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…
Healiva and C4U will collaborate to develop a next-generation gene editing therapy — focusing on new technology to correct gene mutations — for epidermolysis bullosa (EB) patients, who are sometimes known as butterfly children for their fragile skin. Combining C4U’s expertise in gene editing technology and Healiva’s…
The diabetes medication metformin slowed tumor growth and prolonged survival in a mouse model of aggressive squamous cell carcinoma (SCC), the most common type of cancer seen in people with recessive dystrophic epidermolysis bullosa (RDEB), in a recent study. It did so by inhibiting cellular processes used by…
A rare case of epidermolysis bullosa acquista (EBA) was found in an older man in France with systemic sclerosis (SSc), according to a new report by researchers who noted the unusual co-occurrence of the two autoimmune diseases. “Several autoimmune or inflammatory diseases have been reported in association with…
Abeona Therapeutics has been working with the U.S. Food and Drug Administration (FDA) ahead of its planned submission of a biologics license application (BLA) for EB-101, with an aim of maximizing the chances of having its cell therapy approved as a treatment for recessive dystrophic epidermolysis bullosa (RDEB).
Airway involvement is a rare but burdensome and life-threatening manifestation of epidermolysis bullosa (EB) that requires prompt and proper management, according to a recent case report. Researchers in Saudi Arabia described four children with EB who were brought to their clinic showing involvement of their larynx, or voice box,…
EB-101, an investigational gene-corrected cell therapy, safely and effectively heals large, chronic wounds and eases pain in people with recessive dystrophic epidermolysis bullosa (RDEB), according to top-line data from the Phase 3 VIITAL trial. “We are very pleased with the topline results from our pivotal VIITAL study, which…
A pilot trial will investigate the effects of antimicrobial spray APR-TD011 on bacterial colonization in the wounds of people with epidermolysis bullosa (EB), the therapy’s developer, Relief Therapeutics, announced. The treatment’s active ingredient is hypochlorous acid, a chemical with strong and broad-acting antimicrobial properties. It is approved and…
Patients with recessive dystrophic epidermolysis bullosa (RDEB) had distinct blood metabolic profiles compared with healthy people, a study showed. The most significantly altered molecules were amino acids — the building blocks of proteins — most of which were at lower levels in RDEB patients and correlated with disease severity.
Self-reactive antibodies against the stress-induced heat shock protein 70 (Hsp70) were higher in the blood of patients with epidermolysis bullosa acquisita (EBA), a study found. In a corresponding mouse model, the researchers observed the autoantibodies contributed to a worse disease course, likely through the mediation of inflammatory pathways. “This…
INM-755 was found to be safe in five adults with epidermolysis bullosa (EB) who were treated with the investigational cannabinol cream in a Phase 2 trial. The safety data led an independent data monitoring committee to agree that it is safe to enroll adolescents, ages 12–17, in the…
Two new mutations in the COL7A1 gene, one inherited from each parent, were identified as the cause of recessive dystrophic epidermolysis bullosa (DEB) in a 10-year-old boy, a case study reported. Further functional analyses suggested that each mutation had only mild effects — supported by the fact that his…
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