Another potential treatment for recessive dystrophic epidermolysis bullosa (RDEB) is one step closer with the recent news that Abeona Therapeutics have completed their patient clinical trials of their engineered cell therapy, EB-101. Topline results from the study are due within a month and pending a positive outcome, Abeona will then submit to the FDA for approval in the US.

The EB-101 treatment involves using gene transfer to deliver the COL7A1 gene into the patient’s skin cells (keratinocytes and its progenitors) and transplanting those cells back to the patient. B-101 is being investigated for its ability to enable normal Type VII collagen expression which could facilitate wound healing with RDEB patients.

Genetic engineering to fix the broken collagen gene in skin cells of people with RDEB means a sheet of their own cells with working collagen protein can be grown and transplanted onto wounds that are slow to heal. Fixing the gene in a person’s own cells means the transplant won’t be attacked by their immune system and rejected - as it would if another person’s healthy skin was transplanted. If approved, this would be the first skin transplant treatment for people with RDEB.

Dr Sagair Hussain, DEBRA Director of Research

Should EB-101 successfully gain FDA approval, Abeona would then likely submit for EMA (European Medicines Agency) approval for the EU and then, if successful, MHRA (Medical Healthcare & Products Regulatory Agency) approval for use in Great Britain.

The likelihood is that this treatment won’t be available to patients in Great Britain until 2025 at the earliest however it is encouraging to see another potential EB treatment getting one step closer.

To find out more, view the full press release from Abeona Therapeutics.