Search

Displaying results 7721 - 7730 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. … [A258833] Developed by Synlogic Operating Company, Inc., SYNB1353 is being investigated for the treatment of homocystinuria.

  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. … SYNB1020 is an engineered strain of _Escherichia coli_ Nissle 1917 developed by Synlogic Inc.

  4. Drug

    PRX-OTC

    go.drugbank.com/drugs/DB17680
    Experimental

    PRX-OTC is an intracellular enzyme replacement therapy designed to replace missing or defective enzyme in patients with ornithine transcarbamylase deficiency.

  5. 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
  6. 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.

  7. 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
  8. 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]

  9. 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]

  10. 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.