Lifeboat Foundation

This gene therapy treats LCA1, causing early childhood vision loss, affecting under 100,000 people:

“One patient reported for the first time being able to navigate at midnight outdoors only with the light of a bonfire,” said Cideciyan, who is also co-director of the Center for Hereditary Retinal Degenerations.

The clinical trials were co-led by researchers from the Perelman School of Medicine at the University of Pennsylvania.

The gene therapy (ATSN-101) is specifically designed to target and correct the genetic mutation in the GUCY2D gene. This gene creates vision-imparting proteins. ATSN-101 is “adapted from the AAV5 microorganism.”