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Displaying results 71 - 80 of 91 in total
  1. Drug

    AVR-RD-05

    go.drugbank.com/drugs/DB18631
    Experimental

    AVR-RD-05 is a gene therapy comprising genetically modified autologous stem cells transduced _ex vivo_ with a lentiviral vector encoding the human iduronate-2-sulfatase (IDS) enzyme.

    Synonyms: Ex vivo, lentiviral vector (LV)-mediated, genetically modified autologous cell therapy intended for the stable provision of functional human iduronate-2-sulfatase (IDS) enzyme to subjects with Hunter syndrome
  2. Drug

    EB003

    go.drugbank.com/drugs/DB18362
    Experimental

    EB003 is a CRISPR-based sequence-specific antimicrobial (SSAM) targeted against shiga toxin-producing E. coli.

    Synonyms: Phage-derived, non-replicative delivery vector carrying a DNA payload encoding an RNA-guided nuclease that targets stx genes of Shiga toxin-producing Escherichia coli (STEC)
  3. Drug

    Strimvelis

    go.drugbank.com/drugs/DB17594
    Investigational

    Synonyms: Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence
  4. Drug

    AAV9/MFSD8

    go.drugbank.com/drugs/DB18616
    Experimental

    It is an adeno-associated virus serotype 9 (AAV9) vector with an engineered transgene encoding the human CLN7/MFSD8 gene for expression of active human major facilitator superfamily domain containing 8

    Synonyms: An adeno-associated virus serotype 9 (AAV9) vector with engineered transgene encoding the human CLN7/MFSD8 gene for expression of active human major facilitator superfamily domain containing 8
  5. Drug

    BAX 335

    go.drugbank.com/drugs/DB17845
    Investigational

    BAX 335 is an adeno-associated virus vector 8 (AAV8)–based FIX gene therapy being investigated for the treatment of hemophilia B.[A259791]

    Synonyms: An Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing FIX Padua (AAV8.ss-3xCRM8-TTRFIX_R338Lopt)
  6. Drug

    Dabocemagene autoficel

    go.drugbank.com/drugs/DB17878
    Investigational

    Dabocemagene autoficel is an ex vivo, autologous cell-based gene therapy. It comprises autologous dermal fibroblasts genetically modified to express functional type VII collagen. Developed by Castle Creek Biosciences Inc., dabocemagene a...

    Synonyms: AUTOLOGOUS HUMAN DERMAL FIBROBLAST CELLS DERIVED FROM SKIN BIOPSIES, AND TRANSDUCED WITH A NON-REPLICATING, SELF-INACTIVATING LENTIVIRUS VECTOR THAT ENCODES HUMAN COLLAGEN TYPE VII ALPHA 1 (COL7A1) UNDER, DNA (RECOMBINANT LENTIVIRUS VECTOR INXN-2004-TRANSDUCED HUMAN AUTOLOGOUS DYSTROPHIC EPIDERMOLYSIS BULLOSA DERMAL FIBROBLAST CELL FCX-007 INTEGRATED PROVIRAL HUMAN COLLAGEN TYPE VII TRANSGENE COL7A1 PLUS
  7. Drug

    Giroctocogene fitelparvovec

    go.drugbank.com/drugs/DB16174
    Investigational

    Giroctocogene fitelparvovec is under investigation in clinical trial NCT04370054 (Study to Evaluate the Efficacy and Safety of PF-07055480 in Moderately Severe to Severe Hemophilia a Adults).

    Synonyms: Dna (recombinant adeno-associated virus 6 vector pf-07055480 human b-domain-deleted blood-coagulation factor viii-specifying)Categories: Adeno-associated Viral Vector Therapies
  8. Drug

    scAAV9.U7.ACCA

    go.drugbank.com/drugs/DB18582
    Investigational

    scAAV9.U7.ACCA is a non-replicating recombinant adeno-associated virus vector containing 4 antisense sequences designed to target exon 2 in the human DMD gene under the control of U7 small nuclear non-coding

    Synonyms: non-replicating recombinant adeno-associated virus vector containing 4 antisense sequences designed to target exon 2 in the human DMD gene under the control of U7 small nuclear non-coding RNA
  9. Drug

    Botaretigene sparoparvovec

    go.drugbank.com/drugs/DB16814
    Investigational

    Synonyms: DNA (RECOMBINANT PSEUDOTYPED ADENO-ASSOCIATED VIRUS 5 VECTOR AAV-RPGR HUMAN RHODOPSIN KINASE PROMOTER FRAGMENT-REGULATED HUMAN RETINITIS PIGMENTOSA GTPASE REGULATOR RPGR-ORF15 ISOFORM GENE VARIANT PLUSCategories: Adeno-associated Viral Vector Therapies
  10. Drug

    Fordadistrogene movaparvovec

    go.drugbank.com/drugs/DB16800
    Investigational

    Synonyms: DNA (RECOMBINANT ADENO-ASSOCIATED VIRUS 9 VECTOR PF-06939926 HUMAN MUSCLE-SPECIFIC ENHANCER/PROMOTER PLUS HUMAN MINI-DYSTROPHIN GENE PLUS ADENO-ASSOCIATED VIRUS 9 INVERTED TERMINAL REPEAT FLANKS)Categories: Adeno-associated Viral Vector Therapies