Gene therapy pioneers named finalists for $1 million Sanford Lorraine Cross Award

Nov. 1, 2018

Four medical pioneers have been named finalists for Sanford Health’s new $1 million Lorraine Cross Award, with achievements that include reversing a rare form of blindness, enabling children to walk and setting the ethical standard for all gene therapy trials.

The award will be given for contributions to medicine, science and innovation in a Dec. 4 ceremony in Sioux Falls.

“To find our four finalists, we identified medical and research pioneers, which led us specifically to the field of gene therapy,” said David Pearce, president of research at Sanford Health. “These four finalists helped lay the groundwork for much of what is being explored now, and we want to honor them for that.”

The finalists are:

  • Dr. Jean Bennett and Dr. Katherine A. High, whose work with the RPE65 mutation has reversed an inherited form of blindness. Bennett and High pioneered the gene therapy, took it to clinical trials and then received FDA-approval for the treatment, the first FDA approval of a gene therapy for a genetic disease. High also co-founded Spark Therapeutics, a fully integrated, commercial gene therapy company working to accelerate the timeline for bringing new gene therapies to market. Bennett is a professor of ophthalmology at the University of Pennsylvania, and High is president and head of research and development at Spark Therapeutics. 
  • Brian Kaspar, whose lab discovered a gene replacement therapy approach that seeks to change the course of spinal muscular atrophy by addressing its genetic cause.  SMA is a devastating disease that robs babies of basic muscle functions, such as breathing and swallowing, and in its most severe form, type 1, usually leads to death by age 2. An initial clinical trial using the AAV9 vector to treat SMA type 1 demonstrated a dramatic survival benefit and rapid improvement in motor milestones. Kaspar is the scientific founder and chief scientific officer of AveXis, a gene therapy company that was acquired by Novartis in 2018.
  • Dr. James M. Wilson, whose work helped define the scientific and ethical standards for advancing gene therapies through FDA-approved clinical trials. He is the director of the Gene Therapy Program, the Rose H. Weiss Professor and Director of the Orphan Disease Center, and a professor of medicine and pediatrics in the Perelman School of Medicine at the University of Pennsylvania. In 2008, Wilson and the University of Pennsylvania co-founded REGENXBIO Inc., a clinical-stage biotech company designing gene therapy products.

In December, the Sanford International Board will hear presentations from the award finalists.

“These individuals are changing the world,” said David Shulkin, international board member and Sanford Health chief of innovation. “At Sanford, we want to reward innovation and scientific breakthroughs. We’re committed to finding the next frontier in medicine and research, and recognizing others with the same goal.”

Candidates for the award were filtered through nominations from the public and a computer algorithm that sifted through medical publications in search of great discoveries. An interdisciplinary scientific advisory board identified top areas of innovation, and the candidates were narrowed down to the top four.

The award is named after the Lorraine Cross, a symbol recognized around the world as synonymous for those who take action for their passions.  In recent history, Sanford has used it to symbolize innovation in health care.

Gene therapy pioneers named finalists for $1 million Sanford Lorraine Cross Award

They reversed blindness, helped children walk and set the standard for all gene therapy trials. Meet the finalists for Sanford’s first $1 million Lorraine Cross Award.

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