Genzyme collaborates on gene therapy for rare disease that causes childhood blindness

French pharma major Sanofi (Euronext: SAN) subsidiary Genzyme has entered into a research collaboration with the USA’s University of Florida and the University of Pennsylvania to develop a gene therapy for the treatment of a rare genetic disease that causes childhood blindness.

Leber congenital amaurosis type 1 (LCA-1) is usually diagnosed in children who are less than a year old, and patients remain severely visually impaired for the rest of their lives, the company explained.

The majority of the early stage LCA-1 research is being conducted at the University of Florida, with Genzyme taking on increasingly more activities as the program advances toward clinical trials. Genzyme has the option to in-license the potential treatment before it enters clinical trials.