Entrada Therapeutics is working on several treatments for Duchenne muscular dystrophy (DMD). Here’s what’s new with each treatment:
Exon 44 skipping (ENTR-601-44)
Enrolment is finished for Cohort (group) 2 of this study. Earlier results from Cohort 1 were positive. No one had to stop treatment and there were no serious side effects. Participants also showed early improvements in how quickly they could rise from the floor, which is a common measure of muscle strength in DMD studies.
Health authorities in the U.K. and Europe have approved a follow-up study (called ENTR-DMD-202). This means people in Cohort 1 can keep receiving treatment while researchers continue to track safety and movement over a longer period.
What’s next: Results from this longer Cohort 1 period are expected by the end of 2026. Results from Cohort 2, at a higher dose, are expected in early 2027. A third group (Cohort 3) may follow if needed. Separately, based on what they learned from Cohort 1, Entrada plans to talk with the U.S. FDA about starting future U.S. patients on a higher dose than originally planned. More details will come after those discussions.
Exon 45 skipping (ENTR-601-45)
Cohort 1 of this study is fully enrolled and have been dosed. A safety committee reviewed the results from the eight participants and gave the go-ahead to start Cohort 2 at a higher dose with no changes needed to the study plan. All Cohort 1 participants are now continuing treatment in an open-label phase, and Cohort 2 dosing is underway.
What’s next: Results from Cohort 1 are expected in October 2026. Results from Cohort 2 are expected in the first half of 2027. A third group (Cohort 3) may follow if needed.
Exon 50 skipping (ENTR-601-50)
U.K. regulators have given approval to start a new study of this treatment. Entrada plans to apply for approval in other countries once more data comes in from its other programs.
Exon 51 skipping (ENTR-601-51)
This treatment covers the largest group of people with DMD who can benefit from exon-skipping therapies. Entrada has finished the required early studies and plans to apply for approval to begin clinical trials once it has reviewed results from its other programs.
What this means for Canadians
These studies are taking place in the U.S., U.K., and Europe, and there is not yet a confirmed timeline for when any of these treatments might become available in Canada. Nonetheless, progress matters for our community here at home as each milestone helps build the case for future treatments and keeps momentum moving in the right direction.
Defeat Duchenne Canada is following these developments closely, and we will continue advocating so that if a treatment is proven safe and effective, Canadian families have fair and timely access to it. We will keep you updated as more news comes in.
Want to know more about Duchenne Research?
Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: