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Displaying results 8771 - 8780 of 9686 in total
  1. Drug

    Zodasiran

    go.drugbank.com/drugs/DB17626
    Investigational

    ARO-ANG3 is a subcutaneously administered RNA interference (RNAi)-based investigational medicine targeting angiopoietin-like protein 3 (ANGPTL3).

  2. Drug

    RGX-111

    go.drugbank.com/drugs/DB17632
    Investigational

    RGX-111 is an adeno-associated virus vector AAV9 designed to deliver the α-L-iduronidase (IDUA) gene.

    Synonyms: Adeno-associated virus vector serotype 9 expressing human a-L-iduronidase
  3. 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.

  4. Drug

    Phosphocysteamine

    go.drugbank.com/drugs/DB17640
    Experimental

    Categories: Compounds used in a research, industrial, or household setting
  5. 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.

  6. Drug

    EGT-101

    go.drugbank.com/drugs/DB17645
    Experimental

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

  7. 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]

  8. 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.

  9. Drug

    GTP-506

    go.drugbank.com/drugs/DB17682
    Experimental

    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

  10. 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]