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Displaying results 141 - 150 of 10000 in total
  1. Drug

    AAV-AIPL

    go.drugbank.com/drugs/DB17857
    Experimental

    AAV-AIPL is an adenovirus-associated viral vector serotype 8 containing the human _AIPL1_ gene. … Developed by MeiraGTx, it is being investigated for the treatment of inherited retinal dystrophy due to defects in _AIPL1_ gene.

    Synonyms: adenovirus associated viral vector serotype 8 containing the human AIPL1 gene
  2. Drug

    Adenovirus/PNP

    go.drugbank.com/drugs/DB17175
    Investigational

    Synonyms: adenoviral vector expressing the E.coli purine nucleoside phosphorylase gene, Ad/PNP/F-araAMP
  3. Drug

    CypCaps

    go.drugbank.com/drugs/DB17435
    Experimental

    Synonyms: Cytochrome P450 isoform 2B1 gene transfected human embryonic kidney 293 cells encapsulated in polymeric cellulose sulphate
  4. Drug

    Tofersen

    go.drugbank.com/drugs/DB14782
    ApprovedInvestigational

    Tofersen is under an intrathecally administered antisense oligonucleotide targeting the mutated SOD1 gene that causes amyotrophic lateral sclerosis (ALS). … [L46108] Although there were various causes of ALS, 2% of ALS cases are due to SOD1 mutations, with more than 200 SOD1 mutations documented.

    Synonyms: Antisense Oligonucleotide Inhibitor Of The Expression Of Superoxide Dismutase 1 Gene, DNA, D((2'-O-(2-METHOXYETHYL))M5RC-SP-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RG-SP-(2'-O-(2-METHOXYETHYL))RG-(2'-O-(2-METHOXYETHYL))RA-SP-T-SP-A-SP-M5C-SP-A-SP-T-SP-T-SP-T-SP-M5C-SP-T-SP-A-SP
  5. Drug

    Volrubigene ralaparvovec

    go.drugbank.com/drugs/DB16785
    Investigational

    Volrubigene ralaparvovec is under investigation in clinical trial NCT03223194 (Gene Transfer Clinical Study in Crigler-najjar Syndrome).

    Synonyms: Non-replicating recombinant adeno-associated viral vector, serotype 8, expressing the 1A1 isoform of the bilirubin-uridine diphosphate glucuronosyltransferase gene
  6. Drug

    PMC-403

    go.drugbank.com/drugs/DB18132
    Investigational

    PMC-403 is a novel Tie2-activating antibody being investigated for the treatment of systemic capillary leak syndrome.

    Synonyms: a fully human monoclonal antibody that binds and activates the Tie2 receptor
  7. Drug

    OTL-203

    go.drugbank.com/drugs/DB18643
    Investigational

    OTL-203 is an autologous CD34+ enriched cell therapy that contains hematopoietic stem and progenitor cells transduced ex-vivo using a lentiviral vector encoding alpha-L-iduronidase gene.

    Synonyms: Autologous CD34+ enriched cell population that contains hematopoietic stem and progenitor cells transduced ex-vivo using a lentiviral vector encoding alpha-L-iduronidase gene
  8. Drug

    TN-401

    go.drugbank.com/drugs/DB18140
    Investigational

    TN-401 is a native AAV9 capsid with a genomic cassette containing a cardiomyocyte-specific promoter and the wild-type human PKP2 gene.

    Synonyms: A native AAV9 capsid with a genomic cassette containing a cardiomyocyte-specific promoter and the wild-type human PKP2 gene
  9. Drug

    Bromhexine

    go.drugbank.com/drugs/DB09019
    ApprovedInvestigational

    Bromhexine is mucolytic agent used for a variety of respiratory conditions associated with increased mucus secretion.

    Synonyms: N-cyclohexyl-N-methyl-(2-amino-3,5-dibrombenzyl)amine, 3,5-dibromo-N(alpha)-cyclohexyl-N(alpha)-methyltoluene-alpha-2-diamineMixtures: GRIPOFEN-T®, GLICERTOX N F
  10. Drug

    OTL-102

    go.drugbank.com/drugs/DB18642
    Experimental

    OTL-102 consists of autologous CD34+ enriched cells that contain hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human gp91phox gene. … It is being investigated for the treatment of X-linked chronic granulomatous disease.

    Synonyms: Autologous CD34+ enriched cell population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human gp91phox gene