Novel cystic fibrosis option safe, but no sign of efficacy
Translate Bio (Nasdaq: TBIO) stock fell by a quarter in pre-market trading on Thursday, after interim data from a small trial testing mRNA candidate MRT5005 in cystic fibrosis (CF) failed to impress.
MRT5005 is the first clinical-stage prospect of its type to address the underlying cause of CF through a unique mechanism of action.
Importantly, the Phase I/II study showed that repeat dosing of MRT5005 was generally safe and well tolerated, with no serious adverse events.
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