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Displaying results 6651 - 6660 of 10000 in total
  1. Drug

    RGX-121

    go.drugbank.com/drugs/DB17681
    Investigational

    RGX-121 is designed to use the NAV AAV9 vector to deliver the human iduronate-2-sulfatase gene to the central nervous system.

    Synonyms: Non-replicating recombinant adeno-associated virus (AAV) vector of serotype 9 expressing human iduronate-2-sulfatase
  2. Drug

    LB-001

    go.drugbank.com/drugs/DB17685
    Experimental

    LB-001 is a gene editing therapy designed to incorporate a functioning version of the faulty methylmalonyl-COA mutase (MUT) gene into the genome of patients with methylmalonic acidemia.

  3. Drug

    Zinc mesoporphyrin

    go.drugbank.com/drugs/DB17694
    Experimental

    Synonyms: Zincate(2-), (7,12-diethyl-3,8,13,17-tetramethyl-21h,23h-porphine-2,18-dipropanoato(4-)-.kappa.n21,.kappa.n22,.kappa.n23,.kappa.n24)-, hydrogen (1:2), (sp-4-2)-Categories: Heterocyclic Compounds, 1-Ring, Heterocyclic Compounds with 4 or More Rings
  4. 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]

  5. Drug

    DTX-101

    go.drugbank.com/drugs/DB17699
    Investigational

    DTX-101 is an investigational gene therapy consisting of an adeno-associated virus serotype Rh10 vector encoding the human Factor Ix gene. … It was previously investigated for the treatment of hemophilia B; however, the clinical development of DTX-101 was discontinued.[L46103]

  6. Drug

    BBM-H803

    go.drugbank.com/drugs/DB17700
    Investigational

    BBM-H803 is a recombinant adeno-associated virus (AAV) based gene therapy drug for hemophilia A developed and manufactured by Belief BioMed.

  7. Drug

    WP-1301

    go.drugbank.com/drugs/DB17702
    Experimental

    WP1301 is a synthetic peptide derived from hFVIII. It binds to an MHC class II molecule and is recognized by a Factor VIII-specific T cell.[L46138]

  8. Drug

    Coagulin-B

    go.drugbank.com/drugs/DB17703
    Experimental

    Coagulin-B is an adeno-associated viral vector expressing human blood-coagulation Factor IX. … [A259038, A259043] It was previously investigated for the treatment of hemophilia; however, the manufacturing company discontinued the Phase I trial in 2004, halting its further development and investigation

    Synonyms: Coagulin B, Coagulin-B
  9. Drug

    BBM-H901

    go.drugbank.com/drugs/DB17704
    Investigational

    BBM-H901 is a liver-tropic adeno-associated viral (AAV) vector carrying cassette coding hyperactive Padua factor IX (FIX) protein. It is being investigated for the treatment of hemophilia B.[A259048]

  10. Drug

    Dalcinonacog alfa

    go.drugbank.com/drugs/DB17707
    Investigational

    Dalcinonacog alfa is a recombinant human factor IX (FIX) variant. It is being investigated for the treatment of hemophilia B.[A259058]