Prospectively designed for scalability and re-dosability, Immusoft’s engineered B cells represent the next generation of advanced therapeutics
San Francisco, CA., April 8, 2026 — Immusoft of CA, a clinical-stage biotechnology company pioneering engineered B cell therapies, today announced that it will present an invited clinical case review of the world’s first engineered B cell therapy administered to a patient. The presentation will be delivered at the U.S. Food and Drug Administration (FDA) public workshop, “Advancing Pediatric Cell and Gene Therapy Clinical Trials,” on April 9, 2026, from 11:00 a.m.-1:15 p.m. ET. The workshop is co-hosted by the Alliance for Regenerative Medicine (ARM).
The case review will cover the initial clinical experience of a scientific first: the first patient to be treated with an engineered B cell therapy. The presentation will include observations related to safety, feasibility, and early pharmacodynamic activity of ISP-001, the Company’s lead investigational therapy for the treatment of mucopolysaccharidosis type I (MPS I).
Early clinical data from Immusoft’s ongoing Phase 1/2 trial have demonstrated a favorable safety and tolerability profile to date for ISP-001, including the ability to safely re-dose a patient, a watershed moment for the cell and gene therapy field. The first re-dosed patient continues to show encouraging pharmacodynamic, functional, and quality-of-life outcomes. A second patient, dosed early last year at approximately three times the initial dose given to the first patient, has also shown favorable safety and tolerability, with encouraging early pharmacodynamic and functional improvements.
“We are pleased to share this clinical case, which represents an important milestone for the field of engineered cell therapies,” said Robert Sikorski, M.D., Ph.D., Chief Medical Officer of Immusoft. “While still early, these findings demonstrate signs of functional improvement and reductions in pain, providing initial evidence supporting the potential to enable sustained in vivo protein expression via engineered B cells. We appreciate the opportunity to engage with FDA and the broader community as this new therapeutic modality continues to evolve.”
The trial is supported by up to $23 million in funding from the California Institute for Regenerative Medicine (CIRM), which funds cell and gene therapy research and clinical trials in California.
About MPS I (Mucopolysaccharidosis type I)
MPS I (Mucopolysaccharidosis type I) is a rare, lethal childhood genetic disease that currently has no cure. It affects the body’s ability to produce IDUA (alpha-L-iduronidase), an essential enzyme in the breakdown of sugar molecules in cells called glycosaminoglycans (GAGs). When GAGs can’t be broken down, they accumulate in cells throughout the body, causing organ damage and leading to early death. MPS I occurs in approximately 1 in 100,000 live births, with symptoms appearing within the first year of life for the most severe form, Hurler Syndrome. Children affected with the Hurler form of MPS I rarely live more than ten years after diagnosis, if left untreated.
About ISP-001
ISP-001 is the first ever clinical-stage engineered B cell therapy that deploys a patient’s own cells to function as “living biofactories”, which continuously produce and secrete potentially therapeutic proteins. ISP-001 is specifically designed to secrete therapeutic levels of α-L-iduronidase (IDUA), the enzyme deficient in MPS I patients. By leveraging the natural biology of B cells, ISP-001 aims to overcome key challenges associated with current therapies, including the “sawtooth effect” (spike and trough enzyme levels) associated with frequent enzyme replacement therapy infusions, as well as mortality and safety concerns associated with stem cell transplants.
ISP-001 has received Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the U.S. Food and Drug Administration and is currently being evaluated in a Phase 1 clinical trial. For more information about the trial, visit clinicaltrials.gov (NCT05682144).
About Immusoft
Immusoft of CA is a wholly owned subsidiary of Immusoft Corporation. Immusoft is a clinical-stage company focused on developing novel therapies for rare diseases using sustained delivery of protein therapeutics from a patient’s own cells. The company has developed 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 reprogrammed with ISP become miniature protein-therapeutic biofactories that are expected to persist for many years. For more information, visit www.immusoft.com and follow us on LinkedIn.
Media ContactAdam SilversteinSCIENTadam@scientpr.com
Funding will enable the Company to support expanded development of its lead asset, ISP-001, for the treatment of mucopolysaccharidosis type 1 San Francisco, CA., March 30, 2026 — Immusoft of CA, a clinical-stage biotechnology company pioneering engineered B cell therapies, announced today that the California…
