Cystic fibrosis (CF) causes severe damage to the lungs, digestive system and other organs. This inherited disease is caused by a defect in the CFTR gene, which normally makes a protein that controls the movement of salt and water in and out of the body’s cells.
According to the Cystic Fibrosis Foundation Patient Registry, more than 30,000 people are living with CF in the US, and more than 70,000 worldwide. Approximately 1,000 people are newly diagnosed with CF each year.
We have developed an mRNA therapy that targets the CFTR pathway. By providing a new set of instructions to replace those of the defective gene, healthy protein production could potentially be restored for all patients, regardless of mutation type.
The urea cycle is a series of metabolic processes that relies on five liver enzymes to rid the body of ammonia, a toxic byproduct of protein metabolism. Ornithine transcarbamylase (OTC) deficiency is a rare inborn error of metabolism and the most common of the urea-cycle disorders.
OTC deficiency is a devastating disease, occurring in one out of every 40,000 births. Typically, newborns with this condition slip into a coma within 72 hours of birth, and most suffer severe brain damage. Half die in the first month, and half of the survivors die by age five.
Translate Bio is rapidly advancing a treatment utilizing our mRNA technology to restore healthy levels of OTC protein production in the liver.
Rare Liver, Lung and CNS Diseases
Our platform for powering endogenous therapeutic protein production is applicable to a broad range of diseases caused by insufficient protein production. We are committed to making an impact for people suffering from these types of diseases, with a current focus on rare diseases of the liver, lung and central nervous system (CNS).