Entrada Therapeutics announces the FDA has removed the clinical hold on ENTR-601-44, their experimental therapeutic designed to skip exon 44

As an early funder of Entrada Therapeutics, we are pleased to share that the FDA has removed the clinical hold on ENTR-601-44, the experimental therapeutic designed for individuals with Duchenne amenable to skipping exon 44.  ENTR-601-44 is a phosphorodiamidate morpholino oligomer (PMO) conjugated to a proprietary Endosomal Escape Vehicle (EEV™), which aims to facilitate delivery of the exon-skipping PMO to the right compartment within the muscle. 

Entrada will conduct the ELEVATE-44-102 trial in the US, a Phase 1b multiple ascending dose study in 32 ambulatory and non-ambulatory adults with Duchenne.  Dosing will be every 6 weeks, with 4 different dosing levels planned.   Entrada will be looking at safety, tolerability, and target engagement as measured by exon skipping and dystrophin production. 

Entrada also plans to conduct a global, two-part, randomized double-blind placebo controlled Phase 1/2 study, ELEVATE-44-201, outside of the US.

Link to press release:  https://ir.entradatx.com/news-releases/news-release-details/entrada-therapeutics-announces-fda-removal-clinical-hold-entr