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Translate Bio discontinues development of genetic disorder drug

Translate Bio Inc. will discontinue the development of its experimental genetic disorder drug MRT5201 and prioritize its lung disease programs.

The biotechnology company was developing MRT5201 to treat ornithine transcarbamylase deficiency, a genetic disorder that allows ammonia to accumulate in the blood. High levels of ammonia are toxic and affect the nervous system.

Lexington, Mass.-based Translate Bio said results from preclinical trials of MRT5201 do not support the advancement of the program.

The company is prioritizing the development of pulmonary disease programs, including the ongoing development of MRT5005 to treat cystic fibrosis, an inherited disorder that causes severe damage to the lungs, digestive system and other organs in the body. Data from the ongoing study of the drug is expected in 2020.

Translate Bio's stock price was down 8.92% to $9.09 in after-hours trading Sept. 9.