Immusoft has successfully dosed the first patient in a phase 1 trial for its engineered B cell therapy, ISP-001, targeting the rare genetic disorder, Mucopolysaccharidosis type I (MPS I).
The trial, conducted at M Health Fairview University of Minnesota Medical Center, aims to address the genetic disease's impact on enzyme production, causing sugar accumulation in cells and severe complications.
ISP-001, which has received FDA Orphan Drug designation and Rare Pediatric Disease designation, takes a unique approach by genetically programming B cells extracted from a patient's blood, avoiding the conventional focus on bone marrow stem cells or T lymphocytes. Immusoft’s Immune-System Programming (ISP) method allows for easier removal, lab engineering and reinfusion via an IV drip.
MPS I, a rare and often lethal genetic disorder, impacts the body's ability to produce the vital enzyme IDUA (alpha-L-iduronidase). This enzyme is crucial for breaking down long-chain sugars within cells, and in severe cases, occurring in approximately 1 in 100,000 births, symptoms manifest within a child's first year, leading to progressive damage, particularly in the brain. The severe form is typically fatal within the first decade of life and a milder form, attenuated MPS I, affects about 1 in 500,000 births, with symptoms appearing later in childhood.
In 2021, Takeda Pharmaceutical and Immusoft entered a collaboration exceeding $900 million to develop treatments for rare inherited metabolic disorders. Using Immusoft’s ISP technology, the two focused on creating cell therapies for disorders with central nervous system complications.
