SIRNAX, Inc. — Department of Health and Human Services STTR Phase I: This goal of this project is to develop an innovative process for treating patients with W

Description

This goal of this project is to develop an innovative process for treating patients with Waldenstr m's macroglobulinemia by suppressing the production of IgM. Waldenstr m's macroglobulinemia is incurable and fatal. The manifestations of this disease that are due to high concentrations of monoclonal IgM are hyperviscosity with headache, fatigue, impaired cognition, confusion, and stroke, peripheral neuropathy and systemic amyloidosis which may result in death. We hypothesize that the production of IgM by malignant plasma cells can be stopped by using RNA interference which can be administered systemically and specifically targets the plasma cells which produce IgM. This will be accomplished by using an innovative recombinant fusion protein that targets the malignant plasma cells by its antigen binding portion and delivers the RNA interference into the plasma cells by natural internalization. The fusion protein consists of an antibody fragment for cell specific targeting and a modified version of human protami