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Displaying results 51 - 60 of 107 in total
  1. Drug

    Adenovirus/PNP

    go.drugbank.com/drugs/DB17175
    Investigational

    Synonyms: adenoviral vector expressing the E.coli purine nucleoside phosphorylase gene
  2. Drug

    P-BCMA-101

    go.drugbank.com/drugs/DB17371
    Investigational

    P-BCMA-101 is an autologous CAR-T therapy developed using Poseida’s piggyBac platform technology.

    Synonyms: Autologous T-cells transfected with PiggyBac transposon plasmid vector expressing B-cell maturation antigen (CAR-T cells)
  3. 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
  4. 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
  5. Drug

    Mozafancogene autotemcel

    go.drugbank.com/drugs/DB17723
    Investigational

    RP-L102 is a lentiviral vector carrying the Fanconi Anemia-A (FANCA) gene currently being developed by Rocket Pharmaceuticals for the treatment of Fanconi anemia type A

    Synonyms: Autologous CD34+-enriched HSPCs Transduced with Lentiviral Vector Carrying FANCA Gene RP-L102, Autologous CD34+-enriched Hematopoietic Stem and Progenitor Cells Transduced with Lentiviral Vector Carrying FANCA Gene RP-L102
  6. Drug

    AVR-RD-04

    go.drugbank.com/drugs/DB18644
    Experimental

    It consists of autologous CD34+ enriched cells transduced with a lentiviral vector containing RNA resulting in codon-optimized cDNA encoding for functional human cystinosin.

    Synonyms: autologous CD34+ enriched cells transduced with a lentiviral vector containing RNA resulting in codon-optimized cDNA encoding for functional human cystinosin
  7. Drug

    GT-AS

    go.drugbank.com/drugs/DB17883
    Experimental

    GT-AS recombinant adeno-associated virus serotype 9 vector containing the transgene UBE3A encoding for ubiquitin protein ligase E3A/E6-AP.

    Synonyms: Recombinant adeno-associated virus vector containing the active biological substance AAV-GTX-hUBE3A
  8. Drug

    Volrubigene ralaparvovec

    go.drugbank.com/drugs/DB16785
    Investigational

    Volrubigene ralaparvovec is under investigation in clinical trial NCT03223194 (Gene Transfer Clinical Study in Crigler-najjar Syndrome).

    Synonyms: Non-replicating recombinant adeno-associated viral vector, serotype 8, expressing the 1A1 isoform of the bilirubin-uridine diphosphate glucuronosyltransferase geneCategories: Adeno-associated Viral Vector Therapies
  9. Drug

    AGIL-FA

    go.drugbank.com/drugs/DB16973
    Experimental

    Synonyms: Recombinant adeno-associated viral vector serotype 5 carrying the gene for the human frataxin protein
  10. Drug

    AAV-AIPL

    go.drugbank.com/drugs/DB17857
    Experimental

    AAV-AIPL is an adenovirus-associated viral vector serotype 8 containing the human _AIPL1_ gene.

    Synonyms: adenovirus associated viral vector serotype 8 containing the human AIPL1 gene