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Displaying results 6121 - 6130 of 9716 in total
  1. Drug

    Tividenofusp alfa

    go.drugbank.com/drugs/DB17628
    ApprovedInvestigational

    Hunter syndrome is a rare, X-linked lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2-sulfatase (IDS), which leads to the toxic accumulation of glycosaminoglycans (GAGs) throughout … Tividenofusp alfa is a recombinant human IDS fused to a monoclonal antibody that targets the human insulin receptor (HIR), utilizing receptor-mediated transcytosis to gain access to the central nervous

    Synonyms: 676>S, L 678>A, Y, - L-iduronate sulfate sulfatase, EC:3.1.6.13) pro-protein (1-525), fused via the peptide linker 526 GGGGS 530 to a human immunoglobulin G1 C-terminal Fc fragment (531-757) variant (L 544>A, L 545>A, T
  2. Drug

    AEM-28

    go.drugbank.com/drugs/DB17642
    Investigational

    AEM-28 is a 28 amino acid mimetic of apolipoprotein E being investigated for lipid disorders, such as homozygous familial hypercholesterolemia. It possesses anti-inflammatory properties.[A258764]

  3. Drug

    VTX-801

    go.drugbank.com/drugs/DB17646
    Investigational

    VTX-801 is a replication-deficient recombinant adeno-associated viral vector (rAAV) consisting of an AAV liver tropic capsid containing a single-stranded DNA genome carrying a shortened version of the

  4. Drug

    RP-A501

    go.drugbank.com/drugs/DB17647
    Investigational

    RP-A501 is a gene therapy product consisting of a recombinant adeno-associated virus serotype 9 (rAAV9) capsid containing the human LAMP2B transgene (AAV9:LAMP2B), which encodes lysosome-associated membrane

  5. Drug

    EGT-301

    go.drugbank.com/drugs/DB17649
    Experimental

    EGT-301 is a gene therapy being investigated for Hunter Syndrome.

  6. Drug

    Reloxiliase

    go.drugbank.com/drugs/DB17653
    Experimental

    Reloxiliase is a first-in-class, oral crystalline formulation of the oxalate-specific, microbial enzyme oxalate decarboxylase. It is being investigated for the treatment of hyperoxaluria.[A258823]

  7. Drug

    RP-L301

    go.drugbank.com/drugs/DB17661
    Investigational

    RP-L301 is a lentiviral vector containing the human liver and erythroid pyruvate kinase gene.

  8. Drug

    rAAV9-rsATP7A

    go.drugbank.com/drugs/DB17664
    Experimental

    A recombinant adeno-associated viral vector gene therapy under investigation for the treatment of Menkes disease.

  9. Drug

    SYNB1353

    go.drugbank.com/drugs/DB17666
    Investigational

    SYNB1353 is a strain of the probiotic bacteria _Escherichia coli_ (_E. coli_) type Nissle and an engineered probiotic drug.

  10. Drug

    ASP-8731

    go.drugbank.com/drugs/DB17696
    Investigational

    ASP-8731 is a novel BACH1 inhibitor that potentially induces fetal hemoglobin (HbF). Developed by Mitobridge, it is being investigated for the treatment of sickle cell disease.[L46093]