Phenylketonuria is a rare condition where patients are born without a vital enzyme needed to destroy phenylalanine, which is a common amino acid. If phenylalanine levels increase to toxic degrees, there is a potential for patients to experience neurological symptoms, which means they need to adopt highly restrictive diets to avoid consuming phenylalanine.
BioMarin has already developed two drugs which have been approved to lower phenylalanine levels in some patients. However, a gene therapy which replaces the enzyme could provide a long-term treatment to patients living with the disease.
