A global clinical-stage gene therapy company developing gene therapy beyond rare diseases. Our mission is to preserve sight and fight the devastating impact of blindness. Our science is grounded in the genetic understanding of patients with serious eye diseases to make medicines designed to have a meaningful impact. Our initial focus is age-related macular degeneration, or AMD, one of the leading causes of irreversible blindness, affecting more than 196 million people worldwide. We are developing a differentiated pipeline of recombinant adeno-associated virus, or rAAV, gene therapy candidates targeting genetic variants in the complement pathway that we believe to be key drivers of AMD. Our investigational gene therapies are designed as one-time treatments. Our lead investigational gene therapy, GT005, is advancing in an ongoing Phase I/II clinical trial and is being evaluated in Phase II clinical trials in two different genetically defined patient populations with geographic atrophy, or GA, an advanced form of dry AMD.
Lead Underwriter
Citigroup Global Markets Inc, Goldman Sachs & Co. LLC, Morgan Stanley & Co. LLC
