Our Commitment to the CF Community

June 1, 2021

As Cystic Fibrosis (CF) Awareness Month comes to a close, we are taking time to reflect on the commitment of the CF community to find treatments for 100% of people with CF. You are an unstoppable community that doesn’t know the meaning of giving up, and one that always rises up to new challenges. Your steadfast commitment motivates us at Translate Bio as we continue our work towards achieving the same goal. We are unwavering in our mission of developing transformative mRNA medicines and bringing them to patients that need them. Our mission and culture are deeply rooted in the belief that mRNA has the power to treat and prevent many diseases, including CF.

“We are as committed to this goal as you are and we believe mRNA therapeutics have an important role to play in treating pulmonary diseases, including CF.”
Ron Renaud, CEO, Translate Bio

“I continue to be inspired by the CF community’s commitment and dedication to advancing CF research so that one day, 100 percent of people with CF will have treatment options,” said Ron Renaud, chief executive officer of Translate Bio. “We are as committed to this goal as you are and we believe mRNA therapeutics have an important role to play in treating pulmonary diseases, including CF.”

We recently reported interim data from our ongoing Phase 1/2 clinical trial of MRT5005, the first inhaled mRNA therapeutic for the treatment of CF. This trial – the first of its kind – is an important step forward in our efforts to develop an mRNA therapeutic for CF.

“This was the first time patients have ever inhaled mRNA and we believe we have achieved a safety profile that supports repeat dosing of inhaled mRNA and further advancement of the pulmonary platform for chronic dosing,” said Ann Barbier, M.D., Ph.D., chief medical officer of Translate Bio. “We are grateful to the CF patients who continue to engage and support the advancement of science, even in the face of a global pandemic.”

The RESTORE-CF trial is ongoing, and we anticipate sharing additional findings from the trial at a future medical meeting. We are also continuing with ongoing and additional translational studies with MRT5005 and our next-generation CF candidate to support and optimize future clinical development.

“While many have benefited from recent CFTR modulator therapies, it is an ongoing tragedy that over 10% of our community are still waiting for a substantive treatment. mRNA therapeutics have the potential to treat all members of our community,” said Siri Vaeth, Executive Director at Cystic Fibrosis Research Institute. “As the director of a patient advocacy organization and the mother of a young adult with CF, I was extremely pleased to learn that these recent results will be used to advance the science, and that Translate Bio is fully committed to finding a therapy for CF that will benefit all those living with this extraordinarily challenging disease.”

This CF Awareness Month, we are introducing Breathe Innovation, a campaign focused on sharing more about the potential of mRNA therapeutics to treat lung diseases like CF!

Over the last several years, we have developed web and social media campaigns in support of CF Awareness Month, including our Translating Now panel in 2019 and 5on5 series in 2020. This year, we are expanding on these efforts by launching Breathe Innovation with the goal of sharing more information about how mRNA therapeutics work, and why they could be transformative in treating lung diseases like CF. We hope that our work at Translate Bio to develop potentially transformative mRNA therapeutics for lung diseases will help many people breathe in hope for a future with more effective, potentially life-saving treatment options.