipid nanoparticle (LNP). The present invention also provides, among other things, a composition comprising one or more mRNAs encoding a heavy chain and a light chain of an antibody that binds a protein target selected from IL-4, IL-5, IL-6, IL-9, IL-13, IL-25, or IL-33, IL-4R, IL-5R, IL-6R, IL-9R, IL-13R, IL-25R or IL-33R, and wherein the one or more mRNAs are encapsulated in a lipid nanoparticle (LNP).

    GENERATION OF OPTIMIZED NUCLEOTIDE SEQUENCES
    8.
    发明公开
    GENERATION OF OPTIMIZED NUCLEOTIDE SEQUENCES 审中-公开

    公开(公告)号：US20230245721A1

    公开(公告)日：2023-08-03

    申请号：US17923844

    申请日：2021-05-07

    申请人： TRANSLATE BIO, INC.

    发明人： Khang Anh TRAN ,  Anusha DIAS ,  Frank DEROSA

    IPC分类号： G16B30/20 ,  G16B25/10 ,  G16B45/00

    CPC分类号： G16B30/20 ,  G16B25/10 ,  G16B45/00

    摘要： A method for generated an optimized nucleotide sequence is provided. The method comprises at least normalizing a codon usage table and selection of codons for a given amino acid sequence based on the usage frequency of the codons in the normalized codon usage table. The method may comprise generating a list of a plurality of optimized nucleotide sequences encoding the amino acid sequence, filtering the list of optimized nucleotide sequences, synthesizing one or more optimized nucleotide sequence, and/or administering one or more synthesized optimized nucleotide sequence.

    PEG LIPIDOID COMPOUNDS
    9.
    发明公开
    PEG LIPIDOID COMPOUNDS 审中-公开

    公开(公告)号：US20230226219A1

    公开(公告)日：2023-07-20

    申请号：US17998589

    申请日：2021-05-13

    申请人： TRANSLATE BIO, INC.

    发明人： Shrirang KARVE ,  Frank DEROSA ,  Ashish SARODE

    IPC分类号： A61K48/00 ,  A61K9/127 ,  A61K38/45 ,  A61K38/17 ,  C08G65/00

    CPC分类号： A61K48/0041 ,  A61K9/1273 ,  A61K38/45 ,  A61K38/177 ,  C08G65/00

    摘要： The compounds disclosed herein (e.g., compounds having a structure according to Formula (I), (II), (III), (IV), and (V)) are polymers wherein an organic polymeric segment (e.g., a polyethylene glycol (PEG) group) comprises covalent attachments to two or more lipid substructures, and each lipid substructure independently comprises a hydrophobic moiety and a hydrophilic moiety. The compounds provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.