- Translate Bio Inc (NASDAQ: 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.
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