Immusoft Corporation is set to present positive data from its first-in-human clinical trial of ISP-001, an engineered B-cell therapy for Mucopolysaccharidosis type I (MPS I), at the WORLDSymposium™ 2025 in San Diego. The data highlights the safety and initial activity of autologous human B cells genetically engineered to express human iduronidase using the Sleeping Beauty transposon system.
The oral presentation, scheduled for February 7th, will be delivered by Dr. Paul Orchard, Professor at the University of Minnesota Medical School. A poster presentation will also take place on February 5th.
Novel Approach to MPS I Treatment
MPS I is a rare lysosomal storage disease where current treatments, such as enzyme replacement therapy (ERT) and hematopoietic stem cell transplant (HSCT), have limitations. ERT requires frequent infusions, and HSCT involves a risk of severe complications. Immusoft's ISP-001 offers a novel approach by utilizing B cells as biofactories for continuous therapeutic protein delivery.
ISP-001: Engineered B Cells as Protein Factories
ISP-001 is an autologous B cell product engineered to express human alpha-L-iduronidase (IDUA), the enzyme deficient in MPS I patients. The engineered B cells are designed to engraft in the bone marrow and continuously produce IDUA, addressing the underlying cause of the disease.
Avoiding Toxic Preconditioning
A key advantage of ISP-001 is that, in this first-in-human clinical trial, the patient was dosed without the need for a preconditioning regimen (required for gene-modified stem cells) or immunosuppression (required for most systemic virus-delivered gene therapy), both of which can be associated with severe toxicities. This could significantly improve the safety profile compared to other gene therapy approaches.
Addressing Unmet Needs in MPS I
There is a significant unmet need for new therapies with improved efficacy and convenience in MPS I. Immusoft believes that B cells, with their capacity for high-level protein production and natural ability to engraft in the bone marrow, have the potential to address these unmet needs across numerous therapeutic indications.
