NEWS

GENEGRAFT: A European research project on safe and efficient ex vivo gene therapy approach for a permanent treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

The 5 year project started in March 1st 2011.

 

GENEGRAFT was officially launched in Paris on March 4th, gathering the scientist involved on the project.

The GENEGRAFT project combines the complementary expertise of 5 international research groups and one patient organization that aimed to develop a gene therapy strategy of recessive dystrophic epidermolysis bullosa. The selected strategy is the grafting of genetically modified corrected autologous epithelia or skin equivalents.

To achieve this goal, the project was constructed on a 3 step process: a) selection of patients with optimal clinical and biological features for a fist clinical trial; b) Production of clinical lots of the vector under GMP conditions and c) preparation of a phase I/II clinical trial. The successes of this strategy will permit, by using the corresponding gene in each case, to extend the area of application of this technology to other inherited disorders.

The successes of this strategy will permit, by using the corresponding gene in each case, to extend the area of application of this technology to other inherited disorders.

 

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. Read more

CLINICAL GRADE VIRAL BATCHES

 

 

 

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.

Publication: "HEK293-Based Production Platform for γ-Retroviral (Self-Inactivating) Vectors: Application for Safe and Efficient Transfer of COL7A1 cDNA" Hum Gene Ther Clin Dev. 2014 Dec;25(4):218-28. doi: 10.1089/humc.2014.083.

Read more