Immusoft of CA, a wholly owned subsidiary of Immusoft Corporation (“Immusoft”), a clinical-stage cell therapy company dedicated to improving the lives of patients with rare diseases, today announced the first patient dosed with engineered B cell therapy ISP-001 in a Phase 1 trial in MPS I, at M Health Fairview University of Minnesota Medical Center. MPS I is a rare, genetic disease that affects the body’s ability to produce the enzyme alpha-L-iduronidase (IDUA), which is an essential enzyme that helps to break down long-chain sugars inside cells. Without the IDUA enzyme these sugars accumulate in the body, affecting the eyes, heart, bones, etc.

The patient was dosed without the need for a preconditioning regimen (required for gene modified stem cells) or immunosuppression (required for systemic virus-delivered gene therapy). Preconditioning, consisting of myeloablative chemotherapy, can cause hair loss, infertility, severe nausea, the requirement for an extended hospital stay, and may lead to life-threatening infections. Immunosuppression is also associated with substantial morbidity, and can increase the risk of infection. Neither chemotherapy nor immunosuppression is required by […]