RARE Daily

MiNA Reports Pre-Clinical Data Shows Successful in Vivo Delivery of RNA Therapy for Rare Blood Diseases

January 24, 2024

Rare Daily Staff

MiNA Therapeutics said new pre-clinical studies confirmed in vivo delivery of its small activating RNA therapeutics to stem cells in bone marrow that give rise to red blood cells at levels that are clinically meaningful for the treatment of beta-hemoglobinopathies.

The data is being presented in a poster on January 24 at the Keystone Symposia’s Delivery of Nucleic Acid Therapeutics Conference in Banff, Alberta, Canada.

MiNA’s HbF program is designed to increase transcription of the gamma globin (HBG) gene, enabling patients with beta-hemoglobinopathies to produce enhanced levels of HbF. HbF is a compensatory form of hemoglobin which has the potential to achieve a functional cure in patients with severe inherited blood disorders such as sickle cell disease and beta thalassemia.

“These results demonstrate for the first time efficient in vivo delivery of small activating RNA (RNAa) therapeutics to erythroid progenitor cells with a delivery technology that is well-suited for safe and effective treatment in the clinic,” said Robert Habib, CEO of MiNA Therapeutics. He said the data support advancement of the company’s lead program for the treatment of sickle cell disease and beta thalassemia.

MiNA’s HbF program uses a liposomal delivery technology, NOV340, which efficiently delivers RNAa therapeutics in vivo without the need for pre-conditioning or complex cell engineering. NOV340 formulations have established safety and pharmacodynamic activity in clinical trials involving more than 290 patients, including 130 patients treated with MiNA’s first RNAa development candidate, MTL-CEBPA.

MiNA anticipates advancing its HbF program, the first program to emerge from its genetic medicine portfolio, into pre-clinical development in 2024.

In the presentation, data from animal models confirmed in vivo delivery to erythroid progenitor cells at levels that met accepted benchmarks for impactful treatment of beta-hemoglobinopathies. In non-human primates, intravenous administration of encapsulated liposomes resulted in delivery of MiNA’s RNAa compound to more than 60 percent of committed colony-forming unit erythroid cells and pro-erythroblasts in bone marrow. Equivalent levels of delivery efficiency were observed in peripheral blood monocytes, a cell type in which pharmacodynamic activity of NOV340-formulated RNAa therapeutics has previously been shown in clinical studies.

MiNA has previously achieved clinical proof of concept for its RNAa therapeutics platform with MTL-CEBPA. MiNA is exploring out-licensing opportunities for MTL-CEBPA and its immuno-oncology portfolio, which combines the capability to restore or boost any dysregulated gene target with clinically validated in vivo delivery to myeloid immune cells.

Photo: Robert Habib, CEO of MiNA Therapeutics


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