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Displaying results 5541 - 5550 of 7018 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

    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.

  3. Drug

    Rilvegostomig

    go.drugbank.com/drugs/DB18991
    Investigational

    Rilvegostomig is under investigation in clinical trial NCT06109779 (Rilvegostomig + Chemotherapy as Adjuvant Therapy for Biliary Tract Cancer After Resection (Artemide-biliary01)).

  4. Drug

    Senicapoc

    go.drugbank.com/drugs/DB06280
    Investigational

    Senicapoc (ICA-17043) is a novel Gardos channel blocker.[A259068] It is being investigated for the treatment of sickle cell disease.

  5. Drug

    Rifalazil

    go.drugbank.com/drugs/DB04934
    Investigational

    Rifalazil is a derivative of the antibiotic rifamycin. It is being investigated by ActivBiotics for the treatment of various bacterial infections.

  6. Drug

    OSI-930

    go.drugbank.com/drugs/DB05913
    Investigational

    OSI-930 is an orally active inhibitor of two clinically validated targets: c-Kit and the vascular endothelial growth factor receptor-2 (VEGFR-2).

  7. Drug

    R-851

    go.drugbank.com/drugs/DB05747
    Experimental

    R-851 is part of the family of immune response modifier (IRM) and it acts in a novel way to stimulate the human body's immnune system to attack virus-infected cells and tumor cells. … It is expected to be topical treatment for cervical displasia and cervical high-risk human papillomavirus (HPV), the sexually transmitted virus that causes most cases of cervical cancer.

  8. Drug

    Velafermin

    go.drugbank.com/drugs/DB06493
    Investigational

    Velafermin was a novel investigational protein therapeutic, also known as fibroblast growth factor-20 (FGF-20), which promotes both epithelial and mesenchymal cell proliferation in vitro and has demonstrated … Velafermin was investigated as a potential therapy for the prevention and treatment of oral mucositis by fostering the regeneration and repair of the cells lining the gastrointestinal tract.

  9. Drug

    PR006

    go.drugbank.com/drugs/DB17002
    Experimental

    PR006 is a non-replicating recombinant adeno-associated virus serotype 9 containing the progranulin gene. It is being investigated for the treatment of dementia.

  10. Drug

    LY3884961

    go.drugbank.com/drugs/DB17070
    Investigational

    LY3884961 (previously PR001) is being developed by Prevail Therapeutics as a single-dose gene therapy for patients with Parkinson's disease and Gaucher disease.[L43468]