Sanford L Boye,

Sanford Boye is an Associate Scientist of Pediatrics at the University of Florida and Associate Director of the Powell Gene Therapy Center. He is the director of PGTC the Vector Core Laboratory, which specializes in the manufacture of AAV based gene therapy vectors. Sanford’s research focuses on 1) the design, construction and utilization of recombinant adeno-associated virus (AAV) vectors for gene transfer and 2) the evaluation of clinically relevant AAV vectors in animal models of human disease, with emphasis on retinal degenerative disorders. He was a member of the National Eye Institute- funded consortium who developed an AAV gene therapy for the RPE65 version of Leber Congenital Amaurosis (LCA2), designing the clinically relevant AAV vector. This led to several successful, first in human clinical trials of gene therapy for blindness and in part to the FDA’s first approval of a gene therapy for an inherited disease. He has designed AAV vectors that have been used to successfully address a variety of retinal disease models, including autosomal recessive retinitis pigmentosa, achromatopsia, X-linked juvenile retinoschisis, Bardet-Biedl syndrome, Usher syndrome and multiple forms of Leber congenital amaurosis (LCA). Recently his work has focused on modifications of the AAV capsid to improve vector performance and safety. Currently he splits his time between working with Dr. Shannon Boye on both basic science research and translational programs- gaining FDA approval for AAV based treatments for retinal disease and directing Vector Core within the Powell Gene Therapy Center.

Sanford Boye’s research focuses on 1) the design, construction and utilization of recombinant adeno-associated virus (AAV) vectors for gene transfer and 2) the evaluation of clinically relevant AAV vectors in animal models of human disease, with emphasis on retinal degenerative disorders. He was a member of the National Eye Institute- funded consortium who developed an AAV gene therapy for the RPE65 version of Leber Congenital Amaurosis (LCA2), designing the clinically relevant AAV vector. This led to several successful, first in human clinical trials of gene therapy for blindness and in part to the FDA’s first approval of a gene therapy for an inherited disease. He has designed AAV vectors that have been used to successfully address a variety of retinal disease models, including autosomal recessive retinitis pigmentosa, achromatopsia, X-linked juvenile retinoschisis, Bardet-Biedl syndrome, Usher syndrome and multiple forms of Leber congenital amaurosis (LCA). Recently his work has focused on modifications of the AAV capsid to vector performance and safety. Currently he splits his time between working with Dr. Shannon Boye on both basic science research and translational programs- gaining FDA approval for AAV based treatments for retinal disease and directing Vector Core within the Powell Gene Therapy Center.