News

Abeona Therapeutics plans to start a Phase 3 trial in 2019 testing the cell therapy candidate EB-101 as a treatment for recessive dystrophic epidermolysis bullosa (RDEB). The multi-center, randomized study, named VITAL, will compare treatment with EB-101 to untreated wounds in the same patient. In total, 10-15 patients…

Constant Pharmaceuticals recently announced plans to begin a development program for TXA127 for the treatment of epidermolysis bullosa (EB). The potential therapy is expected to enter Phase 2 clinical trials in EB patients in 2020. The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA)…

A 1% diacerein ointment could be a safe and well-tolerated way to treat generalized severe epidermolysis bullosa simplex (EBS), according to a study involving two boys treated for four weeks. In vitro data suggested that the diacerein ointment was retained in the skin for a prolonged period of time. The study,…

Treatment with intravenous immunoglobulin (IVIG) and off-label rituximab can lead to complete remission of epidermolysis bullosa acquisita (EBA), according to a study that also illustrates the variable clinical and immunological presentations of EBA. The study, “Meta-analysis of the clinical and immunopathological characteristics and treatment outcomes in epidermolysis bullosa…

RegeneRx Biopharmaceuticals announced a clinical trial update on RGN-137, an investigational therapy for the treatment of epidermolysis bullosa (EB). The update was provided by GtreeBNT, RegeneRx’s partner for the development of RGN-137 in the United States. RGN-137 is a thymosin βeta-4 (Tβ4)-based dermal gel that being developed…

With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Creditand affordable health insurance to future funding for rare disease research. Yet “whether Democrats take over the House or Senate, or…

Fibrocell Science has completed a type C meeting with the U.S. Food and Drug Administration in which they discussed the design of a Phase 3 clinical trial to assess the efficacy of FCX-007, a gene therapy candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). During the…

The first participant has been enrolled in a Phase 2 clinical trial testing AC-203 for the treatment of inherited epidermolysis bullosa (EB), the trial’s sponsor, TWi Biotechnology, announced. AC-203 (also known as CCP-020) is a proprietary topical formulation of 1% diacerein, a compound able to inhibit factors that contribute to…

A technique used to decipher the DNA sequence, called next-generation sequencing (NGS), was successfully used to identify two new mutations in a patient diagnosed with both simplex and junctional epidermolysis bullosa (EB). The findings were reported in the study “Next generation sequencing identifies double homozygous mutations in two…

The first two adults with recessive dystrophic epidermolysis bullosa (RDEB) treated with the topical gene therapy candidate KB103 experienced increased levels of functional human collagen 7 (COL7) protein and anchoring collagen fibril formation, a Phase 1/2 clinical trial shows. Moreover, wounds treated with topical KB103 closed in two weeks and…

Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based Retrophin. As founder and CEO of another company (then known as Turing Pharmaceuticals), in late 2015 Shkreli bought the rights…

A new gene-editing approach can repair a mutation responsible for recessive dystrophic epidermolysis bullosa (RDEB) in a patient’s tissue. A full description of the technique is in the study “Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair,” recently published in the…