Early Study of RDEB Gene Therapy Using SkinGun as Delivery Vehicle

Early Study of RDEB Gene Therapy Using SkinGun as Delivery Vehicle
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SkinGun, a device designed to gently spray stem cells onto the skin of severe burn victims, is being tested in an early study of a potential gene therapy for people with recessive dystrophic epidermolysis bullosa (RDEB).

The preclinical work, underway at King’s College London, is exploring RenovaCare’s SkinGun in applying cells genetically engineered to promote skin healing.

“We are proud to support this much-needed gene therapy work on RDEB at King’s College London. It’s especially inspiring that this preclinical study marks the first-ever use of our SkinGun for a therapeutic application beyond … burns and wounds,” Alan L. Rubino, president and CEO of RenovaCare, said in a press release.

SkinGun was developed to spray CellMist SolutionRenovaCare’s liquid suspension containing a patient’s own regenerative skin stem cells — directly onto wounds of people with second-degree burns. The company reports that, to date, more than 70 people have received this experimental treatment, avoiding potentially painful skin graft surgeries and long hospital stays.

While RenovaCare is still focusing on its products as initially intended, it received new patent protections in 2019 that enabled extending the use of SkinGun to spray different types of cells and tissues, including the preclinical RDEB gene therapy study.

“Our preclinical investigation of this new gene therapy with the SkinGun provides for a non-invasive gentle spray of regenerative cells to the wound site in order to improve the wound site and minimize pain,” Su Lwin, a study co-principal investigator with St John’s Institute of Dermatology and King’s College London.

According to Lwin, the study is assessing the long-term benefits of the gene therapy delivered with SkinGun in preventing scarring. Such scarring “can severely restrict motion and lead to permanent disfigurement in RDEB,” she said.

The study, funded by DEBRA UK, results from a previous research agreement between RenovaCare and King’s College London.

“This preclinical study will provide us with essential spray-on gene therapy data required for human applications,” Michael Antoniou, head of the Gene Expression and Therapy Group at King’s College London, and a co-principal investigator of the study, said.

Added John McGrath, also a co-principal investigator, chair in cutaneous medicine at King’s College, and head of the St John’s institute: “We hope to translate this data into readily-available clinical therapies to benefit a wider population of RDEB sufferers.” 

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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José holds a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease.

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Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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