Intrexon Corporation, a leader in synthetic biology, and Synthetic Biologics, Inc., a clinical-stage company focused on developing therapeutics to protect the microbiome while targeting pathogen-specific diseases, announced an exclusive channel collaboration (ECC) to pursue the development and commercialisation of novel biotherapeutics for the treatment of patients with phenylketonuria (PKU), a serious and debilitating metabolic disorder.
PKU is a genetic disease that begins at birth characterized by a deficiency in the liver enzyme that breaks down the essential amino acid phenylalanine (Phe), a building block of proteins normally obtained through the foods we eat. As a result, Phe accumulates in the body, becoming toxic and leading to serious health consequences, including profound mental retardation, brain damage, mental illness, behavioural problems, seizures, tremors, limited cognitive ability and hyperactivity. If left untreated, the most severe form of PKU leads to permanent cognitive damage. PKU affects more than 14,000 people in the US and an estimated 50,000 people across developed nations globally. There is no existing cure for PKU, requiring patients to maintain a life-long treatment programme and carefully controlled diet.
Through the ECC, the companies plan to utilise Intrexon's ActoBiotics platform providing a proprietary method of delivering therapeutic protein and peptides to the gastrointestinal (GI) tract through food-grade microbes.