Entrada Therapeutics has announced that the first participant has been dosed in its Phase 1 clinical trial evaluating ENTR-601-44, the company’s proprietary Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO), for the potential treatment of individuals with Duchenne muscular dystrophy who are exon 44 skipping amenable.

Endosomal Escape Vehicle (EEVTM)-conjugated phosphorodiamidate morpholino oligomers (PMO) are a new, next generation class of exon skipping medication that works to induce skipping, which could potentially restore the reading frame in order to produce a shortened dystrophin protein. This is different from other exon skipping therapies—it is hoped that the EEV will increase the uptake of the exon skipping PMO by muscle cells, which could then increase the amount of dystrophin produced. 

The primary objective of Entrada’s Phase 1 clinical trial, which is being conducted in the United Kingdom, is to evaluate the safety and tolerability of a single dose of ENTR-601-44 in healthy males. The trial will also evaluate how the body processes ENTR-601-44 and its ability to localize to muscle cells, as measured by exon skipping in the skeletal muscle. Entrada has indicated that data from the Phase 1 trial is expected to be reported in the second half of 2024.

We look forward to additional updates from Entrada on ENTR-601-44, and are optimistic about the potential of the drug to expand the Duchenne drug pipeline to one day offer yet another treatment option for individuals living with Duchenne.

Read Entrada’s press release here.