MRG-201 for Scleroderma

MRG-201 is a promiR (agonist) to microRNA-29b, and is miRagen Therapeutics‘ lead anti-fibrotic candidate. A promiR is a synthetic microRNA mimic, which is part of a group of innovative biomolecules used by researchers for gene targeting, antisense and cell reprogramming approaches.1 The microRNA-29 family is a well-established negative regulator of a wide variety of genes that play an important role in building collagen and other proteins involved in scar formation.

microRNA-29 expression is inactive in different pathological fibrotic conditions, including cardiac, renal, hepatic, and pulmonary fibrosis, as well as in scleroderma. Studies in cell cultures and animals (genetic replacement) have demonstrated the potential of miRNA-29 normalization to correct drivers of disease-causing fibrosis.2

Clinical testing of MRG-201

miRagen has initiated a Phase 1 study (NCT02603224) of MRG-201 in healthy volunteers to evaluate its safety and tolerability at single-ascending and multiple-ascending doses. MRG-201 is designed to mimic the activity of microRNA-29, and is being studied to determine if it can limit the formation of fibrous scar tissue.

MRG-201 will be tested by injection into intact skin or adjacent to a small skin incision. Healthy participants will be monitored for local reactions in the skin, signs or symptoms of adverse effects, and for the levels of MRG-201 in the blood over time. Skin biopsies will also be collected to study how skin cells respond when exposed to MRG-201.

If this Phase 1 study proves safe in healthy participants, the treatment may be extended to patients with cutaneous scleroderma.3

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