Patient Keynote: How a Gene Therapy Enabled Sight

When Sabrena Gates’s daughter Mia was stumbling while trick or treating, Sabrena thought it was behavioral. A genetic test showed that Mia had Leber congenital amaurosis type 2, with a prognosis of eventual blindness. As Mia received this diagnosis, Sabrena had Niko who was also diagnosed with Leber congenital amaurosis type 2.  

In this inspiring keynote, Sabrena shares her and her children's journey learning about and receiving Luxturna, a gene therapy designed to treat inherited retinal diseases caused by mutations in both copies of the RPE65 gene. Beyond her own experience, Sabrena addresses how collaboration across the community of scientists, drug developers, patients, clinical researchers and more can advance meaningful medicine development.