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Displaying results 5741 - 5750 of 10000 in total
  1. 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.

  2. Drug

    SYNB1353

    go.drugbank.com/drugs/DB17666
    Investigational

    SYNB1353 is a strain of the probiotic bacteria _Escherichia coli_ (_E. coli_) type Nissle and an engineered probiotic drug.

  3. Drug

    SYNB1020

    go.drugbank.com/drugs/DB17667
    Investigational

    discontinued further development of SYNB1020 to treat hyperammonemia due to a lack of evidence of ammonia-lowering activities by SYNB1020 in a Phase 1b/2a study. … It was initially designed to convert ammonia to L-arginine [A258838] and granted orphan designation by the FDA for the treatment of urea cycle disorders in August 2016;[L45988] however, the manufacturer

  4. 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
  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

    DTX-101

    go.drugbank.com/drugs/DB17699
    Investigational

    It was previously investigated for the treatment of hemophilia B; however, the clinical development of DTX-101 was discontinued.[L46103]

  8. 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.

  9. 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]

  10. 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