Gene Therapy Restores Sight to Children with Congenital Blindness

Gene Therapy Restores Sight to Children with Congenital Blindness
Foundation Fighting Blindness Chief Research Officer Available to Discuss Medical Breakthrough


OWINGS MILLS, Md.--(BUSINESS WIRE)--Results from a breakthrough study, conducted by researchers at The Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania School of Medicine, show that gene therapy has restored significant vision in five children and seven adults who were previously blind. The Foundation Fighting Blindness funded the clinical trial and has been the long standing funding source of the research that made the trial possible. The study used gene therapy to treat participants with Leber's congenital amaurosis, a severe form of retinitis pigmentosa that causes blindness or substantial vision loss at birth. Trial participants demonstrated improvements in visual acuity, peripheral vision and light sensitivity. The greatest improvements occurred in four children ages 8-11, all of whom are now able to navigate an obstacle course in dim lighting. These findings have been reported in an online article in The Lancet, one of the world's leading medical journals.

"These results are outstanding. We are delighted by not only the restoration of vision and the vision improvement that has been sustained, but also that the treatment has demonstrated an outstanding safety profile," says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness.

The Foundation Fighting Blindness, the world's largest source of non-government funding for retinal degenerative disease research in the world, has significantly funded the research for the CHOP clinical trial from its conception to this latest breakthrough. Dr. Rose expresses his confidence about the clinical trial's results, adding, "the success of this groundbreaking effort is paving the way to use gene therapy to treat a wide range of retinal degenerative diseases." The Foundation will continue to support the clinical trial and related research.

Suggested Articles

Inovio CEO J. Joseph Kim is undeterred by short sellers and other detractors who doubt his company can shuttle a COVID-19 DNA vaccine to market.

Adding entinostat to hormone therapy did not help patients with HR-positive, HER2-negative breast cancer live longer.

The data add to the evidence in support of the IL-6 drug ahead of a FDA decision to approve it in a rare disease served by Alexion’s Soliris.