Gund, a former owner of the NBA's Cleveland Cavaliers and the NHL's San Jose Sharks, runs the foundation somewhat like he operates his Princeton, N.J.-based venture-capital firm.
The nonprofit provides seed money for early-stage research, when scientists might have a promising hypothesis but are not far enough along to attract grants from the government and other more traditional private sources.
"You get the more conventional funding later if the small enterprise is successful," Gund said. "There's such a long distance between the lab and the trials and FDA approval. We need to de-risk those things."
A key focus is so-called "orphan" diseases, such as Leber's congenital amaurosis (LCA), the eye disease that afflicts the trial participants at Children's. Such ailments are too rare to attract much attention by pharmaceutical companies.
The foundation's first effort was funding the Berman-Gund lab at Harvard. The University of Pennsylvania's Albert Maguire, the surgeon for the trial at Children's Hospital, was working in that lab in 1985 when he and his wife first talked about the idea of correcting flawed genes in the retina.
The foundation also gives out career-development awards, one of which went to Maguire's wife, Penn's Jean Bennett, in the early 1990s. She was the scientific director for the trial at Children's.
Gund himself has a type of blindness related to LCA, part of a broader category of eye disease called retinitis pigmentosa. He lost his night vision over a period of several years, then lost his daytime vision in 1970. He has some sensitivity to light, but cannot see any shapes or colors.
"It's kind of an opaqueness where a little light comes in," he said.
As with the patients at Children's, Gund's blindness was apparently caused by a mutation in a single gene.
But scientists haven't found it yet, though he and some of his siblings have submitted blood samples to aid in the quest. He acknowledges that it would be wonderful to regain some vision, but said that is not a big goal.
"I haven't thought a lot about that," said Gund, an active snow skier, sculptor and fly fisherman. "I try not to. It's not productive."
Instead, he said, he prefers to think about the blind children who could be helped by the foundation's work. And the Children's trial, he said, is a big step.
"It's extraordinary," Gund said. "It's a great event for everybody who has a retinal degenerative disease."
Contact staff writer Tom Avril at 215-854-2430 or tavril@phillynews.com
