BioBlast Pharma Ltd (NasdaqGM: ORPN), a clinical-stage, orphan disease-focused biotechnology company, announced today it has received a notice of allowance from the United States Patent and Trademark Office (USPTO) on a composition and method for the treatment of oculopharyngeal muscular dystrophy (OPMD) with Cabaletta. The Company has demonstrated efficacy in preclinical cell and animal models with Cabaletta, its lead therapeutic program and it is currently the subject of human clinical trials pursuing two indications.

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BioBlast Pharma Ltd. (NasdaqGM: ORPN), a clinical-stage biotechnology company committed to developing meaningful therapies for patients with rare and ultra-rare genetic diseases, announced key senior leadership changes designed to support the Company’s global clinical efforts, operational scale-up, and pre-commercialization activities.

Colin Foster, a 25-year veteran of the life sciences industry has joined BioBlast as its President and Chief Executive Officer and a member of its Board of Directors, effective immediately. Dalia Megiddo, MD, co-founder and former Chief Executive Officer, has been named to the newly-created position of Chief Development Officer and will remain a member of the Board of Directors.

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TEL AVIV, Israel, Jan. 8, 2015 (GLOBE NEWSWIRE) — BioBlast Pharma Ltd. (ORPN), a clinical-stage biotechnology company committed to developing meaningful therapies for patients with rare and ultra-rare genetic diseases, announced today preliminary findings from its HOPEMD Phase 2/3 study investigating Cabaletta (trehalose) in patients with Oculopharyngeal Muscular Dystrophy (OPMD).

The multicenter study is testing the safety and tolerability of Cabaletta in patients suffering from OPMD – a progressive, debilitating genetic disease – across three centers in Israel, Canada and the U.S.