StemRIM (TYO:4599) said a patent has been granted for technology underpinning its stem cell gene therapy candidate SR-GT1, aimed at treating dystrophic epidermolysis bullosa, according to a Monday filing on the Tokyo Stock Exchange.
The patent, filed by Osaka University, covers the use of genetically modified cells administered directly into patients' blisters as a treatment approach for the rare hereditary skin disorder.
Dystrophic epidermolysis bullosa is caused by abnormalities in type VII collagen, which binds the epidermis and dermis.
StemRIM's approach involves collecting mesenchymal stem cells from patients' blisters, introducing a functional type VII collagen gene, and re-administering the modified cells to affected areas. The cells are designed to engraft in the skin and provide sustained collagen production.
The investigational therapy is being manufactured with support from the Japan Agency for Medical Research and Development, with preparations under way for a physician-led clinical trial.
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