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Displaying results 5381 - 5390 of 10000 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

    protein 2 (LAMP-2). … 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

    Synonyms: Adeno-associated virus serotype 9 vector containing the human lysosome-associated membrane glycoprotein 2 isoform B transgene (AAV9.LAMP2B)
  5. Drug

    EGT-201

    go.drugbank.com/drugs/DB17648
    Experimental

    EGT-201 is an adeno-associated viral vector serotype 9 (AAV9) vector-based gene therapy consisting of the alpha-N-acetyglucosaminidase (NAGLU) transgene. … Developed by Esteve Pharmaceuticals, it is being investigated for Sanfilippo syndrome type B.[L45939]

  6. Drug

    EGT-301

    go.drugbank.com/drugs/DB17649
    Experimental

    EGT-301 is a gene therapy being investigated for Hunter Syndrome. … It consists of an adeno-associated viral vector serotype 9 (AAV9) containing the human Iduronate-2-sulfatase (I2S) transgene designed to restore I2S functional deficiency in patients with Hunter syndrome

  7. Drug

    AVR-RD-02

    go.drugbank.com/drugs/DB17650
    Investigational

    It consists of autologous CD34+ enriched hematopoietic stem cells (HSCs) that have been genetically modified _ex vivo_ with a lentiviral vector (LV) to contain a ribonucleic acid (RNA) transcript that,

  8. 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]

  9. Drug

    ABX1100

    go.drugbank.com/drugs/DB17655
    Investigational

    ABX1100 is an investigational Centyrin-siRNA conjugate that targets the Gys1 gene, which encodes glycogen synthase 1 (Gys1), an enzyme that is responsible for glycogen synthesis in the muscle.

  10. Drug

    RP-L301

    go.drugbank.com/drugs/DB17661
    Investigational

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