Adeno-associated viruses, illustration
Credit: Kateryna Kon/Science Photo Library

Immusoft has dosed the first patient with an engineered B cell therapy—ISP-001 for MPS I (Mucopolysaccharidosis type I). This approach has several potential advantages over current gene and cell therapies. It does not require preconditioning (myeloablative chemotherapy) or immunosuppression, is administered as an IV on an outpatient basis, and it appears engineered B cells can be given repeatedly without causing resistance to develop.

“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,” said Sean Ainsworth, CEO, Immusoft. 

Several groups are trying to advance engineered B cell therapy as an alternative to gene or cell therapy, including Be Biopharma and Walking Fish Therapeutics.

Immusoft’s Immune System Programming (ISP) approach is designed to  reprogram a patient’s B cells for constant production of therapeutic proteins. ISP modifies a patient’s B cells and instructs the cells to produce gene-encoded medicines. The B cells then become protein therapeutic biofactories that are expected to persist for many years. ISP-001 is the company’s first product candidate and has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation in MPS I.

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. Currently approved treatments consist of enzyme replacement therapy (ERT), which needs to be done regularly, or hematopoietic stem cell transplantation.

“Existing interventions for MPS I are not curative and do not adequately address many significant disease-related complications,” said Paul Orchard, MD, principal investigator in the ISP-001 Phase I 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. 

He added that, “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.”

“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,” said Abla Creasey, VP of Therapeutics Development, California Institute of Regenerative Medicine (CIRM).

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