BioBlast Receives Notice Of Allowance From USPTO For Its Novel Fusion Protein-Based Platform For The Treatment Of Friedrich's Ataxia

BioBlast Pharma Ltd, ORPN a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases, announced today it has received a notice of allowance from the United States Patent and Trademark Office (USPTO) on a composition-of-matter patent for its novel fusion protein for Friedrich's Ataxia (referred to as BB-FA) that is built on its mitochondrial protein replacement (mPRT) platform. The Company's breakthrough platform technology has produced drug candidates that have demonstrated efficacy in several mitochondrial protein deficiencies in cell and animal models. "BB-FA for Friedrich's Ataxia is our most advanced mPRT preclinical program. We have developed this platform based on a recombinant protein containing targeting signals," stated Dalia Megiddo, MD, MBA Chief Executive Officer of BioBlast. "We are pleased to have received this notice of allowance which covers the significant innovations we have made in the delivery of therapeutic proteins into the mitochondria. We look forward to making further progress toward providing a therapy to help Friedrich's Ataxia patients in need of treatment." Friedrich's Ataxia is an inherited disorder characterized by progressive deterioration of the muscular and nervous system that begins in the first or second decade of life and results in gait disturbance (ataxia), cognitive impairment, progressive heart disease and diabetes. According to Friedrich's Ataxia Research Alliance (FARA), about 1:50,000 people in the U.S. suffer from Friedrich's Ataxia. Most patients are wheelchair-bound within 15 years of diagnosis. Most patients do not survive beyond the fourth or fifth decade of life.