Gene Therapies

Information on our gene therapy GT005

Our investigational gene therapy, GT005, is designed to restore balance to a part of our immune system called the complement system.

When the complement system is overactive it can cause inflammation that damages healthy eye tissues. GT005 is designed to stimulate a person’s cells to create Complement Factor I, a protein that is key to regulating the alternative pathway of the complement system. The goal is to slow, or possibly stop the progression of dry-AMD. 

Patients in our ongoing Phase I/II clinical trial, known as the FOCUS study, receive a single dose of GT005 through an injection below their retina.

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