In a small study in Japan, the topical gene therapy Vyjuvek (beremagene geperpavec) was shown to work similarly to what was seen in an earlier U.S.-based trial, now safely and durably closing wounds for four Japanese people with dystrophic epidermolysis bullosa (DEB). The new data comes from an…
The first recessive dystrophic epidermolysis bullosa (RDEB) patient will be dosed with the cell-based gene therapy Zevaskyn (prademagene zamikeracel) in the next few months, with several others lined up to follow, according to developer Abeona Therapeutics. That patient has already been selected, and their skin cells have been…
PKC412, a medication already used for treating certain blood cancers, has the potential to be repurposed for treating epidermolysis bullosa simplex (EBS), according to a preclinical study. Using skin cells from people with various EBS-causing genetic mutations, scientists found that PKC412, also known as midostaurin, made the cells more…
The first person has been dosed in a Phase 3 trial testing KB803, Krystal Biotech’s experimental eye drop formulation of the gene therapy beremagene geperpavec (B-VEC), for the treatment of eye problems in people with dystrophic epidermolysis bullosa (DEB). Krystal already markets a topical gel formulation of B-VEC…
Long-term treatment with the topical gene therapy Vyjuvek (beremagene geperpavec) was associated with continued wound closure and high patient satisfaction among people with dystrophic epidermolysis bullosa (DEB) who took part in an open-label extension (OLE) study. The therapy was also well tolerated over up to more than two…
Traws Pharma said it will advance development of skin cancer therapy rigosertib, after data showed the therapy led to a partial or complete treatment response for four recessive dystrophic epidermolysis bullosa (RDEB) patients with advanced forms of the skin cancer squamous cell carcinoma (SCC). The data came from two…
The European Commission has approved the topical gene therapy Vyjuvek (beremagene geperpavec) to treat wounds, starting at birth in people with dystrophic epidermolysis bullosa (DEB) who have disease-causing mutations in the COL7A1 gene. The decision, which applies to all European Union countries, and includes Iceland, Norway, and Liechtenstein,…
Long-term treatment with Filsuvez (birch triterpenes) was well tolerated and associated with sustained reductions in wound burden for people with dystrophic epidermolysis bullosa (DEB) and junctional epidermolysis bullosa (JEB). That’s according to final two-year data from the open-label phase (OLP) of the Phase 3 EASE trial…
In interviews, people with epidermolysis bullosa (EB) reported that living with these rare skin diseases has broad impacts on their psychosocial health. This burden was seen across physical, emotional, social, and functional domains, per the study, conducted in Europe. According to the researchers, the findings emphasize the importance of…
A gene-editing therapy made to correct a genetic defect in the LAMB3 gene that’s known to cause junctional epidermolysis bullosa (JEB) worked as intended and restored more normal laminin 332 protein levels in skin cells from a JEB patient. To make the treatment more precise and safer, the scientists…
Researchers have developed a framework for categorizing the severity of mutations in the COL7A1 gene that cause recessive dystrophic epidermolysis bullosa (RDEB) which could eventually be used to better inform clinical care. Patients categorized as having so-called “high-impact” mutations in a recent study were found to be at an…
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…
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