New Updates From Translate Bio in Drug Development for Cystic Fibrosis and OTC Deficiency

According to a story from globenewswire.com, the messenger RNA therapeutics company Translate Bio recently released the latest updates in regards to two of its leading product candidates for the treatment of cystic fibrosis and ornithine transcarbamylase (OTC) deficiency. Translate Bio is dedicated to the development of an innovative class of potentially groundbreaking medicines in order to treat illnesses linked to gene or protein dysfunction.

Updates for MRT5005 (Cystic Fibrosis)

The company’s flagship product candidate is called MRT5005. It is in development as a treatment for cystic fibrosis. At this juncture, a Phase 1/2 clinical trial is underway. This investigational treatment is intended to address the underlying disease mechanism no matter what mutation is responsible for the disorder in a given case. This clinical trial will consist of a single ascending dose and a multiple ascending dose portion. The first patient to receive the multiple dose is expecting to begin in early 2019, and interim data should be available in the second half of the year.

About Cystic Fibrosis

Cystic fibrosis is a type of genetic disorder which can have impacts throughout the body, but it is most characterized by the build up of abnormally thick, sticky mucus in the lungs. Symptoms of cystic fibrosis include progressive decline in lung function, lung and sinus infections, coughing up mucus, fatty stool, poor growth, infertility in males, clubbed digits, and digestive problems. Life expectancy ranges into the 40s and 50s with good care. To learn more about cystic fibrosis, click here.

Updates for MRT5201 (OTC Deficiency)

The company’s second product candidate is designated MRT5201. This experimental therapy is in development as a treatment for OTC deficiency, which is the most widespread urea cycle disorder. Translate Bio has recently sent an Investigational New Drug application to the US Food and Drug Administration for review. This application will help support the beginning of Phase 1/2 clinical trials for the drug. The company hopes to begin the process of finding participants in early 2019. 

About OTC Deficiency

OTC deficiency is a urea cycle disorder characterized by the buildup of dangerous levels of ammonia in the blood. Symptoms can include behavioral changes, seizures, encephalopathy, difficulty controlling breathing and body temperature, and coma. It is potentially fatal without prompt intervention. Treatment may include dialysis, a low protein diet, and liver transplant. To learn more about OTC deficiency, click here.


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