• Proven clinical trial path - enzyme replacement therapy works
• Likely Orphan Drug status, fast track and clinical trial funding
• Clearly defined short-term efficacy endpoints

• MCP‐1 is key mediator of inflammatory diseases with many published preclinical studies demonstrating reduced disease activity in MCP-1 knock down in-vivo models across a wide range of applications including:

                                    • Diabetic nephropathy

                                    • In-stent stenosis

                                    • Contact hypersensitivity