Gene-edited Spray-on Skin Cells May Show Promise for DEB

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by Lindsey Shapiro PhD |

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A gene-edited skin cell therapy — called Spray-On Skin cells — promotes skin healing by correcting the mutation associated with recessive dystrophic epidermolysis bullosa (DEB), preclinical research from Avita Medical shows.

“These data, while early, demonstrate promise … for treatment of epidermolysis bullosa with gene-corrected skin cells,” Mike Perry, PhD, CEO of Avita, said in a press release.

The research, which the company says “successfully established proof of concept in … key areas of cell-based gene therapy,” was conducted in partnership with scientists at the Gates Center for Regenerative Medicine at the University of Colorado.

“These initial results are a meaningful step forward in the advancement of our epidermolysis bullosa program,” said Dennis Roop, PhD, director of the Gates Center.

DEB is caused by mutations in the COL7A1 gene responsible for making part of the type VII collagen protein. Type VII collagen connects layers of the skin; when functioning improperly, the connections are fragile, resulting in blisters, chronic wounds, and scar tissue.

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While there is no cure for DEB, therapies that target the faulty gene could help improve wound healing.

Avita, which seeks to advance the standard of care for burn patients, developed a new technology platform it calls the RECELL system. It uses a person’s own skin sample to grow a suspension of “spray-on skin cells.” The suspension, which can be produced within 30 minutes, contains skin cells and growth factors that promote regeneration.

The RECELL system was approved by the U.S. Food and Drug Administration in September 2018 for the treatment of acute thermal burn wounds in adults.

In collaboration with the Gates Center, Avita is now testing whether a genetically modified form of RECELL can correct deficits in COL7A1 and improve skin regeneration.

To do this, a normal copy of COL7A1 is introduced to skin cells, restoring gene function. These healthier cells may then promote healing when sprayed onto a wound.

Preclinical data showed that treatment with these gene-modified skin cells successfully regenerated skin.

Perry said Avita’s Spray-On Skin cells show promise for “new and broad applications such as skin rejuvenation and genetic skin defects.”

The technology could eventually be an alternative to other cell-based therapies that require sheets of skin to be grown and surgically implanted, the company suggests.

Avita also is partnering with the Houston Methodist Research Institute, in Texas, to investigate the potential of spray-on skin cells for anti-aging skin rejuvenation.

Overall, these findings support the further development of genetically modified spray-on treatments for epidermolysis bullosa and other skin conditions, according to Avita.

“We’re looking forward to continuing to work with Avita Medical on this novel approach to delivering gene-edited skin cells to patients,” Roop concluded.

The Gates Center is involved in other collaborations to find new treatments for DEB, one of four main types of epidermolysis bullosa.

Its partnership with Stanford and Columbia universities, called the Epidermolysis Bullosa iPS Cell Consortium, received $1.6 million in funding in 2020 for the development of stem cell-based therapies for skin disease, including epidermolysis bullosa.

The iPS Cell Consortium was founded by the EB Research Partnership and the EB Medical Research Foundation in 2016 to speed progress toward new treatments.