CAMBRIDGE, Mass. & HAIFA, Israel--(BUSINESS WIRE)--Minovia Therapeutics, a clinical-stage company focused on the development of cell-based Mitochondrial Augmentation Technology (MAT) to treat mitochondrial diseases, announced today the publication of preclinical data demonstrating that mitochondrial augmentation of CD34+ cells confers functional benefit and that mitochondrial transfer occurs between hematopoietic cells in vivo. The dose-dependent ability of isolated mitochondria to enter healthy or diseased human CD34+ cells, termed mitochondrial augmentation, was demonstrated for the first time.
The peer-reviewed paper describes how mitochondrial augmentation of CD34+ cells from either healthy donors or from patient donors with mitochondrial disorders was shown to increase cellular mitochondrial content and oxygen consumption in vitro. The studies further showed that mitochondrial augmentation of patient-derived cells lead to superior engraftment of CD34+ cells in a non-conditioned NSGS mouse model, enabling 2-fold improved bone marrow engraftment relative to that of non-augmented patient-derived CD34+ cells. Furthermore, durable engraftment of augmented HSPCs and persistent mitochondrial transfer was demonstrated in a non-conditioned animal model of mitochondrial dysfunction. In this model, which enabled tracking of mitochondrial transfer from donor augmented HSPCs and their progeny to recipient cells, it was shown that mitochondrial transfer occurred in vivo for up to 4.5 months after a single infusion of augmented HSPCs. The data were published in Nature Regenerative Medicine in an online article entitled “Mitochondrial augmentation of CD34+ cells from healthy donors and patients with mitochondrial DNA disorders confers functional benefit”.
The full article can be found at: https://www.nature.com/articles/s41536-021-00167-7
The study in the Nature Regenerative Medicine publication was conducted as part of a collaboration between scientists at Minovia Therapeutics and the laboratory of Dr. Elad Jacoby, MD and Dr. Amos Toren, MD at the Sheba Medical Center, Tel Hashomer, Israel. Drs. Jacoby and Toren, of the division of pediatric hematology and oncology, are lead Principal Investigators in the Minovia-sponsored clinical trial NCT03384420.
Noa Sher, Chief Scientist at Minovia Therapeutics, said, “We believe these studies showing durability of engraftment in mitochondrially-augmented CD34+ cells and the ability and persistence of mitochondrial transfer support the potential for MAT as a disease-modifying therapy, not only for primary mitochondrial diseases, but also for hematological conditions.”
Added Natalie Yivgi-Ohana, CEO and Co-founder at Minovia Therapeutics, “We remain committed to the continued development of our technology. Most importantly, we are thankful for all of those who contributed with us – from the investigators at Sheba Medical Center, to the incredibly supportive CHAMP Foundation, to the family from Poland who donated cells from their daughter who passed away from Pearson Syndrome at two years of age – we are humbled and reminded of the importance of collaboration between industry, academia, and patient advocacy in the fight against rare diseases.”
About Minovia Therapeutics
Minovia Therapeutics is a clinical stage company and the first to use a cell therapy approach to treat patients affected with mitochondrial diseases using the Mitochondrial Augmentation Technology (MAT) platform. MAT is being developed as a robust and scalable therapeutic platform targeting the root cause of diseases caused by mitochondrial dysfunction. In MAT, patients’ hematopoietic stem and progenitor cells are enriched with healthy mitochondria isolated from an allogeneic or syngeneic source. Minovia’s initial clinical focus is on rare primary mitochondrial diseases for which there are no approved treatments in the United States and the unmet medical need is immense. Minovia has completed an IND-enabled phase I/II clinical trial in Pearson Syndrome, a fatal pediatric disease. Harnessing the power of mitochondria, Minovia is committed to exploring the full potential of its proprietary platform to treat mitochondrial diseases ranging from orphan indications to more common or age-related diseases.
For more information, please visit http://minoviatx.com/.