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Santen Cell & Gene: Changing the Lives of Patients Suffering from Serious Ophthalmic Diseases


Sponsored article provided by Santen

Founded in Japan over a century ago, Santen has been contributing to people’s eye health for the past 130 years. During this time the company has expanded to over 60 countries, reaching the number 1 position in the Japanese and Chinese ophthalmic markets. As a company dedicated to ophthalmology, and as part of our vision towards 2030, we aim to build our capabilities and leverage new technological advancements to reduce the loss of social and economic opportunities due to eye conditions. 

In 2021, Santen established Santen Cell & Gene, a global business unit based in Switzerland, dedicated to changing patient lives through transformative solutions. With its own specialized, dedicated team, and platform that covers all advanced therapy capabilities through the entire value chain, Santen Cell & Gene aims for a global leadership position in ophthalmic cell and gene therapies (CGTs). We strive to leverage Santen’s deep expertise and footprint as an established company, while maintaining the agility and flexibility required to succeed in the dynamic CGT space.

Cell and Gene Therapies have the potential to address significant unmet needs in ophthalmology. Inherited retinal diseases (IRDs), for example, are a group of (relatively common) rare eye diseases affecting between 1 in 2000[1] and 1 in 3000 people[2]. While they represent the primary cause of vision loss in people of working age, and a common cause of visual impairment in childhood[3], there are very few approved treatments for these diseases. At Santen Cell & Gene, we see the opportunity to develop breakthrough therapies that will change the lives of patients suffering from IRDs.

Ophthalmology is a particularly interesting area for CGTs, offering several unique advantages. We aim to take advantage of the immune privilege that the eye offers, which reduces the risk of immune response. Additionally, since most target cells for ocular gene therapy do not renew or mutate, the effects of the therapy have the potential to last longer compared to other areas of the body. There are also a number of gene mutations associated with ocular diseases- for example, around 100 different genes have been found to cause retinitis pigmentosa alone[4], one of the most common IRDs. This provides us with a multitude of options to treat ocular diseases.

Santen Cell & Gene is currently working on the development of a human retinal progenitor cell therapy (hRPC therapy), a potential gene-agnostic treatment for retinitis pigmentosa, for which we in-licensed the rights to development and commercialization for territories outside the US in 2020.  At the same time, as a global business unit dedicated to CGTs, we aim to build a portfolio of assets addressing some of the greatest unmet needs in ophthalmology. We are focusing on finding solutions for patients who have no alternative treatment options such as in IRDs, optic neuropathy and severe corneal dystrophy. We operate on a business model based purely on open innovation, as we believe it is by collaborating with those at the forefront of innovation that we can deliver transformative therapies. We are striving to become a partner of choice in ophthalmic cell and gene therapies, offering the unique combination of a global footprint and over a century of expertise in ophthalmology, along with the specialization of a dedicated business unit.

At Santen, we often refer to a quote from the World Healthy Organization (WHO) World Report on Vision: “the world is built on the ability to see”. Eye diseases are often not life-threatening, and as the CGT sector initially developed, may not have received the same level of attention as other areas such as oncology. However, good vision is critical to many aspects of everyday life. This is demonstrated through the enormous financial burden they represent: IRDs are estimated to cost the US and Canadian economies alone up to USD 33 billion annually[5]. The Santen Cell & Gene team is inspired and guided by the potential of CGTs to change the lives of patients suffering from serious or rare ophthalmic diseases.  




[4] Moore SM, Skowronska-Krawczyk D, Chao DL. Targeting of the NRL Pathway as a Therapeutic Strategy to Treat Retinitis Pigmentosa. J Clin Med. 2020 Jul 13;9(7):2224. doi: 10.3390/jcm9072224. PMID: 32668775; PMCID: PMC7408925.