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…
Treatment with dupilumab, a medicine approved for certain inflammatory disorders, eases itch and disease severity in people with different subtypes of dystrophic epidermolysis bullosa (DEB), a small study suggests. Dupilumab-related benefits seen in a woman before she stopped treatment and got pregnant were mostly maintained throughout her pregnancy and…
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…
Rheacell and AOP Health are partnering to make stem cell therapies available for people with epidermolysis bullosa (EB) in Europe and elsewhere. “This partnership marks a significant milestone for Rheacell and the further development of our stem cell medication programs,” Christoph Ganss, MD, Rheacell’s CEO, said in…
Researchers used a small molecule called M3814 for more accurate editing of genetic mutations causing recessive dystrophic epidermolysis bullosa (RDEB), according to a new study. This method enabled skin cells from three patients to restore their production of type VII collagen (C7) protein, which they lacked, the researchers noted.
Ann & Robert H. Lurie Children’s Hospital of Chicago opened the first Qualified Treatment Center (QTC) in the U.S. authorized to offer Zevaskyn (prademagene zamikeracel) for people with recessive dystrophic epidermolysis bullosa (RDEB). Developed by Abeona Therapeutics, Zevaskyn, formerly known as pz-cel or EB-101, was recently approved…
Note: This story was updated June 18, 2025, to correct the source of the analysis of Phase 3 trial data. Paradigm Therapeutics is planning to seek U.S. Food and Drug Administration (FDA) approval for the experimental cream SD-101 to treat all forms of epidermolysis bullosa (EB). Paradigm said…
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,…
The U.S. Food and Drug Administration (FDA) has approved the cell-based gene therapy prademagene zamikeracel, previously pz-cel or EB-101, for treating wounds in adults and children with recessive dystrophic epidermolysis bullosa (RDEB). The therapy’s developer Abeona Therapeutics will market it under the brand name Zevaskyn. According to Abeona,…
People with severe recessive dystrophic epidermolysis bullosa (RDEB) are more likely to develop disease-related complications, and at a younger age, than those with milder or more localized disease types, a U.K. study suggests. Complications include skin cancer, swallowing and feeding difficulties, with patients commonly needing procedures to improve feeding…
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…
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