A recent Forbes article on the resurrection of the gene therapy business featured The Children’s Hospital of Philadelphia gene therapy expert Katherine A. High, MD. Dr. High is cited as one of a small group of scientists who “kept the field alive” after business interest in gene therapy “dried up” when companies ran into issues bringing gene therapy from the bench to the bedside.
Dr. High felt “gene therapy researchers had been making two mistakes: using a virus that might make the patient very sick (the adenovirus) and not carefully picking the diseases they would treat,” the Forbes article says. “People were choosing targets based on high unmet medical need and the size of the market, not based on the scientific evidence that gene therapy would impact the disease,” Dr. High said.
An internationally recognized hematologist and molecular researcher, Dr. High is a professor of Pediatrics at the University of Pennsylvania, and a Howard Hughes Medical Institute investigator. She has led translational studies in hemophilia, including reporting on the first cure of hemophilia B in a canine model of the disease using gene therapy.
Pointing out that “High’s approach yielded amazing results,” the Forbes article cites Dr. High’s pioneering use of adeno-associated virus as a gene therapy delivery vector and her contribution to a study of RPE65-related inherited retinal dystrophy, a rare retinal disease that progresses to total blindness by adulthood. “In one trial 12 patients, 5 of them kids, had dramatic improvements in their sight. The children were able to move from Braille classrooms to sighted ones. A late-stage study that could result in the treatment’s approval is ongoing,” the Forbes article notes.
The article also mentions the gene therapy company Spark Therapeutics. Based in part on Dr. High’s work, Spark was launched in October 2013 with a $50 million capital commitment from Children’s Hospital. In addition to building on Dr. High’s RPE65-related work, Spark — which received the “Deal of the Year” award at the 2014 Pennsylvania Bio Annual Dinner & Awards Celebration — is also working toward on hemophilia B treatments and neurodegenerative disease programs.
To read more, see the Forbes article, “Gene Therapy’s Big Comeback.”