Translate Bio has been moving forward with the development of their new drug candidate, MRT5005. This mRNA therapy was created to address the underlying cause of cystic fibrosis (CF), and has just entered a Phase 1/2 clinical trial. Through a recent announcement, Translate Bio has released that enrollment and dosing have just been completed for the second interim data analysis.

About Cystic Fibrosis (CF)

Cystic fibrosis is a genetic disorder that is characterized by progressive damage to the respiratory and digestive systems. Those with cystic fibrosis do not have the slippery mucus that is normally found in the lungs, instead they have thick and sticky mucus which builds up in their system. This buildup causes clogs in the airways, which then traps bacteria and causes breathing problems, infections, lung damage, and respiratory failure. It can also block digestive enzymes, which makes it difficult to absorb nutrients. Cystic fibrosis is a recessive disorder, meaning that the mutated gene must be passed down by both parents. The gene responsible for this condition affects the protein that regulates salt movement. The mutation in the gene varies in severity as well.

Symptoms of cystic fibrosis affect the respiratory and digestive systems. They include:

• Persistent coughing and wheezing
• Shortness of breath
• Difficulty exercising
• Frequent lung infections
• Stuffy noses
• Trouble with gaining weight
• Constipation
• Male infertility
• Salty-tasting skin
• Exercise intolerance

About MRT5005

This messenger RNA therapy is the first of its kind, as it is designed to target the underlying cause of cystic fibrosis by bringing a fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein directly to the epithelial cells in the lungs. It does so through nebulization. Unlike other therapies, it can treat all individuals with CF, regardless of genetic mutations.

It has already been granted the Orphan Drug, Rare Pediatric Disease, and Fast Track designations.

About the Trial

At least 40 patients will participate in the trial, which will be double-blind, randomized, and placebo-controlled. Researchers will be focusing on the tolerability and safety of both single and ascending doses, the primary endpoint.

There will be numerous multiple-ascending dose (MAD) groups, who will receive 8, 12, then 16 mg of MRT5005. One group will receive a single-ascending dose (SAD) of 20 mg.

Results are expected in the beginning of the second quarter of 2021.

You can read more about the trial here.