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Displaying results 2591 - 2600 of 10000 in total
  1. Drug

    SAR-439459

    go.drugbank.com/drugs/DB17864
    Investigational

    SAR-439459 is a pan-transforming Growth Factor-beta (TGFβ) neutralizing antibody.[A259862] Developed by Sanofi, it is being investigated for the treatment of cancers and Osteogenesis Imperfecta.

  2. Drug

    QRX-411

    go.drugbank.com/drugs/DB17868
    Experimental

    It addresses the underlying cause of Usher syndrome due to the c.7595-2144A>G mutation in the _USH2A_ gene.[L46777] … QRX-411 is a first-in-class RNA-based oligonucleotide designed to restore wild-type USH2A mRNA, leading to the production of the functional USH2A protein.

  3. Drug

    MANF

    go.drugbank.com/drugs/DB17885
    Experimental

    MANF is a human recombinant mesencephalic, astrocyte-derived neurotrophic factor.

  4. Drug

    GIVI-MPC

    go.drugbank.com/drugs/DB17905
    Experimental

    GIVI-MPC is a therapy that uses human-induced pluripotent stem cell-derived myogenic progenitor cells.

  5. Drug

    DYN-101

    go.drugbank.com/drugs/DB17921
    Investigational

    DYN-101 is an antisense product candidate designed to reduce the expression of dynamin 2 protein.

    Synonyms: 5’-cEtG-sp-cEt5MeU-sp-cEt5MeU-sp-dT-sp-dA-sp-dT-sp-dT-spdA-sp-dT-sp-dA-sp-dG-sp-dG-sp-dG-sp-cEt5MeC-sp-cEt5MeU-sp-cEt5MeU-3’
  6. Drug

    AMT-162

    go.drugbank.com/drugs/DB17922
    Investigational

    AMT-162 is an adeno-associated virus serotype rh10 encoding artificial anti-SOD1 miRNA.

  7. Drug

    Debamestrocel

    go.drugbank.com/drugs/DB17934
    Investigational

    Debamestrocel is under clinical development by BrainStorm Cell Therapeutics and is being investigated for the treatment of Amyotrophic Lateral Sclerosis (ALS).[L47266]

  8. Drug

    ALMB-0166

    go.drugbank.com/drugs/DB17937
    Investigational

    ALMB-0166 is a umanized connexin 43 (Cx43) monoclonal antibody being investigated for the treatment of acute spinal cord injury. It was granted FDA orphan designation in November 2018.[L47067]

  9. Drug

    Suvodirsen

    go.drugbank.com/drugs/DB17940
    Investigational

    Suvodirsen is an antisense oligonucleotide that has been investigated for the treatment of Duchenne muscular dystrophy.

  10. Drug

    RGX-181

    go.drugbank.com/drugs/DB17942
    Experimental

    RGX-181 consists of an adeno-associated virus serotype 9 (AAV9) vector designed to deliver the soluble lysosomal enzyme tripeptidyl peptidase I (TPP1) gene. … Developed by REGENXBIO, it is being investigated for the treatment of late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease.[L47072]