Defeat Duchenne Canada is pleased to share with you some updates from Entrada Therapeutics, currently running multiple exon-skipping clinical trials in Duchenne muscular dystrophy (DMD). Entrada continues to move forward with several clinical trials for people living with DMD in the U.S., U.K., and Europe. Currently, clinical trials through Entrada are not yet available in Canada. Full news release can be found below.
ELEVATE-44-201 (Exon 44) – locations: UK and Europe
- A Phase 1/2 study is ongoing in ambulatory boys with DMD, consisting of three cohorts of the multiple ascending dose (MAD) portion of the clinical trial for those amenable to exon 44 skipping and transitioned to the open label portion of the study (Cohort 1: 6mg/kg); Cohort 2 12 mg/kg; and Cohort 3: up to 18 mg/kg).
- Cohort 1 has completed dosing. Results from this cohort are expected in mid-2026, with data from Cohort 2 later in the year. And data from Cohort 3 following that.ย
- A separate study in adults (ambulatory and non-ambulatory) is expected to begin in early 2026.
- A phase 1b MAD study (called ELEVATE-44-102 taking place in the USA) will open for both ambulatory and non-ambulatory boys with DMD by mid-2026.
ELEVATE-45-201 (Exon 45) – locations: UK and Europe
- Patient dosing has started in a global Phase 1/2 study for ambulatory boys with DMD who are amenable to exon 45 skipping. There are also 3 cohorts in this trial (Cohort one 5mg/kg; Cohort two 10 mg/kg; and Cohort three 15 mg/kg).ย
- Early results are expected in mid-2026 for Cohort 1, with data to follow from the other two cohorts.ย
ELEVATE 50-201 (Exon 50) – locations: UK
- Approval has been received in the U.K. to begin a Phase 1/2 MAD study in ambulatory boys with DMD amenable to exon 50 skipping.ย
- The study is expected to begin by the end of 2026.
ENTR-601-51 (Exon 51) – locations: UK and Europe
- Global regulatory applications are planned for 2026 to establish this trial.ย
These efforts reflect continued progress and commitment to developing new treatment options for the Duchenne community.
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: