Monday, October 16, 2017


ORPN Bio Blast Pharma Ltd. gains 35% Oct 16, 2017

Bio Blast Pharma Ltd., a clinical development-stage biopharmaceutical company, focuses on the identification, licensing, acquisition, development, and commercialization clinically meaningful therapies for patients with rare and ultra-rare genetic diseases. The company's drug candidates under clinical or preclinical development includes Trehalose 90mg/mL IV solution, a mutant protein stabilizer and autophagy enhancer used for protein-based drugs that are administered intravenously to treat patients with oculopharyngeal muscular dystrophy and spinocerebellar ataxia type 3. Its drug candidates under clinical or preclinical development also comprise BB-FA, a mitochondrial protein replacement platform to treat various diseases caused by mutations in genes associated with mitochondrial-related proteins, such as Friedreich's ataxia; and BBrm-02, an azithromycin in a formulation suitable for intrathecal delivery intended to treat spinal muscular atrophy, and other neurodegenerative and neurodevelopmental diseases. Bio Blast Pharma Ltd. was founded in 2012 and is headquartered in New Haven, Connecticut.

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