Entrada Therapeutics has received authorisation to begin a Phase I clinical trial of ENTR-601-44 in healthy volunteers to treat Duchenne muscular dystrophy in the UK.
The authorisation is granted by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) and Research Ethics Committee (REC) for the company’s Clinical Trial of an Investigational Medicinal Product (CTIMP).
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The study’s primary objective is to assess the safety of a single dose of the lead product candidate ENTR-601-44 in healthy subjects, with a target enrolment of nearly 40 participants.
It will also assess pharmacokinetics, tolerability and target engagement, as measured by exon skipping in the skeletal muscle.
Entrada Therapeutics president and CEO Dipal Doshi said: “We are looking forward to this important next step in advancing ENTR-601-44 for the potential treatment of people with exon 44 skipping amenable Duchenne muscular dystrophy, where there exists a profound unmet medical need.
“This milestone, coupled with the extension of our cash runway through the end of 2025, positions Entrada to advance our Duchenne franchise while broadening the potential of our intracellular therapeutics across serious diseases.”
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By GlobalDataThe dosing of the first participant is anticipated in September while ENTR-601-44 study data is expected in the second half of next year.
ENTR-601-44 is an Endosomal Escape Vehicle (EEV)-conjugated phosphorodiamidate morpholino oligomer (PMO) with an oligonucleotide sequence design.
It restores the mRNA reading frame thereby allowing the translation of functional dystrophin protein that is slightly shortened.
Other lead oligonucleotide programmes of the company include ENTR-601-45 for treating Duchenne with exon 44 and 45 skipping amenable and ENTR-701 for targeting myotonic dystrophy type 1.
