Drug Development Pipeline
Restore CFTR Function
MRT5005 is a 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 has been completed. No further development in CF is planned at this time.
This program was sponsored by Translate Bio (previously RaNA Therapeutics). It was conducted through the Therapeutics Development Network (TDN).
Recent MRT5005 Studies
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