Developing transformative therapies to restore sight and free patients from the fear of deteriorating vision

Astellas is a global life sciences company committed to turning innovative science into VALUE for patients. We provide transformative therapies in disease areas that include oncology, ophthalmology, urology, immunology and women's health. Through our research and development programs, we are pioneering new healthcare solutions for diseases with high unmet medical need.

Through our efforts in Primary Focus (PF) Blindness and Regeneration, we are committed to developing transformative therapies with the potential to protect or restore sight and free patients from the fear of deteriorating vision. To deliver on this, we have established an industry-leading ophthalmology pipeline with cell and gene therapies and other modalities in development across eye diseases where there are limited or no treatment options, including geographic atrophy (GA), Stargardt disease, retinitis pigmentosa, and glaucomatous optic neuropathy.

Working to deliver treatments through next-generation technologies
Recognizing the significant challenges of delivering entirely new modalities, we are pursuing a long-term cell and gene therapy strategy to pioneer new treatments for patients. We are establishing a robust and differentiated regenerative medicine platform combining our deep understanding of the biology of eye diseases with cutting-edge cell and gene modalities.

Our differentiated platform technologies include:

  • Pluripotent stem cell (PSC): we are leading the development of the field’s first allogeneic cell therapy with the potential to restore vision.
  • Universal Donor Cell (UDC): our industry-leading universal donor cell technology works like an immune cloaking system to ensure cell therapies evade immune rejection. It could be applied to cell therapies to potentially create ‘off the shelf’ treatments without the need for immunosuppression.
  • Adeno-associated virus (AAV):AAV-based gene therapy offers the possibility of preserving sight for people with glaucomatous optic neuropathy, currently a leading cause of irreversible blindness.

Building a broad, differentiated pipeline of potentially transformative treatments

With a pipeline of several regenerative medicine programs, we have the potential to transform the treatment of multiple eye diseases.

We have several candidates in development across multiple eye diseases:

  • Our lead cell therapy candidate, ASP7317 (retinal pigment epithelium allogeneic cell transplantation), is in clinical development for the treatment of geographic atrophy secondary to age-related macular degeneration and Stargardt disease. This is the first ophthalmic allogeneic pluripotent stem cell-based therapy to enter the clinic and is currently in Phase 1b development.
  • Our preclinical compound, ASP2767, is an AVV-based gene therapy for the treatment of glaucomatous optic neuropathy offering the potential to preserve sight in glaucoma.
  • ASP2020 is another preclinical compound using our universal donor cell-derived allogeneic cell therapy. It aims at treating macular dystrophies by replacing retinal pigment epithelium to restore lost sight.

It is our intent that delivering cell and gene treatments for the eye will be the beginning of, and the foundation for, a continuum of innovation that could potentially benefit patients across many high unmet need disease areas.

Harnessing the power of collaboration

Pioneering new treatments and utilizing new modalities cannot be achieved in isolation. Working collaboratively with experts from fields including ophthalmology, transplantation and regenerative medicine, we have greater potential to succeed. Alongside our collaborations with leading industry scientists and academic partners, we also make intelligent connections across Astellas, identifying expertise to further strengthen our approach.

For more information on our R&D approach and pipeline, please visit: https://www.astellas.com/en/innovation/primary-focus-blindness-regeneration.