Blindness & Regeneration

Our mission for Primary Focus Blindness & Regeneration is to free patients from the fear of vision loss, and offer the hope of recovery of lost sight. By taking advantage of next-generation modalities such as cell therapy and gene therapy for patients with back of the eye diseases at high risk of blindness, we will provide new treatment options to restore and maintain vision. Cell therapy and gene therapy will enable us to meet unmet medical needs that were difficult to meet with existing therapeutic approaches. It has the promise of delivering safe, effective, and sustainable options for patients who previously have not had any treatment options.

Our capabilities range from research, CMC and development of pluripotent stem cell (PSC)-based cell therapy, Universal donor cell technology, design and CMC of adeno-associated virus (AAV)-based gene therapy, in ophthalmology area. Astellas can be uniquely positioned as an innovative company, armed with both cell-based medicine and virus-based medicine as core strengths.

Our Primary Focus Blindness & Regeneration has been working on the below leading programs.

[Human pluripotent stem cell (PSC) -derived cell therapy programs]

  • Retinal pigment epithelial cell (ASP7317, Phase 1)
  • Photoreceptor, retinal ganglion cell, corneal endothelial cell (preclinical)
  • Universal donor cell-based retinal pigmental epithelial cell (preclinical)

[Gene therapy programs based on adeno-associated virus (AAV) ]

  • Next generation gene therapy targeting retinal diseases (preclinical)
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In addition, Astellas has the following capabilities:

  • Creation, manufacturing and development of PSC-derived differentiated cells for therapeutic 
  • Universal donor cell technology
  • Creation of next generation cell products using gene-editing technology
  • Design and manufacturing of AAV-based gene therapy product
  • Preclinical evaluation of cell and gene therapy candidates

Click on the banner below to see the Primary Focus summary.


Partnering with Astellas: new assets, new capabilities and new talent

Astellas continuously pursues innovative opportunities, which are next-generation cell therapy created by gene-editing technology or universal donor cell technology, an evolved gene therapy, oligonucleotide-based therapy, and EV-based therapeutics utilizing Astellas’ cell therapy foundation.

Assets and capabilities of interest:

  • Cell therapy, gene therapy and oligonucleotide therapy for posterior eye diseases/corneal diseases (preclinical, clinical)
  • Genetic regulation and nucleic acid drugs (preclinical, clinical)
  • Technology to enable less-invasive, easy and efficient delivery of drugs into the eye
  • Technology to improve CMC of cell, gene, oligonucleotide and extracellular vesicle (EV)
  • Safer adeno-associated virus vectors


  • Talents who have extensive experience in R&D and CMC in cell and gene therapy


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Message from Primary Focus Lead

Jotaro Suzuki, Ph.D
Vice President
Primary Focus Lead, Blindness and Beyond

At Astellas Institute for Regenerative Medicine (AIRM) in Boston, research, development and manufacturing teams are closely collaborating to accelerate the realization of cell therapy. At the Tsukuba Research Center in Japan, our research unit conducts drug discovery research focusing on gene therapy in collaboration with academia and bioventure companies worldwide. Additionally, our expert team of biomanufacturing works on manufacturing of AAV-based gene therapy at the Tsukuba Biotechnology Research Center in collaboration with Audentes, our center of excellence for gene therapy. Astellas has its core development functions in Chicago, and we are working on the development of innovative drugs by collaborating with various partners around the world. Astellas is strengthening our ability to turn innovative science into VALUE for patients by flexible combination of capabilities existing both inside and outside the company.


Mitochondria Biology

Health is Our Passion and Priority. Astellas’ VISION is to turn innovative science into VALUE for patients. We are committed to making a difference for our patients today and providing them with additional therapeutic options for a brighter future tomorrow.

Our target, the mitochondrion, is remarkably complex. While it is primarily thought of as an energy supplying organelle, it also has multifaceted functions and is controlled by multiple biological pathways. In fact, mitochondria play an important role in the aging process with many clinical and preclinical evidence, and mitochondrial dysfunction causes and/or aggravates a wide variety of diseases. 

Our goal in Primary Focus Mitochondria Biology is to become the global leader in discovering, developing and commercializing mitochondrial biology-based medicine that provides VALUE for patients, clinicians and healthcare systems.

To achieve the goal, Astellas acquired Mitobridge in January 2018 and established it as an Astellas Company based in Cambridge, Massachusetts. Building upon the emerging scientific findings linking mitochondrial function with disease pathophysiology, Mitobridge and Astellas are advancing innovative mitochondrial approaches to the treatment of diseases with high unmet medical needs.

Astellas continues to prioritize investment in the following fields.

  • ASP0367: This is a highly selective Peroxisome Proliferator-Activated Receptor (PPAR) delta modulator with oral formulation. It stimulates mitochondrial respiration through increased fatty acid oxidation-related genes and mitochondrial biogenesis. Thus, ASP0367 is being investigated as a treatment to improve function for Duchenne Muscular Dystrophy (DMD) and Primary Mitochondrial Myopathy (PMM) patients. We have started dosing the first patient in its Phase 1 clinical trial for DMD patients in March 2021, and its pivotal Phase 2/3 clinical trial for PMM patients in June 2021. Visit to learn more about ASP0367 clinical trials. Also, visit the United Mitochondrial Disease Foundation’s website to learn more about the clinical trial for the patients with PMM.
  • ASP8731: Sickle cell disease is a genetic disorder caused by a point mutation in the β-globin subunit resulting in hemoglobin S. This is a highly potent, selective small molecule inhibitor of BTB and CNC homolog 1 (BACH1) capable of activating the nuclear factor erythroid 2-related factor 2 (Nrf2) pathway and inducing fetal hemoglobin in treatment of sickle cell disease. We presented preclinical data of ASP8731 at the 63rd American Society of Hematology Annual Meeting in December 2021, and at Mitochondria-Targeted Drug Development Summit in March 2022. Visit  to learn more about ongoing ASP8731 clinical trial.
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Astellas continues to evaluate several other modulators of mitochondrial function in preclinical studies for potential use in additional indications, and also to expand mitochondrial research capabilities. Astellas acquired Nanna Therapeutics in April 2020, through which Astellas acquired a unique phenotype-based screening platform as well as rapid creation and testing capacities of new chemical libraries. Astellas announced the formation of a strategic collaboration with Minovia Therapeutics, Ltd. in July 2021 to focus on novel mitochondrial cell therapies.


Click on the banner below to see the Primary Focus summary.


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:

Partnering with Astellas: new assets, new capabilities and new talent

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 tools (pre-clinical and clinical) 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 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.


Tong Zhu, PhD, FCP

Message from Primary Focus Lead

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

Astellas has established a team with strong expertise at Mitobridge, in the Cambridge, Massachusetts, and at Nanna Therapeutics, in the Cambridge, United Kingdom, discovering novel therapeutics that improve mitochondrial functions and advancing them into the clinic. 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 leverage a network of our scientific advisors who are leaders in mitochondrial biology, metabolism and aging, experienced in translating novel discoveries into next-generation medicines, help us execute on our “innovation hub” concept in both US and UK. We partner with patient organizations incorporating patient insights in our product development.


Genetic Regulation

Genetic medicines have the potential to be transformative. Genetic deficiencies define almost 7,000 different diseases1 and contribute to the pathophysiology of many common conditions. New technologies to replace or regulate genes safely have advanced rapidly and are now a reality. By targeting disease at the genetic level, we have the potential to develop life-changing therapies for patients who currently have limited or no effective treatment options.

* FAQs About Rare Diseases. Genetic and Rare Diseases Information Center (GARD). Available at Last accessed March 2022.

Our mission at Astellas, through Primary Focus Genetic Regulation and Astellas Gene Therapies, our Gene Therapy Centre of Excellence, is to discover, develop and deliver meaningful gene-based therapies for patients with genetic diseases. Our principal focus is building a portfolio of adeno-associated virus (AAV)-delivered gene therapies for the treatment of rare neuromuscular and central nervous system (CNS) diseases. We continue to enrich our capabilities and pursue new areas of gene therapy science, working to bring our lead therapies to the clinic and expand our diverse pipeline of potentially life-changing treatments.

Our most advanced gene therapy programs are:

  • AT132: (currently on clinical hold) A gene replacement therapy being investigated for X-linked Myotubular Myopathy (XLMTM), a rare and fatal disease caused by a lack of myotubularin protein in skeletal muscle.
  • AT845: A gene replacement therapy being investigated for Pompe Disease, a severe progressive neuromuscular condition caused by lack of acid alpha-glucosidase (GAA) enzyme activity leading to accumulation of glycogen in skeletal and cardiac muscle.

Astellas Gene Therapies is developing a pipeline of potentially transformative therapies and building industry-leading manufacturing capabilities.

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You can also click on the banner below to learn more about Primary Focus Genetic Regulation.


Partnering with Astellas: new assets, new capabilities and new talent

The rapidly evolving field of gene therapy cannot be navigated alone. We collaborate with world-renowned academic and industry partners to overcome the complex challenges of gene therapy research and development. We continually investigate and assess new partnerships that could enhance our diverse portfolio, as well as those that can offer unique technology or research perspectives to better support patients.

We are actively seeking partnerships that enhance our research-to-Investigational New Drug (IND) application stage portfolio, as well as those that can solve fundamental issues associated with gene therapies, such as novel gene constructs, better delivery vectors and technologies that enable re-administration.

Assets and capabilities of interest:

  • Novel project concepts and AAV constructs with potential for transformational VALUE to patients
  • New novel technologies for gene regulation and RNA editing
  • New efficient viral vectors and delivery technologies
  • Improved AAV manufacturing processes
  • Brain delivery for AAV
  • Technologies to enable AAV re-dosing and treatment of patient with pre-existing immunity

We are also keen to hear from organizations with clinical stage gene therapy programs, who might be interested to partner with Astellas and benefit from our in-house expertise and resources to bring their new medicines to patients.

Astellas is actively recruiting talent across the gene therapy value chain including research, development, manufacturing and commercialization.


Richard Wilson

Message from Primary Focus Lead

Richard Wilson
Senior Vice President
Primary Focus Lead, Genetic Regulation

At Astellas, our goal is to push the boundaries of what is possible to deliver breakthrough, first-of-their-kind gene therapies with life-changing VALUE for patients across many disease areas. We are fully committed to transforming the outlook for patients with genetic diseases and the people who care for them.

The rapid advancement of AAV-based genetic regulation research and development has made genetic medicines a therapeutic reality for patients around the world. We are at the beginning of an exciting new era in genetic medicine.

In partnership with leading academic and industry collaborators, we intend to become leaders in the research, development, manufacturing and commercialization of AAV-based genetic medicines. We are building a gene therapy powerhouse with global capabilities to deliver for patients in need and to be a partner of choice for innovators in the field. Astellas is committed to progressing our VISION of turning innovative science into VALUE for patients.



Our mission is to discover, develop and deliver the best innovative cancer medicines to patients and ultimately, cure cancer. We are activating our best innovative capabilities and building a strong network of collaborations with external partners, in order to complete our mission.

Despite the approval of multiple novel cancer treatments, such as immune checkpoint inhibitors, over the past few years, significant unmet needs still exist. Many cancer patients do not respond to a given treatment (“refractory”) or fail to maintain a response (“relapse”) during the treatment, with as many as 80 percent of patients estimated to be refractory to immune checkpoint inhibitors or to relapse during the treatment.

Our strategy is to target multiple aspects of the immune response to the cancer simultaneously.

Our Primary Focus Immuno-Oncology has been working on the below leading programs

We have established a robust and competitive development, and research stage Immuno-Oncology portfolio through internal efforts and strategic external collaborations. We have built internal discovery, protein engineering, pre-clinical and clinical development, translational sciences and manufacturing capabilities in the US (greater Boston area, Seattle, Washington, South-San Francisco, California, and Northbrook, Illinois) and in Tsukuba, Japan. We have also formed collaborations with leading academic institutions and biotech companies worldwide to develop unique therapeutic approaches in Immuno-Oncology.

  • ASP9801: Oncolytic virus (Collaboration with Tottori University)
  • ASP7517: WT1 loaded artificial adjuvant vector cell (aAVC) (Collaboration with Riken)
Immuno-Oncology image


Click on the banner below to see the Primary Focus summary.


Partnering with Astellas: new assets, new capabilities and new talent

We are building multi-functional modality platforms, which can potentially deliver meaningful impact on cancer. We originate our research from deep knowledge of cancer biology and expertise in multi-functional modality discovery, development and commercialization. We are looking to further expand and maximize our Immuno-Oncology capabilities through additional partnerships and acquisitions – sourced via our highly-active and well-coordinated external innovation, venture and business development teams, strategically located in Cambridge, Massachusetts, the Bay Area, and in Tokyo, where biotech and life science innovation is booming. 

Assets and capabilities of interest:

  • Novel immune checkpoint
  • Oncolytic virus
  • Cancer vaccination (Artificial Adjuvant Vector Cells, aAVC)
  • Cancer Cell therapy
  • Bispecific Immune Cell Engager
  • Combinations across our pipeline

We are also extending our talented teams continuously. If you are interested in working in progressive, innovative teams, in an entrepreneurial diverse environment, please contact us.

We are allocating significant, sustained investment in understanding cancer biology and deploy this knowledge to establish multi-functional modality platforms and generate a broad, novel pipeline

We are Building a Pipeline of Multi-Functional Modality Platforms

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Message from Primary Focus Lead

Peter Sandor, M.D. 
Senior Vice President 
Primary Focus Lead, Immuno-Oncology

I believe that cancer can and will be defeated. Success requires determination, partnership and collaboration between the best minds in cancer research and development. We are building teams and a collaboration network, which is determined to make a difference for cancer patients to provide novel, innovative medicines and potentially cure cancer. We have made a strong commitment to Immuno-Oncology. We believe that our competitive strategy will be successful in developing the Immuno-Oncology programs already in the clinic as well as our future pipeline compounds, which will help to address unmet patient needs. We continue to expand our network and contribute to partnerships with our knowledge of science and drug development.


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