News

The U.S. Food and Drug Administration (FDA) is pushing back its timeline for a decision on Abeona Therapeutics’ pz-cel (prademagene zamikeracel), saying the agency needs more time to complete its review of the cell therapy for recessive dystrophic epidermolysis bullosa, known as RDEB. A decision by the FDA on…

In children with epidermolysis bullosa (EB) with airway involvement, a new strategy using both cell and gene therapy showed promise in a new study. Specifically, the reintroduction of the LAMA3 gene that codes for part of a protein called laminin 332 restored the ability of cells to form a healthy barrier…

An inflammatory protein called interleukin (IL)-6 is found at higher levels in people with epidermolysis bullosa who have more severe disease and larger wounds, a study found, suggesting IL-6 may be used to monitor disease progression and response to treatment. The study, “IL-6 levels dominate the serum…

A man with severe, treatment-resistant epidermolysis bullosa acquisita (EBA) experienced sustained clinical remission for at least five years after rituximab therapy. Rituximab (sold in the U.S. as Rituxan and biosimilars) is an antibody that suppresses immune system activity by targeting immune B-cells — responsible for making antibodies, including…

Abeona Therapeutics said its gene-corrected cell therapy, called pz-cel (prademagene zamikeracel), for recessive dystrophic epidermolysis bullosa (RDEB), is on track for a May 25 decision from the U.S. Food and Drug Administration (FDA), and the company is addressing concerns raised during a plant inspection. The FDA conducted a…

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…