▎WuXiEdited by Kant Content Team

Recently, MiNA Therapeutics announced new preclinical data for its primary activating RNA (RNAa) product targeting fetal hemoglobin (HbF). The results of the preclinical in vivo studies indicate that,MiNA Therapeutics' RNAa therapeutic can be delivered to erythroid progenitor cells in the bone marrow, and the drug levels are clinically significant for treating β-hemoglobinopathies.

These data from animal models confirm that MiNA Therapeutics' RNAa therapeutic targeting HbF can efficiently distribute to erythroid progenitor cells, achieving clinically significant delivery levels, and is effective in treating β-hemoglobinopathies.In non-human primates, intravenous administration of the liposomal formulation NOV340 encapsulating MiNA Therapeutics' RNAa complex delivers to over 60% of erythroid colony-forming units (CFU-E) and proerythroblasts (Pro-E) in the bone marrow.Equivalent delivery efficiency was also observed in peripheral blood mononuclear cells, where the pharmacodynamic activity of RNAa therapy formulated with NOV340 in this cell type has been previously confirmed in clinical studies.

MiNA Therapeutics' HbF product aims to increase γ-globin (HBG) gene transcription, leading to higher levels of HbF in patients with β-hemoglobinopathy.HbF is a form of hemoglobin that carries oxygen, naturally present at birth, and as the infant grows, the hemoglobin in the blood converts to the adult form of hemoglobin. HbF, as a compensatory form of hemoglobin, has the potential to achieve functional cures for patients with severe hereditary blood disorders such as sickle cell disease and β-thalassemia.

▲Pipeline of MiNA Therapeutics (Source: MiNA Therapeutics official website)

MiNA Therapeutics' HbF product, utilizing the liposomal delivery technology NOV340, can effectively deliver RNAa therapy in vivo without harmful pre-treatment or complex cellular engineering. Previously, the NOV340 formulation has demonstrated its safety and pharmacodynamic activity in clinical trials involving over 290 patients, including 130 patients treated with MiNA Therapeutics’ first RNAa candidate drug, MTL-CEBPA. The company expects to advance its HbF product into preclinical development in 2024, marking the first product in its gene medicine portfolio.

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