Working collaboratively, we are harnessing cutting-edge methodologies to realize the full potential of our proprietary MAT platform
Based on the proven ability of isolated mitochondria to enter cells, we have researched and developed our science of mitochondrial augmentation into a therapeutic platform: Mitochondrial Augmentation Therapy (MAT), an investigational proprietary cell therapy approach to treat the root cause of diseases caused by mitochondrial dysfunction.
Mitochondrial Augmentation Therapy enriches the patient’s autologous hematopoietic stem and progenitor cells in patients with healthy mitochondria isolated from an allogeneic or syngeneic source.
We have demonstrated, using a variety of visual, molecular, and biochemical methodologies in a preclinical setting that syngeneic or allogeneic mitochondria can enter cells in a dose dependent manner and transfer to neighboring cells.
GFP labeled mitochondria
in CD34+ cells after 8 hours
In the clinical setting, we perform MAT on autologous Hematopoietic Stem and Progenitor Cells (HSPCs). These are the progenitors of every blood and immune cell in the body - both the innate and adaptive immune systems, cells of both myeloid and lymphoid lineages. As stem cells, they are long-lived; as hematopoietic cells, their daughter cells circulate systemically and reach every organ system in the body, a key factor when addressing multi-systemic diseases.
Scientific literature supports the use of HSPCs to treat not only hematopoietic, but also non-hematopoietic disorders. For example, bone marrow transplant (BMT) is used to alleviate brain dysfunction in diseases such as Hurler’s Syndrome. In addition, allogeneic HSCT (HSC transplantation) using bone marrow or umbilical cord blood as the source for HSPCs has been shown to alleviate hematopoietic, but also brain, kidney and liver dysfunction of mitochondrial disease in preclinical and clinical studies. Together, these provide the scientific rationale for using HSPCs to target systemic alleviation of mitochondrial disease associated symptoms. However, due to the associated morbidity and mortality, allogenic HSCT is not generally indicated for patients with mitochondrial disease. The power of MAT is that it can be performed using autologous HSCTs, affording the benefit of a HSPC-based therapy without the associated allogeneic risks.