Saluting Katherine High, MD, Recipient of the 2022 Jerry Mendell Award for Translational Science

Suku Nagendran, MD - April 20, 2022

Suku Nagendran, MD, and his wife Ann are proud to be supporting the next generation of researchers and clinicians in gene therapy through the Jerry Mendell Award for Translational Science.

This year, the American Society of Gene and Cell Therapy (ASGCT) selected Katherine “Kathy” High, MD, to follow in Jerry Mendell’s prestigious footsteps as the 2022 recipient of the Jerry Mendell Award for Translational Science. My wife, Ann, and I are thrilled with this decision, as Kathy is the perfect example of a researcher and clinician whose progress in the field has led to life-changing treatments.

Kathy has played a pivotal role in advancing the development of gene therapies and continues to be a model for the focus and passion it takes to progress a therapy to a clinical stage.  

Kathy’s entire career has focused on pioneering work in the area of gene therapy, including basic, translational and clinical investigation. She is currently president of therapeutics at Asklepios BioPharmaceutical (AskBio), a clinical-stage gene therapy company. She is responsible for driving the strategic direction and execution of AskBio’s preclinical and clinical programs, which cover a broad range of central nervous system, neuromuscular, metabolic, and cardiovascular disorders.

Earlier in her career, Kathy was president and head of research and development at Spark Therapeutics, where she directed the development and regulatory approval of Luxturna®, the first gene therapy for a genetic disease to obtain regulatory approval in both the U.S. and Europe. She is an emeritus professor at the Perelman School of Medicine at the University of Pennsylvania and was the founding director of the center for cellular and molecular therapeutics at Children’s Hospital of Philadelphia, where she was also an investigator of the Howard Hughes Medical Institute. Kathy served a five-year term on the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee and is a former president of ASGCT.

Last year, Ann and I partnered with ASGCT to create the Jerry Mendell Award for Translational Science, which recognizes the extensive efforts of the next generation of researchers and clinicians working to bring gene and cell therapies to clinical trials and ultimately, to patients.

The award is named after Jerry Mendell, a world-renowned researcher and pioneer in the care of neuromuscular diseases, because of his expertise in the clinical area of translational research and his many contributions to the development, delivery, and follow-up of clinical-stage gene therapies. Jerry was the principal investigator for the clinical trial in patients with spinal muscular atrophy (SMA) type 1 that led to the 2019 FDA approval of AveXis’ Zolgensma®, which has since treated more than 1,800 patients with SMA worldwide and had a life-changing effect on patients and their families. For all these reasons, Jerry was named the first recipient of the award. 

On top of what she has already accomplished, Kathy remains committed to advancing gene therapy even further. I am confident Kathy and future recipients of this award will use their expertise and scientific insights to bring gene and cell therapies into clinical development and ultimately provide new treatments to the patients and families who so desperately need them.  

Dr. Nagendran is the president of R&D and chief medical officer at Jaguar Gene Therapy and a Member of ASGCT.