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FDA Lifts Clinical Hold On Translate Bio's Liver Disorder Trial

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FDA Lifts Clinical Hold On Translate Bio's Liver Disorder Trial

Shares of the clinical-stage mRNA therapeutics company Translate Bio Inc (NASDAQ: TBIO), which went public in June 2018, were showing some strength Wednesday. 

What Happened

Translate Bio said Wednesday that the FDA has given its clearance to proceed with a single-ascending dose, or SAD, Phase 1/2 clinical trial of MRT5201 in patients with ornithine transcarbamylase, or OTC, deficiency.

The investigational asset acts by intravenous delivery of mRNA that encodes the fully functional OTC enzyme to the liver to enable the hepatocytes to produce normal OTC enzymes. 

OTC deficiency is a metabolic liver enzyme disorder resulting from a mutation in the OTC gene; it causes toxic levels of ammonia to build up in the blood.

Following the submission of an IND for MRT5201 in December, the FDA placed a clinical hold on the trial pending additional preclinical toxicology data.

The company subsequently had discussions with the FDA and amended the protocol, paving the way for the lifting of the clinical hold and the go-ahead for the the SAD trial.

What Next

TranslateBio said it is conducting additional preclinical studies to support future clinical development of MRT5201, including a multiple ascending dose clinical trial.

The company expects to submit data from these studies to the FDA in the fourth quarter of 2019.

Translate Bio shares were adding 4.99% to $13.05 at the time of publication Wednesday. 

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