Dr. Sarah Hedtrich, associate professor in the School of Biomedical Engineering at UBC, has been sub-awarded US$5.5 million to help develop a first-of-its-kind precision genetic medicine for devastating rare skin diseases. The award is as part of a US$26 million international research initiative funded by the US Advanced Research Projects Agency for Health (ARPA-H)
The project, “Curing Rare Skin Diseases with NueSKIN,” brings together leading experts to develop a topical gene-editing therapy for Epidermolysis Bullosa, a group of rare inherited skin disorders that cause the skin to blister and tear from even minor friction or trauma. Patients with the condition often experience chronic, painful, non-healing wounds, severe infections and significantly reduced quality of life, with some forms of the disease associated with increased mortality
The team’s approach, called NueSKIN (Nucleic acid Skin), is designed to deliver precision gene-editing medicines directly to skin wounds. The technology combines lipid nanoparticles with a base-editing system that can correct disease-causing genetic mutations at their open wounds, offering the potential for a safe, localized treatment with minimal systemic exposure
The project builds on previous work by Dr. Hedtrich showing the potential of topical gene-editing treatments for skin disorders
“Patients with Epidermolysis Bullosa (EB) live with chronic, painful wounds that have no curative treatment,” said Dr. Hedtrich. “Our goal is to harness the power of precision gene editing to develop a topical therapy that corrects the underlying genetic cause of disease directly in the skin. If successful, this approach could not only transform care for patients with EB but also establish a platform for treating many other inherited skin disorders.”
The project will focus on recessive dystrophic Epidermolysis Bullosa (RDEB), one of the most severe forms of EB. By correcting disease-causing mutations directly in patients’ skin, the researchers aim to promote durable wound healing and dramatically improve quality of life
Dr. Hedtrich will work together with the internationally recognized experts Dr. Jean Tang and Dr. Tony Oro, both professors of dermatology at Stanford Medicine, and Dr. Fyodor Urnov of the Innovative Genomics Institute at University of California, Berkeley
The multidisciplinary team brings together expertise in rare genetic skin diseases, gene editing, nanomedicine and dermatology to accelerate the development of a platform technology that could ultimately be adapted to treat many of the more than 1,000 inherited genetic skin disorders
If successful, NueSKIN could establish a new paradigm for topical precision genetic medicines, transforming the treatment of rare skin diseases while creating a platform for broader applications across dermatology and genetic medicine
The award is part of ARPA-H’s Treating Hereditary Rare Diseases with In Vivo Precision Genetic Medicine (THRIVE) program, which aims to advance personalized, curative medicines for rare genetic diseases
A version of this story was originally published on theUBC School of Biomedical Engineering website
