Immusoft's pioneering cell therapy for mucopolysaccharidosis type I (MPS I) has demonstrated positive safety and initial activity in the world's first clinical trial using engineered B cells as therapeutic protein factories. The company announced today that detailed results will be presented at the American Society of Cell and Gene Therapy (ASGCT) 28th Annual Meeting in New Orleans next week.
The first-in-human clinical trial evaluated ISP-001, an autologous B cell product engineered to express human alpha-L-iduronidase (IDUA), the enzyme deficient in patients with MPS I. Notably, the therapy was administered without requiring preconditioning regimens or immunosuppression—procedures typically necessary for gene-modified stem cells or systemic virus-delivered gene therapies that can cause significant toxicities.
Dr. Paul J. Orchard, Professor in the Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy at the University of Minnesota Medical School, will present the clinical data on Wednesday, May 14th, during the Gene Therapy Clinical Trials session at the ASGCT meeting.
Novel B Cell Platform for Protein Delivery
Immusoft's Immune System Programming (ISP™) technology represents a potentially transformative approach to treating genetic disorders. The platform modifies a patient's own B cells to produce therapeutic proteins continuously, creating what the company describes as "miniature drug factories" that may survive in patients for many years.
"B cells have unique characteristics that make them excellent candidates for therapeutic protein delivery," explained a company spokesperson. "They naturally produce high levels of proteins, can engraft in bone marrow without toxic preconditioning, and can manufacture various protein types."
These properties could address significant limitations of current treatment options for MPS I and potentially other rare diseases requiring enzyme or protein replacement.
Addressing Unmet Needs in MPS I Treatment
MPS I is a rare lysosomal storage disease caused by mutations in the gene encoding IDUA, resulting in the accumulation of glycosaminoglycans throughout the body. This accumulation leads to progressive multi-organ dysfunction, including skeletal abnormalities, cardiac disease, respiratory complications, and in severe cases, neurological impairment.
Current standard treatments include enzyme replacement therapy (ERT), which requires frequent infusions, and hematopoietic stem cell transplantation (HSCT), which carries significant risks and complications. Both approaches have limitations in efficacy and convenience for patients.
"There is a significant unmet need for new therapies with improved efficacy and convenience in MPS I," noted Immusoft in their announcement. The company believes their B cell therapy approach could provide a more durable solution with potentially fewer side effects and treatment burdens.
Clinical Trial Design and Future Directions
The clinical trial evaluated the safety and initial activity of ISP-001 in subjects with MPS I. While complete results will be presented at the ASGCT meeting, the announcement indicates positive preliminary findings that support further development of this novel therapeutic approach.
The oral presentation, titled "Autologous human peripheral blood B cells genetically engineered to express human iduronidase: Results from a first-in-human clinical trial in subjects with mucopolysaccharidosis type I (MPS I)," will provide detailed information on the trial design, patient outcomes, and future development plans.
If successful in larger trials, Immusoft's B cell platform could potentially be applied to numerous other conditions requiring therapeutic protein delivery, expanding treatment options for patients with rare genetic disorders.
About Immusoft
Immusoft is a cell therapy company based in Seattle, Washington, focused on developing novel therapies for rare diseases using sustained delivery of protein therapeutics from a patient's own cells. The company's proprietary Immune System Programming (ISP™) technology platform modifies B cells to produce gene-encoded medicines.
The company's lead candidate, ISP-001 for MPS I, represents their first clinical application of this technology. Immusoft continues to explore additional applications of their B cell platform across multiple therapeutic areas with high unmet medical needs.
