Giving sight to dozens and hope to even more.
(Left) Dr. Jean Bennett; F.M. Kirby Professor of Ophthalmology and Cell & Developmental Biology; Director, Center for Advanced Retinal & Ocular Therapeutics; University of Pennsylvania, Perelman School of Medicine. (Right) Dr. Katherine High, President and Head of Research & Development, Spark Therapeutics
Sight is something many of us take for granted. For Dr. Jean Bennett and Dr. Katherine High, it’s a precious gift that they’re helping to restore in dozens of patients. Through a decades-long collaboration, they’ve partnered to spearhead clinical trials and groundbreaking research, crossing the finish line in getting the first FDA approval on a gene therapy drug to cure a specific form of congenital retinal dystrophy. For this groundbreaking innovation and the tenacity to see it through, they are the 2018 co-winners of the Sanford Lorraine Cross Award.
Dr. Jean Bennett & Dr. Katherine High meet and collaborate at several gene therapy seminars and retreats.
Dr. Bennett begins working at the University of Pennsylvania with her husband, Dr. Albert Maguire, to explore gene therapy as a treatment for vision loss.
Dr. Bennett and team successfully restore sight to dogs with a canine form of Leber’s congenital amaurosis (LCA).
Dr. High meets with Dr. Steven Altschuler, securing funding through the Children’s Hospital of Philadelphia (CHOP) for a human clinical trial to test Dr. Bennett’s discoveries.
Dr. Bennett & Dr. High pair up to begin the first human clinical gene therapy trials in LCA patients, yielding extraordinary results.
Spark Therapeutics is founded by CHOP with Dr. High as President to develop this treatment into a drug – Luxturna™.
Luxturna™ becomes the first gene therapy drug to earn FDA approval.
Dr. Bennett & Dr. High win the Sanford Lorraine Cross Award for their groundbreaking innovation and problem solving.
DR. JEAN BENNETT
Dr. Jean Bennett and Dr. Katherine High met early in their careers, bonding over a shared interest in gene therapy and similar backgrounds. Little did they know how pivotal their friendship would become to the field.
Dr. Bennett and Dr. High kept in touch as they pursued their careers researching Leber’s congenital amaurosis (LCA) and hemophilia, respectively. Over the years, both made great strides, but Dr. Bennett’s discoveries stayed in the back of Dr. High’s mind for years.
Dr. Bennett was testing gene therapy – replacing a missing or mutated gene with a healthy one – to repair vision in dogs with LCA, a genetic condition that eventually causes blindness in patients. “Ninety-nine percent of the time we saw this remarkable reversal of the blindness,” she says. “So we were very excited to test this in humans.”
But to pursue human clinical trials, she needed financial support. Support that was scarce after a late-1990s downturn in gene therapy.
“At that time, everyone was walking away,” says Dr. High. “But I was convinced we were not seeing any problems that could not eventually be solved.”
Dr. High met with the head of the Children’s Hospital of Philadelphia (CHOP), walking away with a funding opportunity that she took directly to Dr. Bennett.
“I can remember the exact day,” Dr. Bennett says. “One Dr. Katherine High knocked on my office door, sat down and said, ‘Jean, how would you like to run a clinical trial?’ It took me a billionth of a second to say yes.”
From there, the doctors’ continued collaboration resulted in amazing patient findings and unstoppable success.
“Dr. High and I have the same mission,” says Dr. Bennett. “We were experts at these areas that needed to intersect, and as she said, ‘We move forward joined at the hip.’”
The first human clinical trials came to fruition in 2007, with patients seeing dramatic improvement in just weeks and even days after treatment.
“I had to pinch myself to remind myself that this is real,” says Dr. Bennett.
“Patients are really the reason we do this work,” says Dr. High. “They are the north star of a lot of what we do.”
But to get this treatment across the finish line, Dr. High and Dr. Bennett knew they’d need more than data – they’d need a company. After years of working as a medical investigator, Dr. High found herself in a new role – President and Head of R&D at Spark Therapeutics, a company founded by CHOP to develop and distribute gene therapy drugs.
Their drug, Luxturna™, eventually became the first FDA-approved gene therapy, opening the door for many future breakthroughs. A goal they’ve never forgotten.
“The most important thing is to do a good job on today’s problems,” Dr. High says. “Because if you do that, there will be a tomorrow.”
