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Displaying results 6571 - 6580 of 10000 in total
  1. Drug

    Tefidsogene civaparvovec

    go.drugbank.com/drugs/DB16789
    Investigational

    Tefidsogene civaparvovec, formerly known as SB-913, is an investigational therapy for genome editing. … It consists of an adeno-associated virus (AAV) type 2/6 vector and a zinc finger nuclease (ZFN) system that inserts a correct copy of the Iduronate 2-Sulfatase (IDS) gene into the albumin locus in hepatocytes

    Synonyms: EXONS 2-9 FROM HIDS AND A SPLICE ACCEPTOR SITE (SA), A RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS TYPE 2/6 (RAAV REP2-CAP6) VECTOR, WHICH CONTAINS A PROMOTER-LESS HUMAN IDURONATE 2-SULFATASE (HIDS) TRANSGENE CASSETTE, ENCODING PARTS OF EXON 1 PLUS
  2. Drug

    Devafidugene civaparvovec

    go.drugbank.com/drugs/DB16790
    Investigational

    Developed by Sangamo Therapeutics, Inc., devafidugene civaparvovec is being investigated for the treatment of mucopolysaccharidosis Type I (MPS I).[L46716] … It uses proprietary zinc finger nucleases (ZFN) that insert a functional copy of the alpha-L-iduronidase (IDUA) enzyme.

  3. Drug

    Delandistrogene moxeparvovec

    go.drugbank.com/drugs/DB16802
    ApprovedInvestigational

    Microdystrophin is a truncated, functional form of dystrophin. … [L47026] DMD is an X-linked genetic disorder characterized by mutations in the dystrophin gene, leading to a deficiency in functional dystrophin protein.

  4. Drug

    Isaralgagene civaparvovec

    go.drugbank.com/drugs/DB16808
    Investigational

    Isaralgagene civaparvovec, formerly known as ST-920, is liver-tropic rAAV 2/6 vector carrying the cDNA for human alpha-galactosidase A (α-Gal A) enzyme. … It is developed by Sangamo Therapeutics and investigated in Fabry Disease.

  5. Drug

    Cinchonidine

    go.drugbank.com/drugs/DB16831
    Experimental

    Synonyms: Quinine bisulfate impurity B, Quinine sulfate impurity BCategories: Cytochrome P-450 CYP2D6 Inhibitors, Cytochrome P-450 Enzyme Inhibitors
  6. Drug

    Fadrozole

    go.drugbank.com/drugs/DB16832
    Experimental

    Categories: Heterocyclic Compounds, 1-Ring
  7. Drug

    Bentamapimod

    go.drugbank.com/drugs/DB16851
    Investigational

    Categories: Heterocyclic Compounds, 1-Ring, Heterocyclic Compounds, 2-Ring
  8. Drug

    Tyrosol

    go.drugbank.com/drugs/DB16855
    Experimental

    Synonyms: p-Tyrosol, 4-HydroxyphenylethanolCategories: Cytochrome P-450 Substrates, Cytochrome P-450 CYP2A6 Substrates
  9. Drug

    Indigo

    go.drugbank.com/drugs/DB16862
    Investigational

    Synonyms: D&C Blue No. 6Categories: Heterocyclic Compounds, 2-Ring, Compounds used in a research, industrial, or household setting
  10. Drug

    CB-5083

    go.drugbank.com/drugs/DB16874
    Investigational

    Synonyms: 1-(4-(benzylamino)-7,8-dihydro-5H-pyrano[4,3-d]pyrimidin-2-yl)-2-methyl-1H-indole-4-carboxamide, 1-(7,8-Dihydro-4-((phenylmethyl)amino)-5H-pyrano(4,3-d)pyrimidin-2-yl)-2-methyl-1H-indole-4-carboxamideCategories: Heterocyclic Compounds, 1-Ring, Heterocyclic Compounds, 2-Ring