Phase I/IIa Clinical Trial for Recessive Dystrophic Epidermolysis Bullosa Using Genetically Corrected Autologous Keratinocytes

    April 2017 in “ ˜The œjournal of investigative dermatology/Journal of investigative dermatology ”
    Zurab Siprashvili, Nguyễn Chí Ngôn, Emily S. Gorell, Kylie Loutit, Y. Dutt-Singkh, J. Nazaroff, Phuong Khuu, Louise K. Furukawa, H. Peter Lorenz, Tiffany I. Leung, Douglas R. Keene, Kerri E. Rieger, Paul A. Khavari, Alfred T. Lane, Jean Y. Tang, M. Peter Marinkovich
    TLDR The gene therapy showed significant wound healing and was safe for treating severe RDEB.
    The Phase I/IIa clinical trial investigated ex vivo gene therapy for treating severe Recessive Dystrophic Epidermolysis Bullosa (RDEB) in 6 adult subjects with COL7A1 mutations. Autologous keratinocytes were genetically corrected and grafted onto chronic wounds. The primary endpoint was wound healing, with secondary endpoints including C7 expression and anchoring fibril restoration. Significant wound healing (>75%) was observed in 94% of grafts at 3 months, 67% at 6 months, and 50% at 12 months. C7 expression and normal anchoring fibrils were present up to 2 years but diminished over time. No serious adverse events were reported, indicating a favorable safety profile and potential efficacy of this cell-based therapy for RDEB.
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