About
Bio Blast Pharma Ltd., a development stage biopharmaceutical company, focuses on the identification, licensing, acquisition, development, and commercialization of drugs for rare and ultra-rare genetic and metabolic diseases. The company develops Cabaletta, which is in Phase II/III clinical trials for the treatment of oculopharyngeal muscular dystrophy; and Phase II clinical trials for the treatment of spinocerebellar ataxia type 3, as well as in preclinical studies for the treatment of spinobulbar muscular atrophy. Its preclinical development stage product candidates comprise BBrm1 for spinal muscular atrophy; BB-FA for the treatment of friedrichs ataxia; and BB-OTC for ornithine transcarbamylase deficiency. Bio Blast Pharma Ltd. was founded in 2012 and is headquartered in Tel Aviv, Israel.