The first patient has been dosed with an engineered B cell investigational therapy in a Phase I trial in Mucopolysaccharidosis type I (MPS I).
Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication.
MPS I is a rare, genetic disease that affects the body’s ability to produce the enzyme alpha-L-iduronidase (IDUA).
The patient was dosed without the need for a chemotherapy (required for gene modified stem cells) or immunosuppression (required for systemic virus-delivered gene therapy).
Paul Orchard, Principal Investigator, 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, commented: “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’s Immune System Programming (ISP) approach is designed to enable reprogramming of a patient’s B cells for constant production of therapeutic proteins, mitigating the need for frequent enzyme administration and the potential to improve patient outcomes.
“We’re excited to support Immusoft’s program, as our goal is to always move the most promising research forward as fast as we can,” said Dr Abla Creasey, Vice President of Therapeutics Development, California Institute of Regenerative Medicine (CIRM). “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.”