The California Institute for Regenerative Medicine awarded an $8 million grant to Immusoft of CA to support an engineered B cell clinical program for the treatment of MPS I, a rare childhood genetic disease.
Photo: Sean Ainsworth, CEO of Immusoft
MPS I (Mucopolysaccharidosis type I) is a rare, lethal childhood genetic disease that 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. In its most severe form, children affected rarely live longer than ten years after diagnosis. 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.
The funding will support a phase I study to evaluate the safety and tolerability of ISP-001 (for delivery of alpha-L-iduronidase, or IDUA) in MPS I. ISP-001 will be the first engineered B cell therapy to enter into human clinical trials. Immusoft has pioneered the engineering of B cells to create biofactories for in vivo therapeutic protein delivery. The company’s novel B cell platform was designed to circumvent immunogenicity associated with virus-delivered gene therapy and chemotherapy preconditioning associated with stem cell-mediated gene therapy, while enabling durable therapeutic delivery and the possibility to re-dose. Immunosoft says this platform has the potential to be used for rare diseases and other conditions modifiable with protein therapeutics.
MPS I affects the body’s ability to produce an essential enzyme, IDUA, resulting in progressive damage to tissues and organs. Children with MPS I require frequent infusions, which impacts their quality of life. The current standard of care for MPS I, depending on severity of disease, is hematopoietic stem cell transplantation (HSCT) or enzyme replacement therapy (ERT), which requires weekly infusions (lasting 3-4 hours) throughout the patient’s lifetime. There is a significant unmet need for new therapies with improved efficacy and convenience.
Immusoft’s lead therapeutic, ISP-001, is a novel platform that uses a cell therapy to deliver a gene-encoded medicine. The platform leverages the B cell’s natural ability to produce high levels of antibody proteins. It is the first engineered B cell therapy to enter into human clinical trials. With ISP-001, patient’s B cells are programmed to constantly produce therapeutic proteins, potentially mitigating the need for frequent infusions while potentially improving patient outcomes. This is because ISP-001 cells are expected to release IDUA at therapeutic levels around the clock and on an extended basis – potentially for years.
“ISP-001 has shown promising results in preclinical studies and we have begun screening patients in our phase I clinical trial to evaluate its safety and tolerability in treating patients with MPS I. If successful, this novel candidate has the potential to address a significant unmet need in patients who suffer from MPS I and could become a new treatment option to help slow the progression of the disease and improve the quality of life for affected individuals,” said Sean Ainsworth, CEO of Immusoft.
In addition to the support for its MPS I clinical trial, in November 2021, Immusoft received a $4M CIRM grant to support the development of its ISP-002 (for delivery of iduronate sulfatase) program for MPS II. Immusoft has also entered into a research collaboration and license option agreement with Takeda Pharmaceutical Company Limited aimed at delivering protein therapeutics across the blood-brain barrier to treat neurometabolic disorders.
Immusoft of CA is a cell therapy focused and wholly owned subsidiary of Immusoft Corporation.
Author: Rare Daily Staff
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