News

Almirall has licensed rights to develop and commercialize ZKN-013, an experimental oral treatment for recessive dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB). As part of the deal, Almirall is making an up-front payment of $3 million to ZKN-013’s original developer Eloxx Pharmaceuticals. Eloxx…

The enzyme polo-like kinase 1 (PLK1) may be a potential therapeutic target for squamous cell carcinoma (SCC) associated with recessive dystrophic epidermolysis bullosa (RDEB), according to an analysis of tumor tissue from an RDEB patient with recurring cancer. The gene that encodes PLK1 was almost seven times more active…

The 7th annual Plunge for Elodie is set to raise $500,000 for epidermolysis bullosa (EB) research, which would bring the total raised by the event to date to more than $2.5 million, the EB Research Partnership (EBRP) said. From March 10 to April 27, supporters will jump into…

Blocking the Notch signaling pathway — key to cell-to-cell communication — was found to significantly ease signs of fibrosis, or excessive scar tissue buildup, in skin cells from people with recessive dystrophic epidermolysis bullosa (RDEB) in a new study from Italy. According to the researchers, Notch signaling was elevated…

Recessive dystrophic epidermolysis bullosa (RDEB) is linked with high care costs, particularly for severe forms of the disease, according to a U.K.-based study. Costs associated with dressings and caregivers were significant contributors, despite many of them being unpaid. The findings “highlight the need for adequate…

The gene therapy beremagene geperpavec (B-VEC), applied directly as eye drops, improved the vision in a boy with eye scarring related to recessive dystrophic epidermolysis bullosa (DEB), according to a case report. “Our data support further investigation of B-VEC in the care of patients with dystrophic epidermolysis bullosa with…

The U.S. Food and Drug Administration (FDA)’s review of pz-cel (prademagene zamikeracel), a gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (RDEB), is progressing on schedule. The therapy’s developer Abeona Therapeutics announced it had conducted a biologics license application (BLA) mid-cycle meeting with the FDA and the agency…

A gene-editing therapy to inactivate a harmful mutated copy of the KRT14 gene reversed defects in skin cells taken from a child with epidermolysis bullosa simplex (EBS), according to recent research. The approach restored the ability of these skin cells, called keratinocytes, to form the structural networks that help give the…

Treatment with baricitinib or upadacitinib, two approved medications for rheumatoid arthritis, may be effective for relieving itching in some people with dystrophic epidermolysis bullosa (DEB). That’s according to a small study from Korea in which these medicines were found to reduce patient-rated itch severity scores — in some, by…

Among a group of people with junctional epidermolysis bullosa (JEB), nearly all genetic mutations associated with the condition were unique, which may explain the wide range of symptom presentation and severity among patients, a study reported. Researchers noted that accurately predicting outcomes is challenging because some cases were…

A gene editing strategy corrected a disease-causing mutation in lab-grown cells derived from the skin of two individuals with recessive dystrophic epidermolysis bullosa (DEB), according to a study by researchers in the U.S. and France. “With this promising non-viral approach, we achieved therapeutically relevant specific gene editing,” the researchers…

The presence of loose joints, or hypermobility, contributes to the impaired walking ability seen in many children with epidermolysis bullosa (EB), a new review study reports. Researchers at a U.K. hospital found that 48 children of 59 referred for physiotherapy had an altered gait pattern, meaning their walking ability…