Bioblast Pharma Ltd (NASDAQ:ORPN) is a mover and shaker out on the Street today, with investors sparking the stock on an almost 54% upturn. Why such a bullish parade for biotech firm? Bioblast has received patent rights from European Patent Office (EPO) for its patent titled Mitochondrial Proteins Constructs and Uses.
Back in 2015, BioBlast reported positive preclinical in vitro and in vivo proof-of-concept study results for its mitochondrial protein replacement therapy drug candidate (BB-FA) for Friedreich’s Ataxia. BB-FA demonstrated efficacy in penetrating mitochondria in several patient cell lines and in two different and well established animal models of Friedreich’s Ataxia.
In in-vitro studies performed in Friedreich’s Ataxia patients’ cells, BB-FA successfully penetrated the mitochondria and restored the damaged mitochondrial functions to close to normal levels both in studies of oxidative stress and in several biochemical functions that involve critical respiratory chain metabolism in a dose-dependent and time-dependent manner.
In two different mouse models, BB-FA rescued both brain and heart tissues, returning their activity to near normal function. BB-FA induced weight gain, prevented the development of heart disease (commonly the cause of death in Friedreich’s Ataxia patients) and increased survival.
BB-FA is based on BioBlast’s innovative mitochondrial protein replacement platform, which has the potential to target more than 100 diseases that are caused by deficient or mutated proteins associated with mitochondrial metabolism.
Bioblast Pharma operates as a clinical development-stage biopharmaceutical company. It focuses on the identification, licensing, acquisition, development and commercialization of drugs for rare and ultra-rare genetic diseases. The company also focuses on diseases with severe and debilitating manifestations, where the unmet medical need is clear, the biological mechanism of action is understood, and for which there is no satisfactory treatment. Its product candidates include Protein Stabilizing, Read-through, and Mitochondrial Protein Replacement Platforms. The company was founded by Dalia Megiddo and Ehud Moshe Gilboa on January 22, 2012 and is headquartered in Tel Aviv, Israel.