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Displaying results 6741 - 6750 of 10000 in total
  1. Drug

    CDX-1135

    go.drugbank.com/drugs/DB17607
    Investigational

    CDX-1135, formerly TP-10, is a soluble, recombinant human Complement Receptor Type 1 (sCR1) developed by Avant Immunotherapeutics (now Celldex).

  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 … It is being investigated in Wilson Disease.[L45924]

  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

    AVR-RD-02 is being investigated in Gaucher Disease.[L45958] … 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. … It was first developed by Aro Biotherapeutics and is being investigated for the treatment of Pompe disease.[L45978]

  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.