OTC deficiency leads to a buildup in the blood of ammonia, a neurotoxin. The US Food and Drug Administration has placed a hold on a planned trial of Translate Bio ’s messenger RNA ( mRNA ) drug , citing the need for answers to clinical and nonclinical questions regarding the firm ’s practices . The company announced in December to run a Phase 1/2 study of MRT5201 , an mRNA treatment designed to treat a rare metabolic disorder . Translate Bio said it is expecting written confirmation to follow the verbal notification , and will work with the administration to resolve its queries .
The US Food and Drug Administration has placed a hold on a planned trial of Translate Bio ’s messenger RNA ( mRNA ) drug , citing the need for answers to clinical and nonclinical questions regarding the firm ’s practices . The company announced in December to run a Phase 1/2 study of MRT5201 , an mRNA treatment designed to treat a rare metabolic disorder . Translate Bio said it is expecting written confirmation to follow the verbal notification , and will work with the administration to resolve its queries .