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Displaying results 7071 - 7080 of 10000 in total
  1. Drug

    TAK-611

    go.drugbank.com/drugs/DB17644
    Investigational

    TAK-611, formerly known as SHP611, is a recombinant human arylsulfatase A. First developed by Takeda, it is being investigated for the treatment of metachromatic leukodystrophy.

  2. Drug

    AVR-RD-01

    go.drugbank.com/drugs/DB17651
    Investigational

    It consists of autologous CD34+ cells that transduced with Lentiviral Vector/alpha-galactosidase A (AGA) encoding for the human AGA complementary deoxyribonucleic acid (cDNA) sequence.

  3. Drug

    mRNA-3927

    go.drugbank.com/drugs/DB17669
    Investigational

    mRNA-3927 is an investigational mRNA therapy that consists of two mRNAs that encode for the alpha and beta subunits of the mitochondrial enzyme propionyl-CoA carboxylase (PCC) encapsulated within a lipid

  4. Drug

    HMI-103

    go.drugbank.com/drugs/DB17684
    Investigational

    HMI-102 is an investigational gene therapy designed to encode the phenylalanine-4-hydroxylase (PAH) gene, mutated in people with phenylketonuria.

  5. Drug

    SerpinPC

    go.drugbank.com/drugs/DB17698
    Investigational

    SerpinPC is a mutated alpha-1-antitrypsin (α1AT) and serine protease inhibitor being investigated for the treatment of hemophilia.[A258993]

  6. Drug

    ATX-F8-117

    go.drugbank.com/drugs/DB17712
    Experimental

    Developed by Apitope, it is being investigated for the treatment of hemophilia A.[L46163]

  7. Drug

    Thalagen

    go.drugbank.com/drugs/DB17714
    Experimental

    Thalagen is a branded product developed by San Rocco Therapeutics. … It consists of autologous CD34-positive hematopoietic progenitor cells transduced with TNS9.3.55, a lentiviral vector encoding the normal human beta-globin gene.

  8. Drug

    MOD-5014

    go.drugbank.com/drugs/DB17715
    Investigational

    MOD-5014 is a long-acting carboxy-terminal peptide (CTP)-modified Factor IIa.[A259088] It is being investigated for the treatment of hemophilia.[L46173]

  9. Drug

    mRNA-3351

    go.drugbank.com/drugs/DB17718
    Experimental

    mRNA-3351 is a modified mRNA encoding the UGT1A1 gene. Developed by Moderna, it is currently being investigated as the treatment for Crigler-Najjar syndrome

  10. Drug

    NM3086

    go.drugbank.com/drugs/DB17719
    Experimental

    NM3086 is a recombinant, humanized, aglycosylated, IgG1-kappa anti-properdin monoclonal antibody being developed by Novelmed Therapeutics Inc.[L46193]