Drug Development Pipeline
Restore CFTR Function
MRT5005 is a new drug designed to restore CFTR function by delivering correct copies of CFTR-encoded mRNA to the lungs. mRNA is a molecule that contains genetic instructions to make proteins. Delivery of CFTR-encoded mRNA would allow the lung cells to create normally functioning CFTR protein, regardless of an individual’s specific CFTR gene mutation. This drug is delivered via inhalation.
A phase 1 & 2 study to test the safety and tolerability of MRT5005 is underway.
This program is sponsored by Translate Bio (previously RaNA Therapeutics). It is being conducted through the Therapeutics Development Network (TDN).
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