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.
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