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Displaying results 4771 - 4780 of 7018 in total
  1. Drug

    Devafidugene civaparvovec

    go.drugbank.com/drugs/DB16790
    Investigational

    It uses proprietary zinc finger nucleases (ZFN) that insert a functional copy of the alpha-L-iduronidase (IDUA) enzyme. … Devafidugene civaparvovec is an investigational _in vivo_ genome editing product.

  2. Drug

    CY-1503

    go.drugbank.com/drugs/DB17605
    Investigational

    CY-1503 is a pentasaccharide ethyl glycoside consisting of one alpha-D-sialylosyl residue as a sodium salt, two beta-D-galactopyranosyl residues, one 2-acetamido-beta-Dglucopyranosyl unit, and one alpha-L-fucopyranosyl … It was investigated in cardiopulmonary bypass during surgical repair of congenital heart lesions.

  3. Drug

    NGN-401

    go.drugbank.com/drugs/DB18594
    Investigational

    NGN-401 is a recombinant serotype 9 adeno-associated virus (AAV) encoding an intron encoding a miRNA generating sequence, complementary miRNA binding sites and a full-length human methyl cytosine binding … Developed by Neurogene Inc., it is being investigated for the treatment of Rett syndrome.

    Synonyms: Recombinant serotype 9 adeno-associated virus (AAV) encoding an intron encoding a miRNA generating sequence, complementary miRNA binding sites and a full-length human methyl cytosine binding protein 2
  4. Drug

    AZD2389

    go.drugbank.com/drugs/DB22104
    Investigational

    AZD2389 is an investigational small molecule inhibitor of fibroblast activation protein (FAP).[L54978] It is currently undergoing safety, tolerability, and PK/PD profiling in clinical trials (e.g. … NCT06973005: A Study to Investigate How Multiple Oral Doses of AZD2389 Affect the Pharmacokinetics of Midazolam, Caffeine, and Bupropion in Healthy Participants).

  5. Drug

    WNY0824

    go.drugbank.com/drugs/DB23606
    Experimental

    [A274253] It is a dual inhibitor of PLK-1 and BRET-4, which have both been found to be involved in CRPC via suppression of AR/MYC-mediated transcription. [A274258] … WNY0824 is a preclinical small molecule drug being investigated for the treatment of Castration-Resistant Prostrate Cancer (CRPC).

  6. Drug

    AVR-RD-01

    go.drugbank.com/drugs/DB17651
    Investigational

    AVR-RD-01 is an investigational _ex vivo_ lentiviral gene therapy developed by AVROBIO Inc. … 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.

  7. Drug

    Efavaleukin alfa

    go.drugbank.com/drugs/DB16149
    Investigational

    Efavaleukin alfa is a fusion protein consisting of interleukin mutein fused to the C-terminus of an immunoglobulin G Fc domain by way of a G4S linker. … It is under investigation in clinical trial NCT03422627 (Safety and Efficacy of AMG 592 in Subjects With Steroid Refractory Chronic Graft Versus Host Disease).

  8. Drug

    MK-0354

    go.drugbank.com/drugs/DB05939
    Investigational

    MK-0354, is an orally administered drug candidate under development by Merck for the treatment of atherosclerosis and related disorders. … It targets G protein-coupled receptor, or GPCR, that have the potential to regulate plasma lipid profiles, including HDL, or the good cholesterol, similar to the therapeutic action of niacin.

  9. Drug

    4'-Methylene-5,8,10-trideazaaminopterin

    go.drugbank.com/drugs/DB05616
    Investigational

    CH-1504 is a an antirheumatic agent that has been shown in vitro to be a nonpolyglutamylatable and nonhydroxylatable antifolate that is more efficiently taken up into cells by the reduced folate carrier … It has been investigated for the treatment of Rheumatoid Arthritis (phase II).

  10. Drug

    Dengue virus serotype 1

    go.drugbank.com/drugs/DB22664
    Approved

    [A274008] It is one of four serotypes covered by Qdenga, a live, attenuated, tetravalent vaccine used for the prevention of dengue disease. … Dengue virus serotype 1 is a serotype of _Orthoflavivirus denguei_ known to cause dengue fever.