Astellas, a pharmaceutical company based in Tokyo, is focused on advancing cell and gene therapies to treat eye diseases as part of its long-term vision.

With millions affected globally, vision impairment from degenerative eye diseases is on the rise, largely due to an aging population.

These degenerative conditions, including age-related macular degeneration and glaucoma, lead to the loss of specific retinal cells that do not regenerate naturally.

One of Astellas' key projects is a stem-cell treatment for geographic atrophy, which is currently in phase 1b clinical trials and aims to restore vision by transplanting retinal pigment epithelial cells.

In addition to cell therapy, Astellas is exploring the use of adeno-associated virus (AAV) vectors for gene therapy, targeting both rare and common eye disorders, with promising preclinical results in glaucoma.

However, developing these innovative therapies presents numerous challenges, including scientific, technical, regulatory, and manufacturing hurdles, which require a long-term investment approach.

Safety concerns also accompany regenerative therapies, such as the risk of cell proliferation beyond intended areas and potential immune rejection, although the eye's immune privilege offers some protection.

To address safety issues, Astellas has developed 'kill switch' technology for stem cells to prevent uncontrolled proliferation and is investigating methods to make stem cells less detectable by the immune system.

Collaboration plays a vital role in Astellas' strategy, as the company partners with biotech firms, industry specialists, and academic institutions to enhance the development and manufacturing of these therapies.

Astellas is also advocating for a reassessment of efficacy criteria to better align with patient needs and improve quality of life outcomes.

Regulatory challenges are compounded by the gradual progression of eye diseases, which complicates the evaluation of treatment efficacy and the approval process.

To foster innovation in regenerative medicine, Astellas has established incubator spaces in both the US and Japan, promoting collaboration among researchers and developers.