U.K. Company Receives $16 Million to Advance Choroideremia Gene Therapy

January 31, 2014

On the heels of the report in The Lancet that choroideremia gene therapy restored vision in a human study at Oxford University, a new company called Nightstar has been spun out of the institution to advance the treatment as well as other emerging retinal disease gene therapies. Syncona LLP, an independent subsidiary of the Wellcome Trust, which is funding the choroideremia trial, is making an investment of more than $16 million in Nightstar.

“The early trial results and subsequent investment in Nightstar are outstanding developments for the entire retinal gene therapy field,” says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. “The commercial support will help tremendously in getting gene therapies out to people affected by choroideremia and other vision-robbing retinal degenerative conditions.”

Led by Oxford University’s Robert MacLaren, M.D., the Phase I/II clinical trial evaluated the safety and efficacy of the choroideremia gene therapy in six people. Five participants demonstrated significantly improved vision in dim settings. The two patients who began the trial with the worst visual acuity were able to read several more lines on an eye chart after receiving the treatment.

For more than two decades, the Foundation Fighting Blindness funded lab studies that helped make the choroideremia gene therapy clinical trials possible. Much of that support went to Miguel Seabra, Ph.D., who played a lead role in understanding the disease and developing the treatment.

“The Foundation has been aggressively funding lab and clinical studies of gene therapies for more than 20 years with the goals of proving that they can save vision and making them attractive investments for the biopharmaceutical industry,” says the Foundation’s chief executive officer, William Schmidt. “The capital infusion for Nightstar is a key milestone in the commercial advancement of these highly promising genetic treatments.”

The following Foundation posts provide more information on recent research advancements for choroideremia gene therapies:

Vision Improvements Reported in Choroideremia Gene Therapy Clinical Trial

Choroideremia Gene Therapy Improves Vision in Human Study

Canadian Team to Conduct Clinical Trial for Choroideremia Gene Therapy