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Displaying results 101 - 110 of 4188 in total
  1. Drug

    AMT-130

    go.drugbank.com/drugs/DB16975
    Investigational

    Synonyms: Adeno-associated viral vector serotype 5 encoding a microRNA targeted to human Huntingtin gene
  2. Drug

    NTLA-2002

    go.drugbank.com/drugs/DB17597
    Investigational

    NTLA-2002 is a lipid nanoparticle encapsulating single guide RNA (G012267) targeting the human KLKB1 gene and messenger RNA (mRNA000042) encoding Cas9. … It is an investigational therapy designed to knock out the target gene kallikrein B1 (KLKB1) to reduce plasma kallikrein activity, thereby preventing hereditary angioedema attacks.[L45738]

    Synonyms: lipid nanoparticle encapsulating single guide RNA (G012267) targeting the human KLKB1 gene and messenger RNA (mRNA000042) encoding Cas9
  3. Drug

    Glycerin

    go.drugbank.com/drugs/DB09462
    ApprovedInvestigational

    A trihydroxy sugar alcohol that is an intermediate in carbohydrate and lipid metabolism.

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  4. Drug

    TN-201

    go.drugbank.com/drugs/DB18700
    Investigational

    TN-201 is a gene therapy comprising a native AAV9 capsid with a genomic cassette containing a cardiomyocyte-specific promoter and the wild-type human MYBPC3 gene.

    Synonyms: Native AAV9 capsid with a genomic cassette containing a cardiomyocyte-specific promoter and the wild-type human MYBPC3 gene
  5. Drug

    Allantoin

    go.drugbank.com/drugs/DB11100
    ApprovedInvestigational

    Allantoin is a substance that is endogenous to the human body and also found as a normal component of human diets [FDA Label]. In healthy human volunteers, the mean plasma concentration of allantoin is about 2-3 mg/l. During exercise, th...

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  6. Drug

    ETX101

    go.drugbank.com/drugs/DB18579
    Investigational

    ETX101 is a non-replicating recombinant adeno associated viral vector, serotype 9, designed to promote increased transcription of SCN1A gene.

    Synonyms: non-replicating recombinant adeno associated viral vector, serotype 9, designed to promote increased transcription of SCN1A gene
  7. Drug

    PBFT02

    go.drugbank.com/drugs/DB18580
    Investigational

    PBFT02 is a non-replicating recombinant adeno-associated virus serotype 1 vector delivering human GRN gene encoding the protein progranulin.

    Synonyms: Non-replicating recombinant adeno-associated virus serotype 1 vector delivering human GRN gene encoding the protein progranulin
  8. Drug

    PBCAR20A

    go.drugbank.com/drugs/DB17189
    Investigational

    Synonyms: Allogeneic gene edited CD20-directed CAR (Chimeric Antigen Receptor) T cells
  9. Drug

    Myb34.5

    go.drugbank.com/drugs/DB17437
    Experimental

    Synonyms: Herpes simplex type 1 virus containing cellular B-myb gene as tumor-specific promoter
  10. Drug

    Benzalkonium

    go.drugbank.com/drugs/DB11105
    ApprovedInvestigational

    Benzalkonium is a quaternary ammonium compound used as a biocide, a cationic surfactant, and as a phase transfer agent [FDA Label]. Benzalkonium is more commonly contained in consumer products in its salt form, benzalkonium chloride [FDA...

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