TEL AVIV, Israel, Aug. 18, 2014 (GLOBE NEWSWIRE) -- BioBlast Pharma Ltd, (Nasdaq: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.
About BioBlast
BioBlast Pharma is a clinical-stage biotechnology company committed to developing clinically meaningful therapies for patients with rare and ultra-rare genetic diseases. Founded in 2012, the company is rapidly building a diverse portfolio of product candidates with the potential to address unmet medical needs for incurable diseases. The BioBlast platforms are based on deep understanding of the disease-causing biological processes, and potentially offer solutions for several diseases that share the same biological pathology.
Forward-Looking Statements
This press release may contain forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995 and other Federal securities laws. For example, we are using forward looking statements when we discuss making further progress toward providing a therapy to help Friedrich's Ataxia patients, or that our platforms potentially offer solutions for several diseases that share the same biological pathology. Because such statements deal with future events and are based on BioBlast Pharma Ltd.'s current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of BioBlast Pharma could differ materially from those described in or implied by the statements in this press release, including those discussed under the heading "Risk Factors" in BioBlast Pharma's registration statement on Form F-1 filed with the Securities and Exchange Commission ("SEC") and in any subsequent filings with the SEC. Except as otherwise required by law, BioBlast Pharma disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.