The complement system plays a central role in the body’s immune system as a protective mechanism for host defense, but its dysregulation results in serious, life-threatening complications in a broad range of human diseases including paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic-uremic syndrome (aHUS), neuromyelitis optica, and myasthenia gravis (MG), amongst others.
Complement component C5 – which is predominantly expressed in liver cells – is a genetically and clinically validated disease target. Human mutations resulting in C5 deficiencies are associated with an attenuated immune response against certain infections. Intravenous anti-C5 monoclonal antibody therapy (eculizumab) has demonstrated clinical activity and tolerability in a number of complement-mediated diseases. It is approved for the treatment PNH and aHUS in the U.S., Europe and other countries.
ALN-CC5 is in clinical development as an investigational, subcutaneously administered RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated disease. ALN-CC5 utilizes our proprietary Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology
enabling subcutaneous dosing with increased potency and durability, and a wide therapeutic index. We believe that a subcutaneously administered RNAi therapeutic that silences C5 represents a novel approach to the treatment of complement-mediated diseases.
We believe that ALN-CC5—as a first-in-class C5 synthesis inhibitor—represents an innovative, differentiated, and well-validated approach for the treatment of complement-mediated diseases. Further, we believe that a new medicine to treat excessive complement activity that could be given by infrequent, subcutaneous administration would be a welcome addition to the treatment landscape, and we look forward to the continued clinical advancement of this novel investigational therapeutic.
Please read the latest press releases and data presentations for ALN-CC5 here.