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Displaying results 9091 - 9100 of 10000 in total
  1. Drug

    SPL23-2

    go.drugbank.com/drugs/DB17866
    Experimental

    SPL23-2 is an antisense oligonucleotide (ASO) targeting the W1282X nonsense mutation associated with a severe form of cystic fibrosis.[L46767]

  2. Drug

    GT-AS

    go.drugbank.com/drugs/DB17883
    Experimental

    GT-AS recombinant adeno-associated virus serotype 9 vector containing the transgene UBE3A encoding for ubiquitin protein ligase E3A/E6-AP.

    Synonyms: GT-AS, Recombinant adeno-associated virus vector containing the active biological substance AAV-GTX-hUBE3A
  3. Drug

    OTOF-GT

    go.drugbank.com/drugs/DB18129
    Investigational

    OTOF-GT is a dual vector AAV gene therapy being investigated for the restoration of hearing loss associated with otoferlin deficiency.

  4. Drug

    PBKR03

    go.drugbank.com/drugs/DB18138
    Investigational

    PBKR03 is a non-replicating recombinant adeno-associated virus serotype hu68 vector, containing a transgene encoding the human galactosylceramidase (GALC) enzyme.

  5. Drug

    TTX-MC138

    go.drugbank.com/drugs/DB18628
    Investigational

    TTX-MC138 is an antisense oligonucleotide targeting miR-10b.[L48335] It is under investigation for the treatment of various metastatic cancers.

  6. Drug

    U-0126

    go.drugbank.com/drugs/DB17060
    Experimental

    U-0126 is a direct inhibitor of the mitogen-activated protein-kinase kinase family members, MEK-1 and MEK-2.[A253162]

    Synonyms: 1,4-diamino-2,3-dicyano-1,4-bis(2-aminophenylthio)butadiene, Butanedinitrile, bis(amino((2-aminophenyl)thio)methylene)-, (2z,3z)-Salts: U-0126-EtOH
  7. 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.

  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

    Abagovomab

    go.drugbank.com/drugs/DB16077
    Investigational

    Abagovomab is under investigation in clinical trial NCT00418574 (Efficacy Multicentre Trial of Immunotherapy Vaccination With Abagovomab to Treat Ovarian Cancer Patients).

  10. Drug

    Fallypride

    go.drugbank.com/drugs/DB15167
    Investigational

    Fallypride is under investigation in clinical trial NCT02310360 (Striatal and Extra-striatal Dopamine Release in Response to Food in Healthy Humans).