News

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…

The U.S. Food and Drug Administration (FDA) has approved Filsuvez (birch triterpenes) to treat skin wounds in patients with dystrophic epidermolysis bullosa (DEB) or junctional epidermolysis bullosa (JEB) ages 6 months or older. The announcement makes Filsuvez the first FDA-approved therapy for JEB, according to Chiesi Global…

A new gene-editing strategy that does not require the use of a virus can effectively correct a COL7A1 gene mutation in skin cells derived from people with recessive dystrophic epidermolysis bullosa (RDEB), according to a recent report. Most gene therapies in development require the use of viral carriers to…

Tofacitinib, an oral anti-inflammatory medication, eased itching and reduced skin lesions in a young man with dystrophic epidermolysis bullosa (DEB) pruriginosa caused by two newly identified mutations, according to a report from China. The report, “Novel compound heterozygous mutations of the COL7A1 gene in a Chinese patient with…

The U.S. Food and Drug Administration (FDA) has granted priority review to an application seeking approval of pz-cel (prademagene zamikeracel) — a cell therapy previously known as EB-101 — for people with recessive dystrophic epidermolysis bullosa (RDEB). Granting priority review shortens the time it will take the agency to…

EB Research Partnership (EBRP), an organization dedicated to funding research into treatments for epidermolysis bullosa (EB), has raised more than $5 million through a three-day series of events in October. The outcome is “beyond what we could have ever imagined,” Michael Hund, CEO of EBRP, said in a…