Marisa Wexler, MS,  senior science writer—

A new collaboration between the University Carlos III Madrid (UC3M), Almirall, and the Medina Foundation aims to find treatments for recessive dystrophic epidermolysis bullosa (RDEB) and other genetic diseases caused by nonsense mutations, or those that result in shortened proteins. The project is partially funded by…

The European Medicines Agency (EMA) granted orphan drug designation to an investigational, and yet unnamed, potential therapy for recessive dystrophic epidermolysis bullosa (RDEB) based on CRISPR-Cas9 gene editing. This designation confers certain incentives, including advice from the EMA during the development process, reduced or exempted fees and…

Castle Creek Biosciences has received a $75 million investment to advance its gene therapy program, including FCX-007, a treatment candidate for people with recessive dystrophic epidermolysis bullosa (RDEB) that now in a pivotal clinical trial. “We are proud to have the strategic support of world-class investors whose impact…

InMed Pharmaceuticals does not expect that the COVID-19 pandemic to significantly affect data collection in its Phase 1 trial testing INM-755, a potential epidermolysis bullosa (EB) treatment, in healthy volunteers. INM-755 is a topical cream, whose active ingredient is cannabinol (CBN), a compound present in trace amounts…

Using mesenchymal stem cells to treat adults with recessive dystrophic epidermolysis bullosa (RDEB) appears to be safe and may ease symptoms such as itching for at least two months, a Phase 1/2 clinical trial shows. The results were described in the study “A Phase I/II open-label…

The investigational oral treatment Serlopitant may be able to reduce itching in people with epidermolysis bullosa (EB), results from a small Phase 2 clinical trial suggest. However, most results did not achieve statistical significance, which means further studies will be needed to draw firm conclusions. The results were…

New guidelines on foot care for people with epidermolysis bullosa (EB) highlight the benefits of proper care and footwear, and recommend that a podiatrist be involved in routine health checks for people with EB. The guidelines, “Foot care in Epidermolysis bullosa: Evidence-based Guideline,” were published in the …

A novel gene editing technique was able to correct mutations in COL7A1 in cells taken from people with recessive dystrophic epidermolysis bullosa (RDEB), providing a proof of concept for using the editing technique in the disease. The finding was published in the Journal of Investigative Dermatology in a study titled,…

EB-101, a cell therapy candidate from Abeona Therapeutics, induced sustained wound healing lasting for three years or more in a large proportion of people with recessive dystrophic epidermolysis bullosa (RDEB), the long-term follow-up of a Phase 1/2 trial shows. Data were detailed at the 77th Annual Meeting of the…

A specific class of T cells – called Th17 cells — may drive inflammation in patients with epidermolysis bullosa simplex (EBS) generalized severe, and targeting these inflammatory pathways might lessen blistering in these patients. The study, “Epidermolysis bullosa simplex generalized severe induces a T helper 17 response and…