Spray-on gene therapy for recessive dystrophic epidermolysis bullosa (Lwin 1)
Completed| Project lead | Dr Su Lwin |
| Organisation | King’s College London, London, UK |
| Partner organizations & collaborators | Prof. John McGrath and Dr Michael Antoniou from St. John’s Institute of Dermatology, Department of Medical and Molecular Genetics, Kings College London, UK Collaborators Prof. Alain Hovnanian and Dr. Matthias Titeux, INSERM UMR 1163, Imagine Institute, Paris, France. |
| Project budget | GBP 174,022.66 |
| Start date / Duration | 01. Jun 2019 / 24 months |
| Funder(s) / Co-Funder(s) | DEBRA UK, EB MSAP/EBEP Recommended |
| Research area | Molecular therapy, Cellular therapy |
Project details
Short lay summary
Recessive dystrophic epidermolysis bullosa (RDEB) is one of the most severe forms of EB with significant disease burden and high mortality due to cutaneous squamous cell carcinoma (cSCC). Resulting from mutations in the COL7A1 gene that produces type VII collagen, it leads to blisters and tissue fragility. Only palliative care is currently available; therefore, patient-friendly effective therapies are an unmet need. This group proposes to address this challenge by developing a spray-on gene therapy for RDEB designed for longer-lasting therapeutic benefit, including the prevention of scarring. Building on the recent gene therapy clinical trial Lenticol-F*, the plan is to supplement RDEB patient's own skin cells with functional copies of the COL7A1 gene. To achieve this, they envisage using a form of disabled virus called lentivirus to deliver the gene into keratinocytes and fibroblasts and then spray them on the affected skin with the aid of the SkinGun™ designed by the biotech company RenovaCare Inc. Initially, the project will seek to generate proof-of-concept and evaluate whether the sprayed cells form functional skin using animal models. Their objective is to assess the efficiency with which these viral vectors deliver the functional gene to patients’ own cells and verify whether the therapeutic benefit is long-lasting.
Strategic relevance
The reliance on cell spray application to deliver the gene coding for type VII collagen would eliminate the need for invasive procedures and provide a patient-friendly treatment. Moreover, gene supplemented cells could be more quickly grown and stored at low temperatures until the patient is ready to receive them, which would significantly improve the clinical feasibility of gene therapy.
*LENTICOL F is funded by Cure EB and administrated by DEBRA UK. To read about the project that has contributed to this new research, please click here.