Phenylketonuria (PKU) is a rare genetic disorder estimated to affect 16,500 people in the U.S., according to the National PKU Association. People with PKU are unable to process phenylalanine (Phe), an essential amino acid that enters the body as a component of dietary protein. PKU requires adherence to a strict diet to prevent the toxic accumulation of Phe in the blood and brain, which can impact neurological function. Synlogic has designed a Synthetic Biotic medicine, SYNB1618, to produce phenylalanine-degrading enzymes designed to lower Phe levels to allow those with PKU to consume more natural protein. We are currently conducting clinical trials of SYNB1618 in people with PKU to demonstrate safety and to test whether this could be a useful therapeutic option to help patients manage their blood Phe levels.
Enteric hyperoxaluria is caused by increased absorption of oxalate as a result of certain intestinal diseases, including Crohn’s disease and short bowel syndrome or as a result of surgical procedures such as bariatric weight-loss surgery. The disorder can lead to a range of conditions of increasing severity from urinary tract infections to recurrent kidney stones and kidney disease and, potentially, the need for a kidney transplant. There are an estimated 250,000 patients with enteric hyperoxaluria in the United States, and more than 80,000 patients who experience recurrent kidney stones due to hyperoxaluria. Synlogic has engineered Synthetic Biotic medicines designed to consume oxalate in the GI tract to potentially lower oxalate levels and the risk of kidney damage.
MSUD is an inherited, rare metabolic disease characterized by a deficiency of enzymes required to break down certain amino acids known as branched chain amino acids (BCAA) which are components of proteins. The classic form of the disease becomes apparent soon after birth and, if left untreated, can lead to seizures, coma, and death. Variant forms of the disorder become apparent later in infancy or childhood and are typically milder, but also lead to delayed development and other health problems if not treated. It is estimated that there are approximately 1,500 MSUD patients in the United States, and 3,000 patients worldwide. A low protein/BCAA-restricted diet is the widely accepted long-term disease management strategy. However, this regimen can be problematic since BCAA are also essential nutrients that can only be acquired through diet. Synlogic has engineered Synthetic Biotic medicines designed to consume BCAA as a potential treatment for MSUD.
Cancer cells are known for their ability to evade the body’s immune system, allowing them to persist, proliferate and metastasize. Synlogic is designing Synthetic Biotic medicines that work to combat the tumor’s ability to evade the immune system, boosting a patient’s immune response and promoting the body’s ability to detect and destroy cancer cells. Synlogic has advanced its first clinical candidate in cancer, SYNB1891, into a Phase 1 clinical trial in patients with in patients with advanced solid tumors and lymphoma. More information can be found at www.clinicaltrials.gov. SYNB1891 is designed to activate the STING pathway specifically in antigen presenting cells that naturally sense and engulf bacteria. This results in local ablation of cancer cells as well targeting metastases that spread throughout the body.
Synlogic is also developing novel Synthetic Biotic medicines to treat inflammatory bowel disease.
Synlogic is evaluating its Synthetic Biotic platform for the development of a vaccine for the prevention of SARS-CoV2.