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Progress in developing a skin-grafting gene therapy for RDEB

The EU-funded GENEGRAFT project has developed a safer vector for collagen 7 gene correction, and is now planning a small clinical trial to assess tolerability and efficacy, in Europe for 2016.

The European consortium is developing a skin-grafting gene therapy for RDEB: by the end of the project in late 2016, the aim is to have grafted up to three people with RDEB, in a small-scale, early-stage, clinical trial to demonstrate the effectiveness of the treatment, and to assess how well tolerated it is by the patients.

The project has now achieved several key steps, most notably the development of a safer 'self-inactivating' vector technology for putting the correct collagen 7 gene into the patient's own skin cells (both keratinocytes and fibroblasts), from which the skin grafts are engineered.

The surgical teams to be involved in the GENEGRAFT trial have defined the grafting procedure, post-operative care and follow-up of patients, and are getting these approved too. The production methods are to the highest clinical Good Manufacturing Practice (GMP) standards, and preclinical studies are now thoroughly testing the safety and stability of the vector and collagen gene.

When the essential safety studies are completed, and regulatory approval has been obtained, the project will be at a stage where the first patient can be enrolled in the trial and receive the gene-corrected skin graft. In such an early-stage clinical trial it is important that only people for whom the treatment will be safe, and have the clinical characteristics that mean they could benefit, are invited to participate. After a lengthy process of careful screening of people with RDEB, 10 adults in France and 10 adults in the UK have been selected as possible candidates to be enrolled in the trial. www.genegraft.eu

Dr. Clare Robinson, Head of Research DEBRA International


DEBRA International provides information and news that may be of interest to people with EB, but does not recommend or endorse products, or participation in any particular clinical trial. We strongly recommend that anyone considering a treatment or clinical trial should also speak with their doctor, or medical team, who helps them manage their EB.

Logo of the GENEGRAFT research programme

Logo of the 7th framework programme of the European Union