Researchers Genetically Program B Cells to Treat Genetic Disorder

A Seattle-based biotech company is conducting a trial using genetically programmed B cells to treat a genetic disorder called mucopolysaccharidosis type I (MPS I). The disorder causes a lack of an essential enzyme that breaks down long-chain sugars in the body. This enzyme deficiency leads to life-threatening symptoms, including cloudy eyes, respiratory problems, cognitive issues, and enlarged organs. Patients with MPS I currently receive weekly infusions of the missing enzyme, but Immusoft, the biotech company conducting the trial, aims to use genetically engineered B cells to produce the enzyme instead. B cells are known for their ability to produce high volumes of proteins, which makes them a promising option for this therapy. By reprogramming a patient’s B cells to produce the missing enzyme, Immusoft hopes to eliminate the need for regular infusions. Read more >>