Entrada Therapeutics announced on February 3, 2025, that it received authorization from the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee. This authorization is to initiate ELEVATE-44-201, a Phase 1/2 multiple ascending dose (MAD) clinical study of ENTR-601-44 for Duchenne muscular dystrophy (DMD) patients amenable to exon 44 skipping.
The company is on track to initiate ELEVATE-44-201 in Q2 2025. ELEVATE-44-201 is a global, two-part, randomized, double-blind placebo-controlled study designed to evaluate the safety, tolerability, and effectiveness of ENTR-601-44 in ambulatory DMD patients.
Part A of the study will assess safety, pharmacokinetics, and pharmacodynamics, including exon skipping and dystrophin production, in approximately 24 patients. Planned doses across three cohorts are anticipated to range from 6 mg/kg up to 18 mg/kg, building on positive Phase 1 safety data in healthy volunteers.
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