Alpha-L-iduronidase gene therapy(Christie Hospital Nhs Foundation Trust)

SAN FRANCISCO--( BUSINESS WIRE )-- 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 or genetic diseases, announced today that the California Institute for Regenerative Medicine (CIRM) has awarded the company an $8M grant. This 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 a rare, childhood genetic disease. The Investigational New Drug Application for the study was cleared by the U.S. Food and Drug Administration as previously announced by Immusoft on September 1, 2022. This clinical study is both significant for Immusoft and historic in the field of cell and gene therapies, as 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, leading the field with over 60 issued and pending patents. 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. 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. “We are delighted to partner with CIRM for a second program, this time for MPS I, and appreciate their recognition of the importance of this program. 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, Immusoft’s Chief Executive Officer. “Cell-based gene therapy for MPS I such as ISP-001 has the potential to offer long-term disease control and prevent debilitating complications,” said Robert Hayes, Immusoft’s Chief Scientific Officer. “This novel approach also holds the potential to provide patients with durable delivery of the therapeutic 24/7. We are honored that CIRM has recognized the potential of ISP-001 and we look forward to advancing our candidate in MPS I, a rare, genetic childhood disease where patients currently 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 for 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 work safely in patients.” 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. About MPS I (Mucopolysaccharidosis type I) 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. About Immusoft Immusoft of CA is a wholly owned subsidiary of Immusoft Corporation. Immusoft Corporation 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 (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 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 . FORWARD-LOOKING STATEMENTS Statements in this press release that are not strictly historical are forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995. Forward-looking statements are based on management’s current plans, estimates and projections. The beliefs, assumptions and expectations on which these statements are based can change as a result of many possible events or factors, not all of which are known to Immusoft Corporation or are within its control. Immusoft Corporation undertakes no obligation to update any forward-looking statement in light of new information or future events. Actual results or outcomes may differ from those implied by the forward-looking statements as a result of a number of operational, scientific, regulatory and related risks and uncertainties.