Primary Focus Mitochondria

Mitochondria are specialized structures in cells that have their own maternally inherited DNA (mtDNA). Mitochondrial dysfunction is associated with diseases of the kidneys, liver, muscles, central nervous system, eyes and ears. Many of these diseases have significant unmet medical needs and few treatment options. By targeting mitochondria, we have the potential to create an entirely new way of treating diseases associated with mitochondrial dysfunction.


Our Approach

We leverage our deep understanding of mitochondrial biology, disease pathophysiology and our research platform to identify new leads and innovative target molecules, accelerating selection of scientifically relevant indications.

Our Approach

mtDNA: Mitochondrial deoxyribonucleic acid, ROS: Reactive oxygen species, ATP: Adenosine triphosphate, NAD+: Nicotinamide adenine dinucleotide

We expand into commercially viable indications based on Proof-of-Concept results and the addition of mitochondrial cell therapy approaches.





Partnering with Astellas

Science around mitochondrial biology is emerging. New modalities/technologies, including gene therapy, cell therapy, and mitochondrial transplantation therapy, are also evolving. Developing mitochondrial biology-based medicine is changing at a fast pace, thus, it requires a variety of capabilities. We are eager to work in partnership to develop high impact research at any location that offers the best chance of timely success.

Assets and capabilities of interest:

Astellas is interested in a broad spectrum of assets (pre-clinical and clinical), technology, and capabilities that can be used to improve patient outcomes by modulating mitochondria or related pathways. We have significant internal expertise in traditional (e.g., low molecular weight compounds, antibodies) and newly emerging medical modalities (e.g., cellular and gene therapies).  


Scientific excellence and integrity are required to be successful, but are not enough. We emphasize diversity of experience and backgrounds because this fosters creative and agile drug development collaboration. Astellas and our partners see and understand the emphasis on teamwork, communication, transparency, respect and focus. 


Message from Primary Focus Lead

Message from Primary Focus Lead

Tong Zhu, Ph.D, FCP
Primary Focus Lead, Mitochondria

Astellas has established a team with strong expertise at Mitobridge, in Cambridge, Massachusetts, and at Nanna Therapeutics, in Cambridge, United Kingdom, discovering novel therapeutics that improve mitochondrial functions and advancing them into the clinic.  We leverage a network of our expert scientific advisors who are leaders in mitochondrial biology, metabolism and aging, and experienced in translating novel discoveries into next-generation medicines to help us execute our “innovation hub” concept in both the US and UK. We leverage our success in the development of medicinal products, use innovative development strategy to achieve rapid proof of concept in one disease and then expand into more diseases maximizing the value of our projects for patients. We partner with patient organizations so that we can incorporate patient insights in our product development – in order to deliver on our patient and science-first mindset.

For reference: the following links show us the importance of our research and development in this Primary Focus area to improve the health and quality of life for patients:


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