Iduronicrin genleukocel-T (Immusoft)

Pharmacodynamic effects and functional improvements were observed through nine months post-dosing with a single administration of ISP-001. As of the data cutoff date of September 19, 2024, no adverse events have been reported. Data for ISP-001 for Mucopolysaccharidosis Type I to be presented on October 14, 2024 (details below). SAN FRANCISCO, Sept. 23, 2024 /PRNewswire/ -- Immusoft of CA, a wholly owned subsidiary of Immusoft Corporation ("Immusoft"), a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced positive results from the Phase 1 ISP-001 trial evaluating the therapy in a patient with mucopolysaccharidosis type I (MPS I). The clinical trial is supported by an $8 million award from the California Institute for Regenerative Medicine (CIRM), one of the world's largest institutes dedicated to regenerative medicine. "Although the Phase 1 focus is safety, we have observed activity and functional improvements, even at the low dose, far beyond our initial expectations, and are very excited by this data," said Sean Ainsworth, Immusoft Chief Executive Officer. Initial ISP-001 trial results will be presented during Immusoft's MPS I Clinical Update and Outreach webinar. Title: ISP-001: Data from the first-ever engineered B cell in a human clinical trial Date: Monday, October 14, 2024, 4:00 pm ET / 1:00 pm PT Webinar Registration Agenda: Fireside Chat: Abla Creasey, PhD, Vice President of Therapeutics Development of CIRM; Sean Ainsworth, CEO and Chairman of Immusoft Data Readout: Robert Sikorski, MD, PhD, Chief Medical Officer at Immusoft Caregiver's Perspective: Mark Dant, Executive Director of The Ryan Foundation for Rare Disease Research Panel Discussion: Moderator: R. Scott McIvor, PhD, Chief Development Officer, Immusoft; Professor, Genetics, Cell Biology and Development, University of Minnesota Panelist: Paul Orchard, MD, Principal Investigator, Professor, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy University of Minnesota Medical School Panelist: Paul Harmatz, MD, Investigator, Pediatric Gastroenterologist, UCSF Benioff Children's Hospitals Q&A Closing Remarks & Future Directions: Sean Ainsworth CEO and Chairman of Immusoft The first adult patient to receive ISP-001 in this study, with the Hurler-Scheie form of MPS I, experienced pharmacodynamic improvements, functional improvements, as well as reported improvements in quality of life, activities of daily living, and reductions in pain associated with MPS I. The administration of ISP-001 required no preconditioning regimen typically associated with other cell and gene therapeutics, and the infusion was well-tolerated. There have been no adverse events reported as of September 19, 2024. "The data from this Phase 1 trial are highly encouraging. We have observed pharmacodynamic improvements as well as functional improvements that were unanticipated in an adult patient, where some manifestations were not expected to be reversible," said Paul Orchard, MD, Principal Investigator, Professor, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy University of Minnesota Medical School. "The data to date point to tremendous potential in the treatment of MPS I, as well as numerous other applications. I am excited to follow and be a part of the progress in this current study and future developments," said Paul Harmatz, MD, Pediatric Gastroenterologist, UCSF Benioff Children's Hospitals. In the ISP-001 trial, eligible participants receive a single infusion of ISP-001, with assessments that include biomarkers of disease, functional outcomes, and patient-reported outcomes. This study is currently recruiting patients with the Hurler-Scheie or Scheie forms of MPS I. The first patient received the lowest dose planned in this study and remained on standard of care for a portion of the study. For additional details on the trial protocol, please see clinicaltrials.gov (NCT05682144). For patients interested in participating, please visit the patient recruitment website. The company has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for ISP-001 in MPS 1. "We are enthusiastic about this collaboration with Immusoft leveraging the first-ever engineered B cell platform technology to create novel solutions for this severe rare disease. We believe this innovative platform technology may provide real solutions for unmet medical needs across a broad number of indications," said Dr. Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM. In addition to its clinical MPS I program, Immusoft has programs in other lysosomal storage disorders including MPS II as well as active programs in CNS, metabolic disease, and oncology. 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), an essential enzyme in the breakdown of 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 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 . About the California Institute for Regenerative Medicine (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 one of the world's largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality. Media: Susan Roberts Roberts Communications [email protected] 202-779-0929 SOURCE Immusoft WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

SEATTLE--( BUSINESS WIRE )-- Immusoft Corporation , a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced that it will present at the upcoming WORLDSymposium 2024 Conference, held in San Diego, February 4-9, 2024. WORLDSymposium is the largest international gathering of lysosomal disease researchers. Details of the presentation are as follows: Presentation Title: First-in-human clinical trial of genetically engineered B cells: Application to the treatment of mucopolysaccharidosis type I Date/Time: Thursday, February 8, 2024 - 1:00 PM Presenter: R. Scott McIvor, Ph.D., Chief Development Officer at Immusoft, and Professor of Genetics, Cell Biology and Development at the University of Minnesota. Immusoft is developing a new modality of gene encoded medicine that holds the promise of being durable and redosable. The Company’s lead investigational drug candidate ISP-001, is a first-in-class investigational treatment for mucopolysaccharidosis type I (MPS I), a rare lysosomal storage disease. ISP-001 is an autologous B cell product engineered to express human alpha-L-iduronidase (IDUA) for the treatment of MPS I. In this first-in-human clinical trial, the patient was dosed without the need for a preconditioning regimen (required for gene modified stem cells) or immunosuppression (required for most 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, which supports the concept of redosability of engineered B cells. There is a significant unmet need for new therapies with improved efficacy and convenience in MPS I, where the current standard of care is enzyme replacement therapy (ERT). Due to their capacity for high-level protein production and their natural ability to engraft in bone marrow without need for toxic preconditioning, B cells have the potential to serve as a highly efficacious cellular therapy. Immusoft has established effective conditions for expansion and genetic engineering of B cells across multiple therapeutic indications. Additional information regarding the WORLDSymposium presentation can be found at: https://worldsymposia.org/ . About Immusoft 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 (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 .