News

Gene therapy applied as eye drops improves vision for boy with DEB

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…

FDA review of RDEB cell therapy pz-cel remains on schedule

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…

2 arthritis medications may help relieve itching for DEB patients

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…

Unique JEB mutations explain wide clinical variability, study shows

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…

Gene editing increases type VII collagen production in skin cells

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…

FDA approves Filsuvez to treat dystrophic and junctional EB

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…