Search

Displaying results 8691 - 8700 of 10000 in total
  1. Drug

    RP-A501

    go.drugbank.com/drugs/DB17647
    Investigational

    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

  2. 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]

  3. Drug

    RP-L301

    go.drugbank.com/drugs/DB17661
    Investigational

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

  4. Drug

    rAAV9-rsATP7A

    go.drugbank.com/drugs/DB17664
    Experimental

    A recombinant adeno-associated viral vector gene therapy under investigation for the treatment of Menkes disease.

  5. 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.

  6. 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]

  7. 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]

  8. 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]

  9. Drug

    SIG-007

    go.drugbank.com/drugs/DB17710
    Experimental

    SIG-007 is a gene therapy that consists of cells that are genetically modified with a non-viral vector to express human alpha-galactosidase A.

  10. Drug

    TT 173

    go.drugbank.com/drugs/DB17717
    Investigational

    TT 173 is a Modified Human Tissue Factor currently being investigated to treat bleeding disorders such as hemophilia and von Willebrand disease, as well as bleeding episodes in cutaneous injuries or dental