On March 8th, Abeona Therapeutics, a biotechnology company that specializes in developing gene therapy for severe and rare diseases, said that the European Medicines Agency (EMA) Orphan Drugs Committee gave the company gene therapy EB-101 for the treatment of recessive dystrophic bullae. Oral epidermal release (RDEB) orphan drug status, RDEB is a serious life-threatening hereditary skin disease characterized by blisters in the skin and involving the body.
EB-101 is the fourth orphan drug designated by EMA to Abeona, which has shown promising safety and efficacy data in ongoing clinical phase 1 / 2 trials.
Abeona Therapeutics CEO and President Timothy J. Dr. Miller said: "EB-101 is another gene therapy for orphan drug status, further strengthening the company's orphan drug portfolio, and the grant of orphan drug status proves that the therapy is used in rare diseases that are drug-free. The effectiveness of treatment. The grant of orphan drug status means that EB-101 has gained market exclusive rights in Europe for 10 years, and drugs that have similar mechanisms of action for the same disease will not be available for sale during this period."
The ongoing clinical phase 1 / 2 trial is to re-implant the patient's genetically engineered skin cells (which can express v type II collagen) into the patient for disease treatment. This clinical trial has shown tissue repair and good safety in patients with recessive dystrophic bullous epidermis. Researchers at Stanford University are recruiting adolescents and adults for the clinical phase 2 trial of EB-101 to evaluate the effect of COL7A1 gene repair skin grafting on wound tissue repair (Clinical Trial Registration No. NCT01263379).
Skin damage in patients with RDEB usually lasts for months to years due to the inability of the skin to adhere to the underlying dermal tissue, and skin damage can cover a large area of ​​the body. The results of a clinical study originally involving 4 patients showed that after 3 months of EB-101 therapy, 90% of the biopsy samples reconstituted vII-type collagen (C7) was expressed at the junction of the epidermal and dermal tissues of the transplant site. After 6 months of treatment, the proportion of biopsy samples with the same treatment was 66%; after 12 months, the figure was 42%. More importantly, reconstructed vII collagen can be observed in anchored fibrils. In terms of wound repair, compared with the baseline level of wound repair, the proportion of wound repair was 87% after 3 months of treatment, 67% at 6 months, and 50% at 12 months.
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