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Displaying results 4751 - 4760 of 8196 in total
  1. Drug

    Domofenogene zalfaparvovec

    go.drugbank.com/drugs/DB16810
    Investigational

    Domofenogene zalfaparvovec, formerly known as BMN-307, is an investigational AAV5-phenylalanine hydroxylase (PAH) gene therapy developed by BioMarin Pharmaceutical.

  2. Drug

    AT-GTX-502

    go.drugbank.com/drugs/DB17001
    Experimental

    AT-GTX-502 is an experimental, one-time gene therapy that uses AAV9 to deliver a functional copy of CLN3.

  3. Drug

    ENTR-601-44

    go.drugbank.com/drugs/DB17836
    Investigational

    ENTR-601-44 is an exon 44 skipping phosphorodiamidate morpholino oligonucleotide (PMO). Developed by Entrada Therapeutics, it was investigated for the treatment of Duchenne muscular dystrophy.[L46641]

  4. Drug

    GB-13

    go.drugbank.com/drugs/DB18387
    Experimental

    GB-13 is a genetically engineered recombinant protein consisting of an IL13Ra2-targeted genetically engineered mutant of IL-13 and PE4E, a derivative of Pseudomonas exotoxin A.

  5. Drug

    PAS-004

    go.drugbank.com/drugs/DB18611
    Investigational

    PAS-004 is an allosteric MEK 1/2 inhibitor being developed by Pasithea Therapeutics. It is being investigated for the treatment of neurofibromatosis 1 and Noonan syndrome.

  6. Drug

    Rabeximod

    go.drugbank.com/drugs/DB05772
    Investigational

    Rabeximod is an orally administered compound for treatment of moderate or severe active rheumatoid arthritis that is currently undergoing phase II clinical testing in eight European countries.

  7. 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

  8. Drug

    ECT-001 expanded cord blood

    go.drugbank.com/drugs/DB21745
    Investigational

    [L52013] It is being investigated in hematopoietic cell transplantation as ECT-001 expanded cord blood can demonstrate enhanced stem cell proliferation and engraftment compared to untreated UBCs.

  9. Drug

    Disitamab vedotin

    go.drugbank.com/drugs/DB17208
    Investigational

    Disitamab vedotin is an antibody-drug conjugate drug targeting human epidermal growth factor receptor 2 (HER2). It is currently being investigated for cancers.[A254721]

  10. Drug

    MGN-4893

    go.drugbank.com/drugs/DB17338
    Experimental

    MGN-4893 is an inhibitor of microRNA-451. In February 2011, it was granted FDA orphan designation for the treatment of polycythemia vera.[L47356]