C2 siRNA Therapy
We are developing a novel and efficient method to treat MG by using siRNA targeting the C2 gene involved in classical complement pathway, thus preserving the alternative complement pathway (factor B, C3 and C5-9) to protect against microbial infection. The proof of concept study has been completed in the mouse model of MG. This technology will have a major impact in treating complement-mediated autoimmune and inflammatory diseases and acute transplant rejection in a specific manner with C2 siRNA with limited side effects. Our first product will be human C2 siRNA developed for the treatment of MG. Following a C2 siRNA delivery and toxicology study, we plan to submit an application to the FDA for IND approval. Next we will target other complement mediated diseases and acute transplant rejection.
Huda R, Tüzün E, Christadoss P. Complement C2 siRNA mediated therapy of myasthenia gravis in mice. J .Autoimmunity. 2013, 42: 94-104 PMID: 23410585 View PDF.