Genzyme Enters Gene Therapy R&D Alliance

Genzyme, a Sanofi company, has established a research collaboration with the University of Florida and the University of Pennsylvania to develop a gene therapy for the treatment of Leber congenital amaurosis type 1 (LCA-1), rare genetic disease that causes childhood blindness. Genzyme is providing $900,000 over three years to Dr. Shannon Boye, assistant professor of ophthalmology at the U of Florida, to fund her LCA-1 research focused on the gene mutation called guanylate cyclase (GUCY2D).
 
The majority of the early stage LCA-1 research is being conducted at U of Florida, with Genzyme taking more activities as the program advances toward clinical trials. Genzyme has the option to in-license the potential treatment before it enters clinical trials.
 
“Genzyme is a very collaborative partner and has been wonderful to work with,” said Dr. Boye. “We share a heartfelt commitment to helping LCA-1 patients and their families and to pursuing a treatment building upon prior, promising non-clinical studies.”
 
“We are proud to partner with Dr. Shannon Boye and her team at the University of Florida, who are leading experts in the field of ocular gene therapy, as well as our clinical colleagues from the University of Pennsylvania, who are sharing important insights about LCA-1 patients’ needs,” said Rich Gregory, Ph.D., head of the Sanofi Genzyme R&D Center. “Together, we are striving to provide new hope to children who might not otherwise be able to see.”