Complement Alternative Pathway is activated in a variety of orphan diseases including paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), C3 Glomerulopathy (C3G), Wet and Dry age related macular degeneration (AMD), Uveitis, and neuromyelitis optica (NMO) to name a few. Our literature searches revealed that there are in total of more than 100 orphan/non orphan, acute/chronic clinical conditions where complement system is activated. Our intellectual property, trade secrets, and know-hows cover all such diseases. With time, we plan to target the diseases where our lead candidates would show therapeutic efficacy.
Our laboratory model systems have proven that our clinical candidates effectively prevent formation and deposition of C3b and C5b-9 in both in vitro and ex vivo disease models of PNH, the two most important complement activation products. Our current therapeutic antibody landscape is pretty unique and is therefore tailored to treat orphan diseases of our time that need new strategies. Our drug candidates do not interfere with the complement classical pathway (CCP) which is important to be kept unaffected, a very critical approach to prevent infections during disease treatment.