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news.Bio Blast Pharma 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 Patent Issuance for patent no. 8,912,147 from the US Patent and Trademark Office (USPTO) for 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.
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"This patent covers the significant innovations we have made in the delivery of therapeutic proteins into mitochondria," said Dalia Megiddo, MD, Chief Executive Officer of BioBlast.
"We are pleased to have added to our patent and patent applications estate and 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.