Immusoft to Present Data at ASGCT 2025, Demonstrating Positive Results from the World's First Engineered B Cell in a Human Clinical Trial

Safety and initial activity of autologous human B cells engineered to express the enzyme iduronidase.

Results from a first-in-human clinical trial in subjects with MPS I

SAN FRANCISCO, May 13, 2025 /PRNewswire/ -- Immusoft of CA, a cell therapy company dedicated to improving the lives of patients with diseases modifiable with therapeutic proteins, today announced that Paul J. Orchard, MD., Professor, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota Medical School, will present recent clinical data at the American Society of Cell and Gene Therapy (ASGCT) 28th Annual Meeting, taking place May 13-17, 2025, in New Orleans, LA. 

Immusoft will present the following:

Oral Abstract Session: Gene Therapy Clinical Trials

Title: 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)

Date and Location: Wednesday, May 14^th, 1:30 PM—3:15 PM CT, New Orleans Theater B.

Presenter: Paul J. Orchard, MD., Professor, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, University of Minnesota Medical School

Immusoft is developing B cells as biofactories for therapeutic protein delivery. This new modality has the potential to be both durable and redosable. The Company's lead drug candidate, ISP-001, is a first-in-class investigational treatment for mucopolysaccharidosis type I (MPS I), a rare lysosomal storage disease. ISP-001 is an autologous B cell product engineered to express human alpha-L-iduronidase (IDUA) for treating MPS I.

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.

There is a significant unmet need for new therapies with improved efficacy and convenience in MPS I, where the current standard of care is enzyme replacement therapy (ERT) or hematopoietic stem cell therapy transplant (HSCT). Due to their capacity for high-level protein production, their natural ability to engraft in the bone marrow without the need for toxic preconditioning, and their ability to manufacture a multitude of protein types, B cells have the potential to address unmet needs across numerous therapeutic indications.

Additional information regarding the ASGCT presentation can be found at ASGCT.

About Immusoft

Immusoft is a cell therapy company focused on developing novel therapies for rare diseases using a sustained delivery of protein therapeutics from a patient's cells. The company is developing a technology platform called Immune System Programming (ISP™), which modifies a patient's B cells and instructs the cells to produce gene-encoded medicines. The B cells that are reprogrammed using ISP become miniature drug factories that are expected to survive in patients for many years. The company is based in Seattle, WA. For more information, visit www.immusoft.com.

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SOURCE Immusoft