Immusoft of CA, a wholly owned subsidiary of Immusoft Corporation, a cell therapy company dedicated to improving the lives of patients with rare diseases, announced today that the California Institute for Regenerative Medicine (CIRM) has awarded the company a $4M grant to support the development of its ISP- 002 (for delivery of iduronate sulfatase) program in mucopolysaccharidosis type II (MPS II), an inherited disease for which patients have limited options.
MPS II is a rare genetic disease in children, which causes abnormalities across many organs, including the heart and lungs, and the musculoskeletal system. If untreated, individuals with severe MPS II will not live past their teen years. Currently available treatments for MPS II do not sufficiently provide therapeutic agents at stable levels, resulting in considerable residual burden of disease.
Immusoft’s therapeutic is specially tailored to combine a cell therapy with a gene-encoded medicine. In the case of ISP- 002, the patient’s own B cells are programmed to express the therapeutic enzyme iduronate sulfatase (IDS). In healthy individuals, IDS contributes to the breakdown of glycosaminoglycans (GAGs), which are harmful at high levels. Once clinical testing is initiated for ISP-002, the cells will be delivered back to the patient where they are expected to release IDS at therapeutic levels, around the clock – potentially for years.
