(Public Domain Pictures, Pixabay)
26 Nov. 2019. A company making regenerative skin therapies and a medical school research center are developing a spray-on treatment for a genetic skin disorder. University of Colorado’s medical school in Anschutz is collaborating with Avita Medical Ltd. in Melbourne, Australia on the technology, and received an National Institutes of Health grant to fund the project.
Epidermolysis bullosa is a group of genetic disorders causing the skin to become fragile and easily blister, even from mild rubbing or scratching. People with epidermolysis bullosa do not produce the type 7 collagen protein that strengthens and supports connective tissue and binds together different skin layers, from a mutation in the COL7A1 gene. The missing protein causes a debilitating condition, which in severe cases can lead to widespread blistering, disfigurement, vision loss, and can be life threatening. The only treatments available are palliative, such as bandages.
The Gates Center for Regenerative Medicine at Colorado’s medical school is developing a therapy for epidermolysis bullosa with genetically modified stem cells, with colleagues at Stanford and Columbia universities, in a collaboration that began in 2016. The researchers so far advanced the stem cell technology to produce gene-edited stem cells for films and skin grafts applied to wounds and blistering from epidermolysis bullosa.
The partnership with Avita Medical Ltd. seeks to design spray-on stem cells as another treatment option. Avita Medical develops spray-on treatments for burns and skin wounds using a sample of a patient’s skin cells in a suspension to help regenerate new outer skin layers. The company’s Recell product that harvests a patients skin cells and sprays the suspended cells on wounds was cleared by FDA in September 2018 for treating burns.
In the agreement with University of Colorado, Avita and the university’s medical school are collaborating on a treatment for epidermolysis bullosa that sprays gene-edited stem cells on affected skin areas. A sprayed treatment can help in cases where a faster treatment is needed than skin grafts. The collaboration covers joint research on the treatments through preclinical testing. Under the agreement, Avita Medical has an option to license the technology developed by the project, but financial details were not disclosed.
The spray-on stem cell treatments are one part of a recent research grant to University of Colorado’s medical school from National Institute of Arthritis and Musculoskeletal and Skin Diseases, part of National Institutes of Health. The two year, $800,000 award to the Colorado-Stanford-Columbia consortium also supports development of a systemic treatment for epidermolysis bullosa that can treat internal injuries caused by the disease, as well as on the skin. Dennis Roop, director of the Gates Center is the principal investigator on the study, with dermatology professor and Gates Center colleague Ganna Bilousova as co-investigator.
“We believe that spray-on skin cells technology combined with our genetically corrected cells has the potential to be game changing in the treatment of this disease,” says Bilousova in a joint university-company statement. “This combination could reduce time to treatment, lower manufacturing complexity, reduce costs, and improve patient outcomes.”
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