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Research Corner: Translate Bio Recruiting Class 1 and Nonsense Mutation Patients

By Ella Balasa

We published a blog back in the fall, sharing information about MRT-5005, Translate Bio’s drug candidate which is a novel therapy delivering mRNA to form functional proteins in the lung epithelial cells. This treatment, unlike current modulator therapies, would be useful for all patients regardless of mutation! If you are interested in learning more about the science of how it works and to learn about preliminary results from their single ascending dose (SAD) portion Phase 1/2 trial, check out our blog: MRT5005 – A Novel Therapy for Treating CF.


Since Translate Bio released its data on the first portion of this trial in mid-2019, Trikafta has been approved changing the trajectory of disease for many patients. However, for those who have two nonsense or class I mutations, and no current modulator options, the feeling of being left behind is unmistakable. It’s these patients who Translate Bio is especially targeting to get into their current trial. I spoke with the Chief Medical Officer, Dr. Ann Barbier, who clarified some of the questions surrounding inclusion criteria, recruitment, and dosing.


...for those who have two nonsense or class I mutations, and no current modulator options, the feeling of being left behind is unmistakable. It’s these patients who Translate Bio is especially targeting to get into their current trial.

In light of Trikafta being approved, what are the changes in the exclusion criteria for this phase of the trial? Patients taking Kalydeco or Trikafta are not eligible to participate in the ongoing clinical trial of MRT5005 at the current time. Depending on the uptake of Trikafta and the enrollment into the trial, these exclusion criteria may be reconsidered.

However, patients on a stable regimen of Orkambi or Symdeko are not excluded from participating, but the number of these patients will become fewer as more start on Trikafta.


To reach the 40 patient recruitment goal, patients who are not eligible for modulator therapies are encouraged to participate. If you fall into this category and are interested, please inquire from your CF care center. You can find more information about the 15 sites that are enrolling as well as contact information at https://clinicaltrials.gov/ct2/show/NCT03375047


Now, to review some of the specifics of the study.


What is the dose concentrations and frequency of administration? Patients in the current Multiple Ascending Dose (MAD) portion of the study will receive 5 once-weekly doses of MRT5005 or placebo in three cohorts, at 8, 12 and 16mg dose levels. Depending on the data from the study, they may choose to implement a fourth cohort, where the dose level would be 20 mg.


Translate Bio anticipates sharing data from the first 3 dose groups in the MAD portion in the third quarter of 2020. As a reminder, the single-ascending dose (SAD) results indicated that MRT5005 was generally well tolerated with FEV1 increases in several patients higher than one would expect to see based on known variability of FEV1.


Having treatment available to the remaining small percentage of patients is fueling the CF community’s interest and anticipation for this novel therapy! As a proponent of clinical trial participation to advance research rapidly, if you are eligible, I encourage you to reach out to your care team or to Translate Bio directly.

About the Author: Ella is a director for USACFA. She is 27 years old and was diagnosed with CF at 18 months old. She has a B.S. in biology and has worked in an environmental microbiology lab. She serves on various research committees, planning virtual events, and writes to provide a scientific voice and encourage empowerment to the CF community as well as introspectively writing about the hardship yet triumph that comes along with a chronic disease. When she is not taking care of her health, she enjoys cooking, drawing, and spending time with friends. Follow her life experiences on Instagram @thisgirlella.

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*The CF Roundtable does not give medical advice. Any medical opinions represented in these articles are those of the writer and do not represent the views of USACFA, any of our community partners, or any other group or individual. We strongly suggest you consult your doctors regarding any medical references and before altering your medical regimen in any way. USACFA does not endorse any products or procedures. 

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