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Genetic deficiencies define almost 7,000 different diseases* 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.
Our mission at Astellas, through Primary Focus Genetic Regulation, is to discover, develop and deliver meaningful gene-based therapies for patients with genetic diseases. We lead with a science-first approach using a focused set of criteria to select target indications, which we believe increases the chances of success for each of our programs, including a current focus on monogenic diseases.
Combining our deep understanding of disease biology and adeno-associated virus (AAV)-delivered gene therapies, our focus is on developing treatments for rare neuromuscular and central nervous system (CNS) diseases. Our ambition is to expand to other organs and more common diseases. We leverage our end-to-end capabilities and pursue new areas of gene therapy science to bring our lead therapies to the clinic and expand our diverse pipeline of potentially life-changing treatments.
Our Flagship program under Genetic Regulation is AT845 – an investigational gene replacement therapy in development for Pompe Disease. Pompe Disease is 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.
Please view our pipeline for Genetic Regulation below. You can also access the full Astellas pipeline page here.
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 keen to hear from organizations that might be interested in partnering with Astellas and could benefit from our in-house expertise and resources to bring their new medicines to patients.
To learn more about how to collaborate with Astellas, please visit our Partnering page.
Astellas strives to match the best science and the best talent in order to deliver on our mission to turn innovative science into VALUE for patients. We encourage you to visit our Careers page to learn how you can join our work to turn innovative science into VALUE for patients.
