- Immusoft announces historic first patient dosed with an engineered B cell investigational therapy ISP-001 in Phase 1 trial for patients with MPS I (Mucopolysaccharidosis type I)
- Received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for ISP-001 in MPS I
SAN FRANCISCO–(BUSINESS WIRE)–#Bcells–Immusoft of CA, a wholly owned subsidiary of Immusoft Corporation (“Immusoft”), a clinical-stage cell therapy company dedicated to improving the lives of patients with rare diseases, today announced the first patient dosed with engineered B cell therapy ISP-001 in a Phase 1 trial in MPS I, at M Health Fairview University of Minnesota Medical Center. MPS I is a rare, genetic disease that affects the body’s ability to produce the enzyme alpha-L-iduronidase (IDUA), which is an essential enzyme that helps to break down long-chain sugars inside cells. Without the IDUA enzyme these sugars accumulate in the body, affecting the eyes, heart, bones, etc.
The patient was dosed without the need for a preconditioning regimen (required for gene modified stem cells) or immunosuppression (required for 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. This supports the concept of redosability of engineered B cells.
Immusoft has pioneered B cell engineering to create biofactories for durable in vivo therapeutic protein delivery, leading the field with over 60 issued and pending patents. Immusoft’s Immune System Programming (ISP)™ approach is designed to enable reprogramming of a patient’s B cells for constant production of therapeutic proteins, mitigating the need for frequent enzyme administration and the potential to improve patient outcomes. ISP-001 represents the first product candidate developed using the company’s proprietary platform. The company has also received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for ISP-001 in MPS I.
“This achievement marks an important milestone for patients with MPS I who have a significant need for additional therapeutic options,” said Sean Ainsworth, CEO, Immusoft. “This is the first patient in the world to receive an engineered B cell therapy – a major accomplishment for Immusoft and a major advancement in cell and gene therapy.”
“Existing interventions for MPS I are not curative and do not adequately address many significant disease-related complications,” said Paul Orchard, M.D., principal investigator in the ISP-001 Phase 1 clinical trial, a Professor in the Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy at University of Minnesota Medical School, and a Pediatric Blood and Marrow Transplant Physician with M Health Fairview. “A non-viral methodology for engineering cells, such as ISP-001 that does not require myeloablative conditioning, could be of tremendous advantage for patients with rare disorders. There is a clear unmet need in providing safer and more effective therapeutic modalities for these patients.”
“We’re excited to support Immusoft’s program, as our goal is to always move the most promising research forward as fast as we can,” said Dr. Abla Creasey, Vice President of Therapeutics Development, California Institute of Regenerative Medicine (CIRM). “Immusoft is advancing the world’s first engineered B cell therapy, which may have significant impact for patients with this rare disease and potentially many others. We look forward to supporting the Company in bringing this life-changing potential to patients with MPS I.”
“I look forward to the next steps with this pioneering team of scientists and investigators as we advance the initial clinical trial in MPS I. We are excited by the potential of expanding the Immusoft engineered B cell platform to trials in additional therapeutic areas,” said Robert Sikorski, M.D., Ph.D., Chief Medical Officer, Immusoft.
About MPS I (Mucopolysaccharidosis type I)
MPS I is a rare, lethal genetic disease that in its severe form is lethal in the first decade of life. It affects the body’s ability to produce IDUA (alpha-L-iduronidase), which is an essential enzyme that helps to break down long-chain sugars inside cells. When the sugar chains cannot be broken down and disposed of, they accumulate in the cells and cause progressive damage. This accumulation can happen in the tissues, including the brain. Severe MPS I occurs in about 1 in 100,000 births, and symptoms appear within a child’s first year of life. In what is referred to as attenuated MPS I, symptoms appear later in childhood. It occurs in about 1 in 500,000 births.
About Immusoft
Immusoft of CA is a wholly owned subsidiary of Immusoft Corporation. Immusoft is a clinical stage 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 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 that are reprogrammed using ISP become miniature protein therapeutic biofactories that are expected to persist for many years. For more information, visit www.immusoft.com.
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Kimberly Ha
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