Immusoft Awarded $15 Million in Funding from the California Institute for Regenerative Medicine
Funding will enable the Company to support expanded development of its lead asset, ISP-001, for the treatment of mucopolysaccharidosis type 1
SAN FRANCISCO, March 30, 2026 (GLOBE NEWSWIRE) -- Immusoft of CA, a clinical-stage biotechnology company pioneering engineered B cell therapies, announced today that the California Institute for Regenerative Medicine (CIRM) has awarded the company a $15 million grant to support the expanded clinical development of ISP-001, its lead investigational therapy for the treatment of mucopolysaccharidosis type I (MPS I), a rare and life-threatening lysosomal storage disorder.
“There is no cure for MPS I, and there is a significant unmet need for therapies that can deliver meaningful functional improvements for patients,” said Sean Ainsworth, Chief Executive Officer of Immusoft. “ISP-001 has shown early signs of functional improvement and reductions in pain, highlighting its potential for long-term, continuous therapeutic protein delivery that may avoid many of the limitations of other approaches being developed. We are grateful for our ongoing partnership with CIRM and look forward to advancing this candidate, including into pediatric patients.”
Early clinical data from Immusoft's ongoing Phase 1/2 trial have demonstrated a favorable safety and tolerability profile 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 positive 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.
“Advancing innovative, high-impact therapies for patients with serious diseases such as MPS I is central to CIRM’s mission,” said Jonathan Thomas, PhD, JD, President and Chief Executive Officer of CIRM. “This trial brings hope to patients with MPS I who otherwise have no effective therapies. This new hope and promise of future therapies is why the people of California created CIRM.”
CIRM’s CLIN2 funding program supports clinical trials for regenerative medicine therapies that address significant unmet medical needs and have the potential to provide transformative benefits to patients, their families, and the healthcare system.
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.
About CIRM
The California Institute for Regenerative Medicine (CIRM) is a state agency created by California voters to accelerate stem cell and gene therapies for people with unmet medical needs. Since 2004, Californians have entrusted CIRM with $8.5 billion to accelerate promising discoveries through clinical trials, train a regenerative medicine workforce, strengthen the state’s biotechnology economy, and expand access to transformative treatments. Today, CIRM is pioneering new models of therapy development and accelerating medical breakthroughs that change lives — in California and around the world. For more information, visit www.cirm.ca.gov.
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