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.
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