Intrexon Corporation (NYSE: XON), a leader in synthetic biology, and Synthetic Biologics, Inc. (NYSE MKT: SYN), a clinical-stage company focused on developing therapeutics to protect the microbiome while targeting pathogen-specific diseases, today announced an Exclusive Channel Collaboration (ECC) to pursue the development and commercialization of novel biotherapeutics for the treatment of patients with phenylketonuria (PKU), a serious and debilitating metabolic disorder. Through the ECC, the companies plan to utilize Intrexon's ActoBiotics™ platform providing a proprietary method of delivering therapeutic protein and peptides to the gastrointestinal (GI) tract through food-grade microbes.
"We are excited to build on our existing relationship with Intrexon through this new collaboration," commented Jeffrey Riley, Chief Executive Officer, of Synthetic Biologics. "We believe a biotherapeutics-based approach has great potential to provide relief for patients managing this devastating disease and an opportunity to improve upon current therapies that rely on constant dietary monitoring and drugs to increase phenylalanine breakdown. The development of these biotherapeutics expands our pipeline of products targeted for release in the GI tract without adverse impact on the natural balance of the patient's gut microbiome."
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, behavioral 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 U.S. 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 program and carefully controlled diet.
Samuel Broder, M.D., Senior Vice President and Head of Intrexon's Health Sector, said, "The goal of our collaboration is for our ActoBiotics™ program to deliver a key enzyme to facilitate the breakdown of phenylalanine and thereby prevent the toxicity in these patients." Dr. Broder added, "We are very pleased that Intrexon's suite of complementary technologies and proprietary industrial processes will facilitate our ECC partner, Synthetic Biologics, in the development of new therapies for PKU."
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