Translating scientific insights into therapies

Dear Readers, Two major developments in the eye-care sector had converged in 2017 when we decided to set up the Institute of Molecular and Clinical Ophthalmology Basel (IOB): First, technological and scientific advances made it possible to gain a deeper understanding of the genesis of many eye diseases and work on new treatments. Second, Basel had the academic, personnel and industrial infrastructure to close the gap between basic and clinical research, which had been growing apart over the years given the absence of convincing medical breakthroughs. The University of Basel, the University Hospital Basel, and Novartis took the opportunity to create an institute which would bridge this gap. The goal was to increase the touch points between scientists and clinicians and take advantage of the recent breakthroughs in genetic research and organoid science. Combined, these developments provided the basis for much-needed new insights in the ophthalmology space that had lagged the innovation power of other scientific fields. The venture has proved a success so far. The IOB not only made rapid progress in its research and clinical efforts. The institute also carved out a leading position within the scientific community, becoming one of the pre-eminent translational research centers in the world. Since its foundation, the IOB has attracted more than 140 scientists from over 30 countries, won almost 80 awards and has increased its publication output from 13 papers in 2018 to over 500 in 2023. More importantly, its research strategy of focusing on disease areas that lend themselves to the development of new treatments has proven the right way to go. Not even seven years after launching its activities from a small office in Basel with a few people, the IOB has already spun out one of its first key research projects in the realm of cone optogenetics, which will be pursued further by RhyGaze AG and has received fresh funding from venture capital companies. Other projects may follow suit in a similar or different form – but all with the view to developing efficient treatments for patients with impaired vision or vision loss. There is a major need for this work. According to the World Health Organization, more than 2 billion people worldwide have a near or distance vision impairment, triggering annual global costs of more than 400 billion US dollars. According to a survey published in the International Journal of Ophthalmology, doctors cited wide-spread eye conditions such as dry age-related macular degeneration, wet age-related macular degeneration, glaucoma, diabetic macular edema, and dry eye among the biggest immediate medical needs. But there are many other diseases for which the medical community has found no answer to date. Many of these conditions lead to blindness, signifying a devastating verdict for the patients and their families. And although some rare, hereditary forms of blindness can be treated today with the help of novel gene therapies, the field has only made its first, very humble steps. The scientific community will need to up its efforts forcibly to develop innovative treatments that can make a visible difference for patients. The IOB has what it takes to make an impact. Currently, more than seven groups at IOB are working on an array of research projects that include novel approaches to tackle myopia, Stargardt disease, retinal dystrophy, and Usher syndrome, to name but a few. At the same time, the IOB is also working hard to develop new technologies that can help accelerate the development of new treatments. Among others, it has worked on state-of-the-art organoid, protein engineering and virus platforms, which are crucial to gain a deeper understanding of disease mechanisms. The speed at which the IOB is working reflects the dedication of its scientists and clinicians, a diverse team of high-caliber researchers ranging from computer scientists and optical engineers to clinical experts who have daily contact with patients and their immediate needs. It is this combination that makes the IOB stand out within the ophthalmology field, which is receiving important impulses from the efforts undertaken here in Basel’s Klybeck quarter – once the headquarter of Novartis predecessor Ciba. Looking ahead, I am convinced that the IOB will create substantial value over time, not only in the realm of ophthalmology. It will also benefit the Basel research community and Switzerland in general. Much like the foundation of the Friedrich Miescher Institute in 1970, when Novartis predecessor companies Ciba and Geigy combined their forces with the University of Basel to build what is today one of the foremost research institutes in Europe, the IOB will contribute to the country’s leading research position and uncover insights that can be translated into novel treatments. Joerg Reinhardt Chair of the Board of Directors of Novartis (until March 2025)