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    Home > Active Ingredient News > Drugs Articles > EMA grants gene therapy eb-101 orphan drug status to abeona

    EMA grants gene therapy eb-101 orphan drug status to abeona

    • Last Update: 2017-03-10
    • Source: Internet
    • Author: User
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    Source: on March 8, 2017, abeona therapeutics, a biotech company focused on developing gene therapy methods for severe and rare diseases, said that the orphan drug committee of EMA gave eb-101 the orphan drug status of gene therapy to treat recessive dystrophic epidermolysis bullosa (RDEB) It is a kind of hereditary skin disease which is characterized by blistering and involving the whole body Eb-101 is the fourth orphan drug designated by EMA to abeona, which has shown promising safety and efficacy data in the ongoing phase 1 / 2 clinical trials Dr Timothy J Miller, CEO and President of abeona therapeutics, said: "eb-101 is another gene therapy for the company to obtain orphan drug status, further strengthening the company's orphan drug product portfolio, and the granting of orphan drug status proves the effectiveness of this therapy in the treatment of rare diseases without drug availability The granting of orphan drug status means that eb-101 has gained a 10-year market monopoly in Europe, and drugs with similar mechanisms of action for the same disease will not be available for sale in this period " In the ongoing phase 1 / 2 clinical trial, patients' skin cells that have been genetically modified (which can express type II collagen) are transplanted to patients to achieve the purpose of disease treatment This clinical trial has shown the effect of tissue repair and good safety in patients with recessive dystrophic epidermolysis bullosa Stanford University researchers are recruiting adolescent and adult patients for the eb-101 clinical phase 2 trial to evaluate the effect of COL7A1 gene repair skin transplantation on wound tissue repair (Clinical Trial Registration No nct01263379) Skin damage in patients with RDEB usually lasts for months to years due to the skin's inability to attach to the underlying dermal tissue, and the skin damage will cover a large area of the body The results of the initial clinical study involving four patients showed that 90% of the reconstructed collagen type II (C7) in the biopsy samples was expressed at the junction of the epidermis and dermis of the transplantation site 3 months after eb-101 treatment, while 66% of the biopsy samples showed the same treatment results 6 months after treatment, and 42% 12 months later More importantly, the reconstructed type II collagen can be observed in the anchor fibril species In terms of wound repair, compared with the baseline level of wound repair, the proportion of wound repair after 3 months of treatment was 87%, 67% at 6 months and 50% at 12 months Steven H rouhandeh, executive chairman of abeona therapeutics, said: "the encouraging results of the eb-101 clinical trial enhance our confidence in further promoting clinical research, and also urge us to further strengthen our cooperation with research institutions (Stanford University) We are very proud to be able to work with groups such as the bullous dermatology research foundation and bullous dermatology research organization to accelerate the use of eb-101 in patients with recessive dystrophic epidermolysis bullosa.
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