Lucile Packard Children’s Hospital Stanford, a medical facility in the San Francisco Bay Area, has completed the requirements needed to become a qualified treatment center for Zevaskyn (prademagene zamikeracel), a gene therapy approved earlier this year for recessive dystrophic epidermolysis bullosa (RDEB), according to a press release. The…
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 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,…
Note: This story was updated April 3, 2025, to correct that enrollment criteria in Europe differs from criteria at other sites. A Phase 2/3 clinical trial testing AC-203 (diacerein 1% ointment) in people with epidermolysis bullosa simplex (EBS) is recruiting participants. The trial, EBShield (NCT06073132), seeks to enroll…
Castle Creek Biosciences has secured $75 million in financing to support a Phase 3 trial testing its gene therapy to promote wound healing in people with dystrophic epidermolysis bullosa (DEB). The royalty financing agreement for Castle Creek‘s treatment, D-Fi, was led by Ligand Pharmaceuticals, which made…
France-based pharma company Theravia has obtained rights to an experimental molecule that may be used to treat epidermolysis bullosa (EB). The molecule, known as DCN-tCRK, was discovered by scientists at Tampere University, in Finland. Under the new licensing agreement, Theravia will be in charge of further drug development…
The U.S. Food and Drug Administration (FDA) is expected to decide whether or not to approve the cell therapy pz-cel (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa (RDEB) by April 29, 2025. Abeona Therapeutics, the developer of pz-cel, announced that the FDA has agreed to review a…
Pain is a frequent problem for people with recessive dystrophic epidermolysis bullosa (RDEB), and available medications are often not sufficient to control it, a study reported. “Pain is an almost universal symptom across all types of RDEB,” the researchers wrote, noting that worse pain “generally correlates with worse quality…
Treatment with SNT-6935, an experimental compound that acts to reduce the activity of an enzyme that breaks down proteins, reduced skin damage in a lab model of epidermolysis bullosa acquisita (EBA), a study showed. The study, “Granzyme B inhibition reduces autoantibody-induced dermal–epidermal separation in an ex…
Scientists have devised a new, more efficient method to create gene-edited stem cell therapies for dystrophic epidermolysis bullosa (DEB). “Solving critical bottlenecks, we refine a practical and simplified … protocol for the generation of genetically corrected … skin grafts … for the long-term healing of DEB patient wounds,” the…
Both bone marrow transplant and treatment with stem cells derived from bone marrow may help ease symptoms in some people with epidermolysis bullosa (EB), although a bone marrow transplant carries substantial greater safety risks. That’s according to the review study “Bone marrow transplantation and bone marrow-derived…
Dupilumab, an injection therapy approved for certain inflammatory disorders, may be an effective option to help ease itch and reduce blistering in people with different subtypes of epidermolysis bullosa (EB). That’s according to a study, “Dupilumab improves itch and blistering in different subtypes of epidermolysis bullosa,”…
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