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Pharma, Biotech Partner on Off-the-Shelf Cell Therapies

Mitochondria illustration

Mitochondria (Charlie Jones, Pixabay. https://pixabay.com/illustrations/mitochondria-cell-biology-science-3016868/)

30 July 2021. A biotechnology enterprise is teaming with a pharmaceutical company to develop off-the-shelf therapies for malfunctioning mitochondria, energy centers of cells. The collaboration and licensing deal with Astellas Pharma Inc. in Tokyo is expected to bring biotechnology company Minovia Therapeutics Ltd. in Haifa, Israel $20 million up-front and $420 million per product developed from the collaboration.

Minovia Therapeutics develops therapies for diseases caused by dysfunctional mitochondria, the energy components of cells. Mitochondria convert oxygen and nutrients to adenosine triphosphate, the chemical providing energy to power a cell’s metabolism. Deterioration in mitochondria can be caused by inherited or environmental factors, and result in errors in mitochondrial DNA, leading to a range of diseases, particularly with advancing age.

Minovia designs treatments for mitochondrial disorders that aim to restore proper functioning of cells’ energy components. The company’s technology, called mitochondrial augmentation, extracts mitochondria from cells donated by healthy individuals. At the same time, blood-forming stem cells are taken from patients’ blood with apheresis, which uses a centrifuge to separate various blood components. From the separated components, Minovia extracts a certain type of progenitor stem cell known as 34+ that transforms into blood-forming colonies in bone marrow. The patient’s CD34+ stem cells are then combined with donated healthy mitochondria, and infused back into the patient’s blood stream.

Therapies derived from transferred healthy mitochondria

Astellas is already exploring cell therapies, including for mitochondrial disorders, through its Institute for Regenerative Medicine and subsidiary company Universal Cells Inc. in Seattle. Naoki Okamura, Astella’s chief strategy officer, notes in a company statement that Astellas has “positioned mitochondrial biology as one of the primary focuses of our research and development strategy to develop therapies for patients with unmet medical needs. One of the aspirations of this primary focus is to establish a mitochondrial cell therapy platform.”

Minovia Therapeutics and Astellas plan to work together on creating allogeneic or off-the-shelf mitochondrial cell therapies. The companies aim to research treatments derived from Astellas’s genetically engineered, induced pluripotent stem cells, also known as adult stem cells, supplemented with Minovia’s mitochondrial augmentation technology. The goal is therapies for mitochondrial disorders derived from transferred healthy mitochondria.

Under the agreement, Astellas is paying Minovia Therapeutics $20 million in an initial payment. Minovia is also eligible for another $420 million in future development, regulatory, and commercial milestone payments for each new mitochondrial therapy product developed by Astellas from the collaboration. The announcement gave no number of programs covered by the agreement.

“We share with Astellas both their passion for mitochondrial science and their commitment to patients in need of new therapies,” says  Minovia’s co-founder and CEO Natalie Yivgi-Ohana. “As Minovia continues the development of mitochondrial augmentation therapy, we believe this partnership is critical to accelerate the development of off-the-shelf, allogeneic cell therapy programs for the many patients living with mitochondrial diseases caused by mitochondrial dysfunction.”

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