Age-related macular degeneration (AMD) is the most common cause of irreversible blindness in the western world—affecting millions of people in the U.S. alone1. While anti-VEGF therapeutics have transformed the treatment of the wet form of AMD, most patients have the dry form of the disease — for which there are no approved therapies. As dry AMD progresses, regions of the retina are damaged, and eventually patches of atrophy can develop and expand resulting in irreversible vision loss. Since AMD typically affects central vision, which is used to read, drive a car or recognize the faces of loved ones — the impact on quality of life can be significant. Patients with certain genetic variants may have a high risk of faster disease progression and subsequent vision loss (and may also be at risk of developing systemic morbidities).
AMD is a complex disorder, with genetics, lifestyle and environmental factors influencing the risk of vision loss2. Millions of people are born with common gene variants, which can more than double the risk of developing advanced AMD. Recently, scientists have identified rare variants that put a person at extremely high risk (e.g. 20X or more) for advanced AMD1,3, increase the likelihood they will develop AMD earlier in life, while also increasing the risk of systemic morbidities and associated rare diseases. At Gemini, we see a future where dry AMD therapeutics will be selected for each patient based on what's written into their DNA. We've built our pipeline from the disease on up, using genetics to select our targets, biology to define our approach, and our product engine to create the best therapeutic candidates.
Human genetics is at the core of our therapeutic and development strategies. To build our pipeline, we focused on patients first — particularly patients with a high genetic risk profile. We assessed the functional implications of their underlying mutations. We then matched each molecular abnormality with an appropriate therapeutic candidate. We are currently pursuing three distinct genetically defined patient populations in our ocular portfolio..