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Displaying results 8831 - 8840 of 9698 in total
  1. Drug

    RGX-501

    go.drugbank.com/drugs/DB17639
    Investigational

    RGX-501 is an adeno-associated virus vector 8 (AAV8)-mediated gene therapy that delivers a functional copy of the human low-density lipoprotein receptor (LDLR) gene to liver cells.

  2. Drug

    Phosphocysteamine

    go.drugbank.com/drugs/DB17640
    Experimental

    Categories: Compounds used in a research, industrial, or household setting
  3. Drug

    AEM-28-14

    go.drugbank.com/drugs/DB17643
    Experimental

    AEM-28-14 is an analog of [AEM-28], an apolipoprotein E mimetic peptide, with a 400 percent greater cholesterol-lowering efficacy and a several-fold increase in drug tolerability.

  4. Drug

    EGT-101

    go.drugbank.com/drugs/DB17645
    Experimental

    Developed by Esteve Pharmaceuticals, it is being investigated for Sanfilippo syndrome type A.[L45919]

  5. Drug

    OTL-201

    go.drugbank.com/drugs/DB17652
    Experimental

    It consists of a lentiviral vector to insert a functional copy of the human N-Sulfoglucosamine Sulfohydrolase (SGSH) gene into autologous CD34+ hematopoietic stem cells.[L45973]

  6. Drug

    Tralesinidase alfa

    go.drugbank.com/drugs/DB17657
    Investigational

    Tralesinidase alfa is a chimeric fusion protein comprised of recombinant human NAGLU and a modified human insulin-like growth factor 2.

  7. Drug

    GTP-506

    go.drugbank.com/drugs/DB17682
    Experimental

    the desired genetic correction. … GTP-506 is a gene therapy formed by two vectors, a nuclease vector targeting gene editing in the well-characterized PCSK9 gene locus and a therapeutic donor vector that inserts the OTC gene to provide

  8. Drug

    CB-010

    go.drugbank.com/drugs/DB17711
    Investigational

    CB-010 is a CRISPR-edited allogeneic CAR-T cell therapy targeting CD19. It is being investigated for the treatment of follicular lymphoma.[L46158]

  9. Drug

    Mozafancogene autotemcel

    go.drugbank.com/drugs/DB17723
    Investigational

    RP-L102 is a lentiviral vector carrying the Fanconi Anemia-A (FANCA) gene currently being developed by Rocket Pharmaceuticals for the treatment of Fanconi anemia type A

    Synonyms: Autologous CD34+ HSPCs Transduced with LV Carrying FANCA Gene RP
  10. Drug

    NKTT-120

    go.drugbank.com/drugs/DB17730
    Investigational

    NKTT-120 is a recombinant humanized IgG1k monoclonal antibody to human invariant T cell receptor (iTCR).