Entrada Therapeutics has received clearance to test the highest dose in its Exon 44 Skipping Study for Duchenne muscular dystrophy (DMD). The independent Data Monitoring Committee (DMC) has recommended starting Cohort 3 at 18 mg/kg in the ELEVATE-44-201 clinical trial, following its review of available safety and pharmacokinetic data from participants in Cohort 2.
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Entrada Advances to the 18 mg/kg Dose
The DMC reviewed safety and pharmacokinetic (PK) data from all eight participants enrolled in Cohort 2 of the trial’s double-blind, placebo-controlled, multiple ascending dose study. Learn More: DMDWarrior Exon Deletion Tool
Following this review, the committee recommended moving to Cohort 3 at 18 mg/kg, the highest dose planned in the study so far. This represents an increase from 12 mg/kg in Cohort 2 and 6 mg/kg in Cohort 1.
ELEVATE-44-201 is a Phase 1/2 clinical study evaluating ENTR-601-44 in people with Duchenne who have a confirmed DMD gene mutation amenable to exon 44 skipping.
What Is ENTR-601-44?
ENTR-601-44 is an experimental treatment developed by Entrada Therapeutics for people with Duchenne who have mutations suitable for exon 44 skipping. This group represents approximately 8% of the global Duchenne patient population.
The treatment uses an Endosomal Escape Vehicle (EEV™)-conjugated oligonucleotide designed to help the body’s naturally occurring DMD gene produce functional dystrophin.
Dystrophin is an important protein for muscle function, and Duchenne is caused by mutations in the DMD gene that affect its production or function.
What Does the Higher Dose Mean?
According to Natarajan Sethuraman, PhD, President of Research and Development at Entrada Therapeutics, advancing to 18 mg/kg is an important step in the clinical development of ENTR-601-44.
The company expects the higher dose could lead to greater drug exposure, increased dystrophin production and potential functional benefits. However, these are possibilities that the ongoing study aims to investigate, not confirmed clinical outcomes.
The dose escalation will also help researchers better understand the treatment’s safety, pharmacokinetic profile and pharmacodynamic effects.
When Will More Data Be Available?
Entrada Therapeutics expects to report data from Cohort 1 participants after one year of treatment in the open-label period later in 2026. Data from Cohort 2 are expected in the first quarter of 2027.
In December 2025, the U.S. Food and Drug Administration (FDA) granted ENTR-601-44 Rare Pediatric Disease Designation.
The move to the highest dose marks another step in the development of ENTR-601-44. Future clinical data will be important for understanding its safety and potential benefits for people living with Duchenne who are eligible for exon 44 skipping.
Discover More: Duchenne Clinical Trial Locations Map
