Bioblast Pharma Ltd. (NASDAQ: ORPN) was founded in 2012, headquartered in Tel Aviv, Israel, with 2 full-time employees. It is a clinical-stage biotechnology company focusing on the development of clinically meaningful therapies for patients with rare and ultra-rare genetic diseases .
Bioblast Pharma Ltd. (ORPN):
Founded in 2012, Bioblast Pharma is a listed clinical stage biotechnology company with the goal of discovering, developing and providing clinically meaningful therapies for patients with rare diseases. The company is led by a group of passionate and experienced people who work together to achieve a goal-to improve the lives of patients with rare and extremely rare genetic diseases.
The main product candidate of Bioblast Pharma is Trehalose intravenous 90mg/mL solution (Trehalose intravenous 90mg/mL solution), a protein stabilizer and autophagy enhancer for the treatment of ophthalmopharyngeal muscular dystrophy and type 3 spinocerebellar Ataxia.