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Krystal Biotech recently dosed the first patient in a Phase 1/2 clinical trial testing KB103, a topical gene therapy candidate for the treatment of dystrophic epidermolysis bullosa (DEB). DEB is a progressive and painful form of epidermolysis bullosa characterized by blistering of the skin. The blistering happens…

Collagen type VII-filled vesicles released by bone marrow-derived mesenchymal stem cells may explain the therapeutic benefits of these cells in recessive dystrophic epidermolysis bullosa (RDEB), a study suggests. The study, “Dual mechanism of type VII collagen transfer by bone marrow mesenchymal stem cell extracellular vesicles to recessive…

Epidermolysis bullosa (EB) can affect the gastrointestinal tract, leading to the development of esophageal stenosis, or a narrowing of the esophagus, according to a new case report. The report, “A rare case of skin blistering and esophageal stenosis in the course of epidermolysis bullosa – case report…

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…

The U.S. Food and Drug Administration has cleared Krystal Biotech‘s investigational new drug application for KB103, a topical gene therapy for dystrophic epidermolysis bullosa (DEB). The company can now start enrolling patients for a clinical trial to test the experimental therapy. KB103 is based on the company’s…

It’s not every day a Southeast Asian monarchy bestows a $100,000 prize on a U.S. government research agency. In fact, it rarely happens. But this year is different. Thailand’s Prince Mahidol Award Foundation has chosen the National Human Genome Research Institute — a division of the National Institutes of Health (NIH)…

Krystal Biotech recently announced that the European Medicines Agency (EMA) has granted the company’s investigational therapy KB103 orphan medicinal product designation for the treatment of dystrophic epidermolysis bullosa (DEB). KB103 is the first investigational non-infectious viral vector (HSV-1) gene therapy for DEB to receive this designation in Europe. In…

The U.S. Department of Defense awarded $3.8 million to a consortium working to develop stem cell-based therapies for patients with inherited skin diseases such as epidermolysis bullosa (EB) and for wound care. The federal defense grant is to advance “discoveries in stem cell-created skin grafts into the manufacturing stage,” according…

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. The Office of Rare Diseases Research (ORDR), headquartered in Bethesda, Maryland, was established in 1993 within the NIH Office of the…

Using a simple blood sample may be sufficient to detect a biomarker for skin cancer in epidermolysis bullosa patients, a new study suggests. This is because the cancer-specific variant of the SLCO1B3 gene is found exclusively in extracellular vesicles detected in blood circulation. The study reporting the findings is titled “Extracellular Vesicles as…

Scientists have found that using inhibitors on specific parts of the so-called complement system, part of the immune system that aids the functioning of antibodies, led to improvements in blister formation in a mouse model of epidermolysis bullosa acquisita (EBA). The findings support the therapeutic potential in EBA of…

Patients with epidermolysis bullosa acquisita sometimes can develop acute renal failure, according to a case report. The report, “Acute renal failure in a patient with epidermolysis bullosa acquisita,” was published in the journal Anais Brasileiros de Dermatologia. Epidermolysis bullosa acquisita (EBA) is a severe autoimmune skin…