Entrada Therapeutics, Inc., today announced a significant step forward in their Duchenne muscular dystrophy (DMD) clinical development programme. They have received authorisation from Health Authorities and Ethics Committees across multiple European Union countries, under the EU Clinical Trial Regulation (EU-CTR), to proceed with their ELEVATE-45-201 study. This new study will investigate a potential treatment for individuals living with DMD who are amenable to exon 45 skipping.
This authorisation marks a crucial milestone for Entrada, as they now have regulatory clearance to conduct both their ELEVATE-44-201 and ELEVATE-45-201 studies across both the UK and Europe. This expands on their US ELEVATE-44-102 study, which received FDA clearance and is due to commence in 2026.
Below are brief summaries of Entrada’s key studies:
- ELEVATE-44-201 Study: A global, two-part, randomised, double-blind, placebo-controlled Phase 1/2 study. It aims to evaluate the safety, tolerability, and effectiveness of ENTR-601-44 in ambulant patients with DMD who are amenable to exon 44 skipping. Entrada Therapeutics is on track to initiate this study in Q2 2025, with study sites planned across the UK and Europe.
- ELEVATE-45-201 Study: Also a global, two-part, randomised, double-blind, placebo-controlled Phase 1/2 study, ELEVATE-45-201 will evaluate the safety, tolerability, and effectiveness of ENTR-601-45 in ambulant patients with Duchenne who are amenable to exon 45 skipping. Entrada Therapeutics anticipates initiating this study in Q3 2025, with study sites expected in the UK and Europe.
ENTR-601-44/45
ENTR-601-45 is a new investigational medicine from Entrada Therapeutics designed to help people with Duchenne muscular dystrophy (DMD) who have specific genetic changes that make them suitable for exon 44 and 45 skipping. This therapy uses a special delivery system called Endosomal Escape Vehicle (EEV) technology to get its active ingredient, a phosphorodiamidate morpholino oligomer (PMO), directly into muscle cells. Once inside, the PMO works to “skip over” a faulty section of the genetic code, which helps the body produce a slightly shorter, but still working version of the dystrophin protein. The goal is to restore the mRNA reading frame, which addresses the underlying cause of DMD.
Action Duchenne will continue to monitor the progress of these and other promising clinical trials, providing updates as they become available.
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