Translate Bio's mRNA Therapeutic For Respiratory Tract Infections Shows Promise In Animal Studies

Translate Bio Inc TBIO has presented positive results from a preclinical study of its mRNA-based therapeutic designed to treat the pulmonary component of primary ciliary dyskinesia (PCD).
Mutations in the genes that cause PCD result in ineffective mucociliary clearance, which can lead to lung disease.
The data were presented at the American Thoracic Society 2021 International Conference.
Study results suggest that delivery of an mRNA-based therapeutic to the lungs can lead to the expression of DNAI1, which could potentially restore mucociliary clearance, the cellular mechanism of the disease.
Multiple iterations of codon optimization resulted in selecting an mRNA sequence that had further improved protein expression by 30%.
DNAI1 mRNA packaged in proprietary LNPs and delivered by an intratracheal or nebulized route of administration resulted in DNAI1 expression in multiciliated airway epithelial cells in vivo.
Repeat administration showed an increased number of DNAI1-expressing cells and suggested a steady-state of restoration.
The company expects to advance a lead PCD candidate into IND-enabling studies in the second half of 2021.
Price Action: TBIO shares are up 3.62% at $17.61 during the market session on the last check Tuesday.