Safety and initial activity of autologous human B cells genetically engineered to express human iduronidase using the Sleeping Beauty transposon system.
Results from a first-in-human clinical trial in subjects with MPS I
SAN FRANCISCO, CA, USA I January 27, 2025 I Immusoft of CA, a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced that it will feature oral and poster presentations at the upcoming WORLDSymposium™ 2025, held in San Diego, February 3-7, 2025.
Immusoft will present the following:
Oral Presentation: Friday, February 7th, 9:00 AM PST, Dr. Paul Orchard, MD.
Title: Safety and initial activity of autologous human B cells genetically engineered to express human iduronidase using the Sleeping Beauty transposon system: Results from a first-in-human clinical trial in subjects with MPS I
Presenter: Paul J. Orchard, MD., Professor, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy University of Minnesota Medical School
Poster Presentation: Wednesday, February 5th, 2026, 3:30-5:30 PM PST, Kiosk 27-B.
Title: Safety and initial activity of autologous human B cells genetically engineered to express human iduronidase using the Sleeping Beauty transposon system: Results from a first-in-human clinical trial in subjects with MPS I
Presentation Category: Translational Research & Late-Breaking Science Posters (LB-35 to LB-62)
Immusoft is developing B cells as biofactories for therapeutic protein delivery. This new modality has the potential to be both durable and redosable. The Company’s lead drug candidate, ISP-001, is the first ever engineered B cell in a human clinical trial and 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), both of which can be associated with severe toxicities.
The 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.
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) or hematopoietic stem cell transplant (HSCT). Due to their capacity for high-level protein production, their natural ability to engraft in the bone marrow without the need for toxic preconditioning, and their ability to manufacture a multitude of protein types, B cells have the potential to address unmet needs across numerous 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 approaches to treat rare diseases by sustained delivery of protein therapeutics from a patient’s own B 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 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.
SOURCE: Immusoft
Post Views: 293
Safety and initial activity of autologous human B cells genetically engineered to express human iduronidase using the Sleeping Beauty transposon system.
Results from a first-in-human clinical trial in subjects with MPS I
SAN FRANCISCO, CA, USA I January 27, 2025 I Immusoft of CA, a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced that it will feature oral and poster presentations at the upcoming WORLDSymposium™ 2025, held in San Diego, February 3-7, 2025.
Immusoft will present the following:
Oral Presentation: Friday, February 7th, 9:00 AM PST, Dr. Paul Orchard, MD.
Title: Safety and initial activity of autologous human B cells genetically engineered to express human iduronidase using the Sleeping Beauty transposon system: Results from a first-in-human clinical trial in subjects with MPS I
Presenter: Paul J. Orchard, MD., Professor, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy University of Minnesota Medical School
Poster Presentation: Wednesday, February 5th, 2026, 3:30-5:30 PM PST, Kiosk 27-B.
Title: Safety and initial activity of autologous human B cells genetically engineered to express human iduronidase using the Sleeping Beauty transposon system: Results from a first-in-human clinical trial in subjects with MPS I
Presentation Category: Translational Research & Late-Breaking Science Posters (LB-35 to LB-62)
Immusoft is developing B cells as biofactories for therapeutic protein delivery. This new modality has the potential to be both durable and redosable. The Company’s lead drug candidate, ISP-001, is the first ever engineered B cell in a human clinical trial and 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), both of which can be associated with severe toxicities.
The 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.
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) or hematopoietic stem cell transplant (HSCT). Due to their capacity for high-level protein production, their natural ability to engraft in the bone marrow without the need for toxic preconditioning, and their ability to manufacture a multitude of protein types, B cells have the potential to address unmet needs across numerous 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 approaches to treat rare diseases by sustained delivery of protein therapeutics from a patient’s own B 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 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.
SOURCE: Immusoft
Post Views: 293
