US-based biotechnology company Entrada Therapeutics has dosed the first subject in a Phase I clinical trial of ENTR-601-44 for the treatment of Duchenne muscular dystrophy (DMD).

The study is being carried out in the UK and its key goal is to assess the tolerability and safety of a single dose of ENTR-601-44 in healthy male volunteers with a target enrolment of nearly 40 subjects.

Target engagement, as measured by exon skipping in the skeletal muscle, and the pharmacokinetics of ENTR-601-44 will also be evaluated in the study.

Entrada received approval for the trial from UK regulatory authorities last month.

Entrada Therapeutics president and CEO Dipal Doshi said: “The initiation of our Phase I trial for ENTR-601-44 is an important step forward in addressing this relentlessly progressive neurodegenerative disease.

“As we enter our next phase of growth, Entrada is well-positioned to advance this initial programme, as well as our broader Duchenne franchise, and we expect to report data from the Phase I trial in the second half of 2024.”

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ENTR-601-44 is a part of Entrada’s lead oligonucleotide programmes, which also include ENTR-601-45.

These candidates are proprietary Endosomal Escape Vehicle (EEV) conjugated phosphorodiamidate morpholino oligomers (PMO).

Based on the EEV platform, Entrada is also developing enzyme-based, antibody and RNA programmes to treat immunological, ocular, metabolic and neuromuscular diseases among others.

In the second half of next year, the company plans to submit an investigational new drug (IND) application of ENTR-601-45 for the treatment of DMD patients who are exon 45 skipping amenable.

It announced ENTR-601-45 as the second clinical candidate in its DMD franchise in January this year.

DMD is a rare genetic disease that leads to progressive muscle degeneration and weakness throughout the body.

The condition is caused by mutations in the DMD gene preventing the body from producing enough dystrophin.