Patients and Families

We are working tirelessly to develop a new class of potentially transformative mRNA medicines for patients living with genetic diseases.


As a clinical-stage mRNA therapeutics company, we are focused on supporting patients throughout the development of mRNA therapeutics to treat diseases caused by protein or gene dysfunction. We recognize patients and their families need support throughout their treatment journey, and we are committed to providing resources and engaging with the patient communities we hope to serve in the future. Every year, we participate in and support patient advocacy groups in the rare disease community that increase disease awareness, further research, and provide community support for patients and families.

Photo: Rare Disease Day

Each year, Translate Bio joins the rare disease community in recognizing Rare Disease Day on February 28. In 2020, Translators created a handprint mural to join in the “We are the 300 million” movement.

Photo: Translate Now

During CF awareness month, Translate Bio hosted the “Translating Now” Panel as a rallying point for the CF community of advocates, HCPs, caregivers, and patients on how the fight continues to develop CF treatment options that may ease the burden still carried by 10% of patients. Read more.

Photo: Great Strides

Translate Bio proudly sponsored and participated in the Cystic Fibrosis Foundation Massachusetts/Rhode Island Chapter’s Great Strides event in May 2019.

Photo: Translate in Belgium

In partnership with CF Europe, Translate Bio’s clinical team traveled to Brussels, Belgium, in October 2019 to conduct a community advisory board meeting with cystic fibrosis patients, caregivers, and advocacy organizations to plan for future clinical development of our CF program.

Support & Resources

We are proud to support and partner with the following organizations. Visit their websites for additional resources and to learn more about their commitment to the CF community:

Logo: BEF Logo: Cystic Fibrosis Foundation Logo: CFLF Logo: CFRI

Logo: Roundtable Logo: Emily's Entourage Logo: Jerry Cahill Logo: Love to Breathe
Logo: Roadmap to CF Logo: Rock CF Logo: Take a Breather Logo: Vivian Lee

About the MRT5005 Phase 1/2 Clinical Trial

The randomized, double-blind, placebo-controlled Phase 1/2 clinical trial of MRT5005 is designed to enroll at least 40 adult patients with CF who have two Class I and/or Class II mutations. The primary endpoint of the trial will be the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization.  Percent predicted forced expiratory volume in one second (ppFEV1), which is a well-defined and accepted endpoint measuring lung function, will also be measured at pre-defined timepoints throughout the trial. The Phase 1/2 clinical trial of MRT5005 for the treatment of CF is being conducted in collaboration with the Cystic Fibrosis Foundation Therapeutics Development Network. In July 2019, the Company announced interim results from single-ascending dose (SAD) portion of Phase 1/2 clinical trial of MRT5005 in patients with CF. Get more information about the Phase 1/2 clinical trial here.

Expanded Access

Translate Bio is committed to developing a new class of medicines to treat diseases caused by protein or gene dysfunction. Our pipeline includes MRT5005, a phase 1/2 product candidate designed to deliver mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to the lung. Prior to regulatory approval, Translate Bio believes the optimal way to gain access to an investigational medicine is to participate in an ongoing clinical trial. For information about our clinical trials, please consult Expanded access is not available at this time. As clinical development progresses and more clinical data is available, Translate Bio will evaluate the possibility of expanded access.

For more information about our involvement in the community, please contact Maura Gavaghan, Associate Director of Corporate Communications at