The problem: Inherited metabolic disorders happen when a defective gene creates an enzyme deficiency in the body. There are a lot of them, and some can be treated by managing diet, but others require regular enzyme infusions for a patient’s entire life.
The solution: Biotech company Immusoft has genetically altered B cells, which normally produce antibodies, to produce the enzymes a body is deficient in. Only a small portion of the body’s billions of B cells are removed to have a new DNA sequence added to its genome, leaving the rest to continue producing antibodies as normal.
The test was completed in a patient with MPS I, which causes respiratory problems, cognitive issues, enlarged organs, and — in severe cases — death before the end of childhood. The first trial was just to see if the modification could be completed safely — from here, the patient will be taken off of his regular infusions to see if the B cells are an effective replacement. Immusoft will also observe how long the modified B cells last in the body.