MRT5005 has been designed to address the primary cause of cystic fibrosis (CF) irrespective of genetic mutation

Reports confirm that Translate Bio, an mRNA therapeutics company, has recently announced the interim results from a first-in-human Phase 1/2 clinical study evaluating multiple and single ascending dosages of MRT5005 in patients with cystic fibrosis.

Apparently, MRT5005 has been designed to address the primary cause of cystic fibrosis (CF), irrespective of genetic mutation, by supplying mRNA encoding fully functional CF transmembrane conductance regulator protein to lung cells via nebulization.

Sources familiar with the matter informed that the recent results of the first clinical research of an inhaled mRNA therapeutic, inform about the single ascending dose portion of the clinical study in 12 patients from one month follow up after the treatment. On the other hand, the multiple ascending dose portion of the clinical trial is currently ongoing with data anticipated in 2020.

According to director of the Gregory Fleming James Cystic Fibrosis Center and principal investigator of the Phase 1/2 clinical trial of MRT5005, Steven Rowe, M.D., efficiently delivering the required mRNA sequence is a vital step to creating functional CFTR protein.

Dr. Rowe said that some ppFEV₁ flexibility from day to day among CF patients is anticipated in a clinical trial of this size. The company believes the improvements witnessed in ppFEV₁ from baseline to the timecourse of the effect support a CFTR related mechanism and possibly suggest that MRT5005 can allow the production of functional protein.

Seemingly, these initial promising data warrant continuous MRT5005 evaluation to enhance the dosing regimen and evaluate the effect with repeated administration. Once future data confirms this initial positive signal, MRT5005 has the potential to offer clinically meaningful benefit for patients with CF independent of CFTR genotype.

Chief medical officer of Translate Bio, Ann Barbier, M.D., Ph.D., stated that despite substantial advances in the treatment of patients with CF, major unmet needs still remain among patients whose genetic mutations are believed non-amenable to CFTR modulators.

Source Credits: https://investors.translate.bio/news-releases/news-release-details/translate-bio-announces-interim-results-phase-12-clinical-trial