![]() ![]() Spark Therapeutics was formed in 2013, in collaboration with CHOP, to develop and market gene therapies. And then it took another four years for the FDA to say yes. A couple days following his surgery in 2013, when his eye patch was removed and he opened his eye, Christian said, “Mom, Papa? Is that you?” Christian Guardino was one of the kids who received a new RPE65. “We had to figure out how to do it to help children.”īut it would take almost a decade before Bennett could conduct trials on children. ![]() “It was crystal clear at that point it was going to work,” Bennett says. Bennett was soon playing tag and fetch with Lancelot and his siblings. Now they bounced down the halls, tracking where they were going by turning their heads, using their treated eyes. Bennett got a call two weeks later to come see the dogs. Al Maguire injected three four-month-old puppies in one eye. Bennett tested them and found the problem was with RPE65, the retinal gene she’d end up working on. She also had a stroke of luck: A co-researcher at Penn told Bennett about a blind Swedish briard dog that had been bred to produce puppies who were also blind. She could still tap some NIH money, and Penn didn’t put the kibosh on her research. Then, in 1999, disaster struck: An 18-year-old boy named Jesse Gelsinger died in a gene-therapy trial overseen by Wilson, and suddenly the whole field came to something of a standstill. Penn Med was a hotbed then of gene-therapy research, led by Jim Wilson, who’d been brought in by über-aggressive CEO Bill Kelley to build a cutting-edge lab with the best collection of genetic scientists, Jean Bennett believes, in the world. She got hired by Penn in 1992 as a professor in ophthalmology her husband, Al Maguire, an eye surgeon at Children’s Hospital, helped in her retinal research, wheeling large lasers between Penn and CHOP. Bennett worked with mice and with rabbits, whose eyes are closer in size and shape to human eyes. Yet developing the process took years of research. ![]() This wasn’t a disease like Alzheimer’s, but more straightforward: a genetic defect identified a healthy gene cloned in a lab, then delivered within a virus that would be surgically implanted. Nothing in gene therapy, she would find, is simple.Įventually Bennett would partner with another researcher working on genetic disease in the retina. It wouldn’t, Bennett soon discovered, be quite that simple. She began, as many scientists do, wanting to conquer her field in a big way, believing that if the genetic bases for gnarly diseases could be identified, they could be fixed. Jean Bennett had been a gene-therapy researcher for two decades at that point, starting with a lab at the NIH. The dogs told her it would work, back in 2000. Her research, along with that of Jean Bennett, gives Spark big potential. ![]()
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