Based on a Focus Area approach, we concentrate our R&D investments on Primary Focus, and work to develop innovative treatments.
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 1b/2)
- 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) ]
- Optogenetics (ASP1361, preclinical)
- Next generation gene therapy targeting retinal diseases (preclinical)
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
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 has the potential to improve function for Duchenne Muscular Dystrophy (DMD) and Primary Mitochondrial Myopathy (PMM) patients. (Visit clinicaltrials.gov to learn more about ASP0367 clinical trials).
- 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 clinicaltrials.gov to learn more about ongoing ASP1128 clinical trials).
Astellas is also evaluating several other modulators of mitochondrial function in preclinical studies for potential use in additional indications.Close
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 https://www.omim.org/statistics/geneMap (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.
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.Close
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)
We are Building a Pipeline of Multi-Functional Modality Platforms