| | DECEMBER 2021 9"descendant" of stem cells derived from fetal retinas. They release neurotrophic factors that restore cellular function, including that of diseased photoreceptors and neurons, and have the ability to differentiate under certain conditions. This means they have a huge potential in a number of degenerative retinal diseases, including retinitis pigmentosa, a rare inherited eye condition caused by various genetic defects, and one of the leading causes of inherited blindness. The main risk associated with allogeneic therapies is immune rejection. In some indications (where there is exposure to the systemic immune system), an immunosuppressant may be administered in order to reduce immune response. For such conditions, the use of autologous therapies means that the invasive procedures required to obtain cells from the patient undergoing the treatment is justified.However, the eye is one of the few sites in the body that possesses a degree of immune privilege. While this privilege is not absolute and depends on a number of factors, including an intact retinal pigment epithelium monolayer, the risk of ocular immune response has been shown to below, giving allogeneic stem cell therapies the potential to be safe to use in a wide variety of ophthalmic indications. Compared to their autologous counterparts, allogeneic therapies generate a number of benefits owing to their "off-the-shelf" characteristic. They rely on limited, sometimes single sources of cells that can be replicated many times, frozen, and subsequently used to treat large numbers of patients. This gives rise to considerable potential economies of scale in addition to the greatly reduced treatment burden upon the patient.In addition, the administration procedure in allogeneic therapies has the potential to be minimally invasive, such as with intravitreal delivery when administering hRPCs to treat retinal disorders where neurotrophic factors are relied upon as the mechanism of action.Santen, Advanced Therapies and Achieving Happiness Through VisionSanten is a global specialist in ophthalmology, committed to reducing the loss of social and economic opportunities for people around the world due to eye conditions. We have a commercial presence in over 60 countries and a portfolio that includes both over-the-counter and prescription medicines as well as medical devices. In 2020, we made the first of what we hope will be many investments in the advanced therapy field, through the conclusion of our licensing agreement for an investigational hRPC therapy in late-stage clinical development. If approved, it has the potential to positively impact the condition of patients with retinitis pigmentosa and potentially other retinal degenerative diseases. To support our entry into the field, we are building a business model that combines the agility of a start-up with Santen's expertise and heritage in order to develop innovations at a pace that can benefit society and transform lives. While our hRPC therapy remains our primary focus, we are also building a longer-term strategy focused on developing a pipeline of advanced therapies. Our new expert team will maintain an entrepreneurial and agile mindset so that we can accelerate our hRPC therapy program and build a successful and sustainable platform in this field. We see this addition to our pipeline as a significant step in achieving our world vision of creating happiness around the world through vision. Entering the advanced medicines space will allow us to develop and commercialize therapies that can address important unmet medical needs in ophthalmology, ultimately moving us towards our goal of creating a brighter future and better life for patients impacted by rare and sight-threatening diseases. Gil CarrasquinhoCompared to their autologous counterparts, allogeneic therapies generate a number of benefits owing to their "off-the-shelf" characteristic
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