Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is a condition that affects several parts of the body, particularly the digestive and nervous systems.
MNGIE is caused by mutations in the TYMP gene. These mutations result in the insufficient activity of an enzyme called thymidine phosphorylase (TP). TP is important for the metabolism of two nucleosides, thymidine and deoxyuridine. Without TP, MNGIE patients accumulate thymidine and deoxyuridine in their tissues and blood, causing mitochondrial damage. Over time, mitochondrial damage results in characteristic MNGIE symptoms, including dramatic weight loss as well as gastrointestinal and neurological symptoms.
MNGIE is a rare disease and is often underdiagnosed and misdiagnosed as neuropathy, anorexia, Crohn’s disease, or irritable bowel syndrome.
Entrada is developing ENTR-501 as an intracellular enzyme replacement therapy (IC-ERT) for MNGIE. ENTR-501 consists of Entrada’s proprietary EEV linked to TP enzyme and is designed to address the deficiency of TP activity inside the cells of MNGIE patients.
In 2020, Entrada received Orphan Drug Designation from the U.S. FDA for ENTR-501 for the treatment of MNGIE.
HOW ENTR-501 WORKS
MNGIE Natural History Study
Entrada is planning a natural history study to better understand the disease progression of MNGIE patients.