UK-based MiNA Therapeutics has started the phase I clinical study (OUTREACH) of MTL-CEBPA for the treatment of patients with severe liver cancer.
The MTL-CEBPA is composed of a double-stranded RNA formulated into a SMARTICLES liposomal nanoparticle, and is intended to activate the CEBPA gene, which encodes the CCAAT / enhancer binding protein alpha (C/EBP-a).
C/EBP-a is a transcription factor that solely regulates cell lineage determination and differentiation in several tissues including liver, myeloid cells and adipose tissue.
Within the liver, the C/EBP-a is instrumental in the normal hepatocyte function and response to injury.
OUTREACH is the first-in-human, multi-centre, Phase I clinical trial of a small activating RNA (saRNA) and is designed to assess the safety and tolerability of MTL-CEBPA, an saRNA restoring the expression of C/EBP-a, in patients with advanced primary or metastatic liver cancer who are ineligible or resistant to standard therapies.
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MiNA Therapeutics CEO Robert Habib said: "Initiation of the Phase I study is an important achievement in our mission to improve patients’ lives with our groundbreaking class of medicines, known as small activating RNAs.
"There is increasing excitement about the possibility of using RNA to induce therapeutic protein production.
"We believe our unique approach, here applied to the upregulation of C/EBP-a protein, may provide to patients significant benefits over conventional medicines."
The trial will involve a dose-escalation followed by a dose expansion, wherein the MTL-CEBPA will initially administered as an intravenous infusion once weekly for three weeks, followed by one week of rest.