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)
Blindness & Regeneration image


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

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Assets, capabilities, and talents which Astellas wishes to achieve from its partners

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]

  • 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 clear 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.
  • ASP1128: This is a highly selective PPAR-delta modulator which is administered as an i.v. formulation. ASP1128 is being evaluated for the treatment of acute kidney injury (AKI) after Coronary Artery Bypass Grafting (CABG) and a POC study started in November 2019. Visit to learn more about ongoing ASP1128 clinical trials.
Mitochondria Biology image

Astellas is also evaluating several other modulators of mitochondrial function in preclinical studies for potential use in additional indications.


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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:

Assets, capabilities, and talents which Astellas wishes to achieve from its partners

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]
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.


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Message from Primary Focus Lead

Itsuro Nagase, DVM, Ph.D.
Vice President
Primary Focus Lead, Mitochondria Biology

Mitobridge has assembled a strong team with expertise in advancing ground-breaking therapeutics into the clinic. Our scientific advisors include leaders in mitochondrial biology, metabolism and aging, experienced in translating novel discoveries into next-generation medicines, help us execute on our “Boston innovation hub” concept. We are leveraging our success in development of medical products through close collaboration with our partners.


Genetic Regulation

Genetic medicines have the potential to be transformative. Mutations or deficiencies in genetic code cause nearly 7,000 human diseases* and contribute to the pathophysiology of many common diseases. New technologies to replace or regulate genes safely have advanced rapidly and are now a reality. One single treatment intervention has the potential to deliver transformative benefits to patients with few or no alternative treatment options.

Our vision and strategy aim to build capabilities for global leadership in Adeno-Associated Virus(AAV)-based genetic medicines. In January 2020 we completed the acquisition of Audentes Therapeutics, a leader in developing AAV-based genetic medicines with a focus on rare neuromuscular diseases. Audentes will become our Center of Excellence for Genetic Regulation as we aspire to address unmet medical need in patients with rare as well as more common diseases. Audentes plans to further strengthen its manufacturing capacity with a new cutting-edge gene therapy manufacturing facility in North Carolina which will become operational and GMP ready by mid-2022.

*: Online Mendelian Inheritance in Man® (OMIM®) Gene Map Statistics available at (Accessed December 9, 2020)

Our Primary Focus Genetic Regulation has been working on the below leading programs

  • AT132: A gene replacement therapy being investigated for X-linked Myotubular Myopathy (XLMTM), a rare and fatal disease caused by lack of myotubularin protein in skeletal muscle cells.
  • 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.
  • AT702/751/753: A vectorized exon skipping gene regulation therapy being investigated for the treatment of Duchenne Muscular Dystrophy (DMD), a severe progressive neuromuscular disease caused by mutations in the gene coding for dystrophin.
Genetic Regulation image

With Audentes as our Center of Excellence we have access to a pipeline of transformative therapies, including AT132 in late stage clinical development, and industry-leading manufacturing capabilities.


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Assets, capabilities, and talents which Astellas wishes to achieve from its partners 

We are growing and building for the future with the awareness that we have yet to see new disruptive innovations and meet unique and diverse talent. We are committed to further strengthening our position and are always looking for new opportunities and collaborations with partners to pursue cutting-edge science.

[Assets and capabilities]

  • 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 actively recruiting talent across the value chain of Research, Development, Manufacturing, and Commercialization at Audentes, our Center of Excellence.


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Message from Primary Focus Lead

Ulf Tollemar, 
Vice President 
Primary Focus Lead, Genetic Regulation

The current rapid advancement of AAV-based genetic regulation research and development has made genetic medicines a therapeutic reality for patients around the world. Astellas is committed to progressing our VISION of turning innovative science into VALUE for patients. Through our collaboration efforts, we are building toward leadership in research, development, manufacturing and commercialization of AAV-based genetic medicines with Audentes as our Center of Excellence.



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.

  • ASP1948: Anti-NRP1 antibody
  • ASP1951: GITR agonistic antibody
  • ASP9801: Oncolytic virus (Collaboration with Tottori University)
  • ASP7517: WT1 loaded artificial adjuvant vector cell (aAVC) (Collaboration with Riken)
Immuno-Oncology image


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Assets, capabilities, and talents which Astellas wishes to achieve from its partners

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]

  • 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.


Related Links