January 03, 2024

Immusoft To Present on ISP-001 Clinical Trial at WORLDSymposium™ 2024 Conference

  • R. Scott McIvor, Ph.D., Chief Development Officer at Immusoft will present on Immusoft’s first-in-human clinical program
  • ISP-001 is the first ever engineered B cell dosed in a human clinical trial

SEATTLE–(BUSINESS WIRE)– Immusoft Corporation, a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced that it will present at the upcoming WORLDSymposium 2024 Conference, held in San Diego, February 4-9, 2024. WORLDSymposium is the largest international gathering of lysosomal disease researchers.

Details of the presentation are as follows:

Presentation Title: First-in-human clinical trial of genetically engineered B cells: Application to the treatment of mucopolysaccharidosis type I

Date/Time: Thursday, February 8, 2024 – 1:00 PM

Presenter: R. Scott McIvor, Ph.D., Chief Development Officer at Immusoft, and Professor of Genetics, Cell Biology and Development at the University of Minnesota.

Immusoft is developing a new modality of gene encoded medicine that holds the promise of being durable and redosable. The Company’s lead investigational 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 the treatment of 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). 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 Immusoft’s novel engineered B cell approach, which supports the concept of redosability of engineered B cells.

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). Due to their capacity for high-level protein production and their natural ability to engraft in bone marrow without need for toxic preconditioning, B cells have the potential to serve as a highly efficacious cellular therapy. Immusoft has established effective conditions for expansion and genetic engineering of B cells across multiple therapeutic indications.

Additional information regarding the WORLDSymposium presentation can be found at:

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 own 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,

Kimberly Ha
KKH Advisors