SAN FRANCISCO–(BUSINESS WIRE)– Immusoft of CA, a wholly owned subsidiary of Immusoft Corporation, a cell therapy company dedicated to improving the lives of patients with rare diseases, announced today that the California Institute for Regenerative Medicine (CIRM) has awarded the company a $4M grant to support the development of its ISP- 002 (for delivery of iduronate sulfatase) program in mucopolysaccharidosis type II (MPS II), an inherited disease for which patients have limited options.
MPS II is a rare genetic disease in children, which causes abnormalities across many organs, including the heart and lungs, and the musculoskeletal system. If untreated, individuals with severe MPS II will not live past their teen years. Currently available treatments for MPS II do not sufficiently provide therapeutic agents at stable levels, resulting in considerable residual burden of disease.
Immusoft’s therapeutic is specially tailored to combine a cell therapy with a gene-encoded medicine. In the case of ISP- 002, the patient’s own B cells are programmed to express the therapeutic enzyme iduronate sulfatase (IDS). In healthy individuals, IDS contributes to the breakdown of glycosaminoglycans (GAGs), which are harmful at high levels. Once clinical testing is initiated for ISP-002, the cells will be delivered back to the patient where they are expected to release IDS at therapeutic levels, around the clock – potentially for years.
“ISP-002 has the potential to address a significant unmet need in children who suffer from mucopolysaccharidosis type II (MPS II). Preclinical data suggests ISP-002 can meaningfully reduce accumulated glycosaminoglycans in various tissues, and our approach also holds the potential to provide patients with durable delivery of the therapeutic continuously,” said Sean Ainsworth, Chief Executive Officer, Immusoft. “We are honored that CIRM has recognized the potential value of ISP- 002 and we look forward to advancing our candidate into clinical trials in this rare genetic disease where patients have limited options.”
“Our goal is to always move the most promising research forward as fast as we can,” said Dr. Maria Millan, President and Chief Executive Officer, California Institute of Regenerative Medicine (CIRM). “That’s why these programs are so important. They reflect potential therapeutic approaches that have shown promise in the lab and are ready to take the next step, to undergo further testing and examination to see if they might be able to work in patients.”
Immusoft is developing a new modality of gene delivery that holds the promise of being both durable and re-dosable. There is significant potential across multiple high-value disease states to replace suboptimal standard of care, beginning with Hurler Syndrome (MPS I). Immusoft’s lead candidate ISP-001 is expected to enter Phase I clinical trials for MPS I in 2022. The current standard of care for MPS I is enzyme replacement therapy (ERT), and there is a significant unmet need for new therapies with improved efficacy and convenience.
About Immusoft
Immusoft of CA is a wholly owned subsidiary of Immusoft Corporation. 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 (ISPTM), 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.
About CIRM
At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission.
To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies.
With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is the world’s largest institution dedicated to helping people by bringing the future of cellular medicine closer to reality.
For more information go to www.cirm.ca.gov
Contacts
KKH AdvisorsKimberly Ha kimberly.ha@kkhadvisors.com 917-291-5744
Source: Immusoft Corporation
First-in-human clinical trial of genetically engineered B cells: Application to the treatment of mucopolysaccharidosis type I
