Alice Melao,  —

Fibrocell Science’s investigational gene therapy FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) has received the regenerative medicine advanced therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA). The new status adds to the rare pediatric disease and fast track designations…

Fibrocell Science and Castle Creek Pharmaceuticals are partnering to further develop the investigational gene therapy FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Under the collaboration, Castle Creek will support all development and manufacturing costs and will receive an exclusive license to commercialize FCX-007…

Patients with recessive dystrophic epidermolysis bullosa (RDEB), particularly those with delayed puberty, have a higher risk of osteoporosis — lower bone density — in the lumbar spine than healthy people of the same age, a study shows. The study, “Retrospective…

The care and management of a 6-month-old baby with epidermolysis bullosa (EB) simplex during surgery for a corneal transplant was described for the first time in a recent case report. The study, “Anaesthetic management of a rare case of paediatric epidermolysis bullosa,” was published in the Indian Journal…

Krystal Biotech has completed the construction of its new manufacturing facility Ancoris, which will support the clinical and commercial development of KB103, its lead product candidate for the treatment of dystrophic epidermolysis bullosa (DEB). Ancoris was designed to meet all projected commercial demands of KB103. Located near…

After an interim analysis of a Phase 3 trial testing Amryt Pharma’s investigational therapy AP101 in epidermolysis bullosa (EB) patients, an independent data monitoring committee (IDMC) has recommended the trial’s continuation, along with an increase in participants. The enrollment of 48 additional patients, up to a total of 230, is…

The EB Research Partnership (EBRP) and Epidermolysis Bullosa Medical Research Foundation (EBMRF) granted $900,000 to Fibrocell, a gene therapy company, to support the clinical development of FCX-007 to possibly treat recessive dystrophic epidermolysis bullosa (RDEB). “We are grateful for this investment from EBRP and EBMRF that…

The commonly used antibiotic gentamicin (brand name Garamycin) can reverse the effects of about 80% of genetic mutations affecting the LAMB3 gene that cause Herlitz junctional epidermolysis bullosa (H-JEB), according to researchers. This finding was reported in the study, “Gentamicin induces LAMB3 nonsense mutation readthrough and restores…

Menlo Therapeutics’ investigative therapy serlopitant seems to effectively reduce itch in patients with epidermolysis bullosa, according to results from a small exploratory Phase 2 trial. The trial results were presented by researchers from the dermatology department at Stanford University School of Medicine at the International Investigative Dermatology 2018…

A rare case of autoimmune-driven epidermolysis bullosa (EB) was described in a patient who also had an undiagnosed mild genetic subtype of the disease. The case study, “Epidermolysis Bullosa (EB) Acquisita in an Adult Patient with Previously Unrecognized Mild Dystrophic EB and Biallelic COL7A1 Mutations…

Amryt Pharma’s lead investigative therapy AP101, developed to reduce the time it takes for skin wounds to close, is recruiting patients with epidermolysis bullosa for a pivotal Phase 3 trial. AP101, also known as oleogel-S10, is a topical product based on refined birch bark extract comprising 72%…

Krystal Biotech plans to file an investigational new drug (IND) application for its lead therapy candidate KB103 for the treatment of dystrophic epidermolysis bullosa, the company announced. The IND’s approval would allow a Phase 1/2 clinical trial to move forward to assess KB103’s safety, tolerability, and efficacy. The trial already…