Bio Blast Pharma Ltd. (BioBlast) is a 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. The Company is advancing its Cabaletta platform product candidates through clinical development and advancing its other product candidates, BB-FA, BB-OTC, and BBrm1, as well as its other early stage research projects, through preclinical development. The Company’s three principal product candidate platforms include Protein Stabilizing Platform, Read-through Platform and Mitochondrial Protein Replacement Platform. The Company is developing Cabaletta for the treatment of Oculopharyngeal Muscular Dystrophy (OPMD); spinocerebellar ataxia type 3 (SCA3), and Spinobulbar Muscular Atrophy (SBMA). It is conducting preclinical studies of BBrm1 for the treatment of Spinal Muscular Atrophy (SMA).
