Bioblast Pharma Ltd (BioBlast) is an Israel-based development-stage biopharmaceutical company. The Company is focused on the identification, licensing, acquisition, development and commercialization of drugs for rare and ultra-rare genetic and metabolic diseases. It focuses on therapeutic platforms that offer solutions for several diseases that share a common pathophysiological mechanism, which are the functional changes that accompany a particular syndrome or disease. It also focuses on diseases with severe and debilitating manifestations. The Company has several drug candidates in clinical or preclinical development targeting neuromuscular/central nervous system (CNS) and mitochondrial diseases. Its drug candidates include Trehalose IV Solution, BB-FA (Bioblast-Friedreich's ataxia) and BBrm-02. The Company's product candidate platforms include Protein Stabilizing Platform, Read-through Platform and Mitochondrial Protein Replacement Platform.