Results for EB 101 (gene therapy) in treating recessive dystrophic epidermolysis bullosa published in JAMA- Abeona Therapeutics

Abeona Therapeutics has announced that positive clinical trial results from the EB 101 (gene therapy) outcomes following genetically corrected autologous epidermal grafts in patients with recessive dystrophic epidermolysis bullosa has been published in the Journal of the American Medical Association (JAMA). Abeona recently announced commencing enrollment in the Phase II portion of the clinical study (NCT01263379).

Results from the clinical study demonstrated that treatment with EB 101 restored Type VII collagen expression at the dermal-epidermal junction at the graft sites in 90% of the biopsy samples at 3 months' post-treatment, in 66% at 6 months' post-treatment, and in 42% samples at 12-months post-treatment. Importantly, correct type VII collagen localization was observed at anchoring fibrils. Wounds that demonstrated type VII collagen at graft sites displayed 87% healing at 3 months, 67% at 6 months, 50% at 12 months compared with baseline wound sites.

Comment: The Phase I clinical trial with gene-corrected skin grafts has shown promising wound healing and safety in patients with RDEB. Investigators at Stanford University are enrolling adolescent and adult patients for the Phase II EB-101 trial to determine the safety and efficacy of COL7A1 gene-corrected grafts on wound healing.

See: "Safety and wound outcomes following genetically corrected autologous epidermal grafts in patients with recessive dystrophic epidermolysis bullosa" Zurab Siprashvili, PhD; Ngon T. Nguyen, BS; Emily S. Gorell, MS; et al., Journal of the American Medical Association (JAMA) published 1 November 2016 doi:10.1001/jama.2016.15588