Abeona therapeutics announces publication of positive long-term data from phase 1/2a clinical trial evaluating eb-101 gene therapy for recessive dystrophic epidermolysis bullosa

Abeona therapeutics inc. announced positive long term efficacy and safety results from its phase 1/2a clinical trial evaluating eb-101, a gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (rdeb). treatment with eb-101 resulted in sustained wound healing with a favorable safety profile at three years post-treatment. these long-term follow-up data in seven rdeb participants with 42 treated wounds were published in the peer-reviewed journal jci insight. key study findings include: wounds selected for treatment were present for a mean of 11.2 years (range 3-20 years). three years after treatment with eb-101, a majority of rdeb patients had sustained wound healing, with 80% (16/20) of wounds achieving ³50% healing, and 70% (14/20) achieving =75%. two years after treatment, only 1 of 6 untreated (17%), prospectively selected control wounds, had =50% healing. 50% or greater wound healing was associated with no pain (0/16) and no itch (0/16) at treated sites three years post-treatment, compared with presence of pain in 53% (20/38) and itch in 61% (23/38) of wound sites at baseline. eb-101 was associated with long-term molecular expression of type vii collagen protein, which plays an important role in anchoring the dermal and epidermal layers of the skin. no serious treatment-related adverse events were observed during the three-year observation period. no replication competent virus was present at any time point. researchers from stanford university school of medicine conducted the phase 1/2a single-center, open-label clinical trial to evaluate the long-term wound healing and safety of eb-101 in seven adult patients with severe generalized rdeb and to assess patient-reported outcomes following treatment. chronic open wounds, defined as wounds present and unhealed for at least 12 weeks, with a total area of at least 100 cm2, were required for enrollment. in the trial, gene-corrected eb-101 skin grafts (35 cm2 each) were transplanted onto six wound sites in each of the seven adult participants (n= 42 sites total) and wounds selected for treatment had been present for a mean of 11.2 years (range: 3-20 years). participants were followed for two to five years after transplantation of eb-101 and received standard of care therapies including iron supplementation and esophageal dilations during the study.
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