Sanofi gets USFDA fast track designation for neovascular age-related macular degeneration gene therapy

SAR402663 delivers genetic material encoding soluble FLT01 designed to inhibit vascular endothelial growth factor (VEGF). Sanofi is currently evaluating SAR402663 in a phase 1/2 (clinical study identifier: NCT06660667), for the treatment of patients with neovascular AMD. The gene therapy aims to address the underlying disease pathology by inhibiting abnormal blood vessel growth, reducing vascular leakage and minimizing retina damage, while significantly reducing treatment burden through the elimination of frequent intravitreal injections.

AMD is an acquired progressive degeneration of the retina that affects approximately 200 million people globally. Neovascular, or “wet”, AMD is a severe form of macular degeneration. It is characterized by growth of abnormal blood vessels beneath the retina, which can lead to vision loss, potentially progressing to blindness in advanced cases. Neovascular AMD affects more than one million people in the US and six million people worldwide; it has a profound impact on quality of life, including ability to read, drive and perform other daily activities.