Introducing the Jerry Mendell Award for Translational Science

Every so often, you encounter someone who impacts you profoundly, both personally and professionally. For me, that person is Jerry Mendell, M.D., a world-renowned researcher and pioneer in the care of neuromuscular diseases for more than 40 years. Among Jerry’s many achievements, he was the first person to study viral mediated gene therapy for muscular dystrophy in humans. He was also the principal investigator for the clinical trial in patients with spinal muscular atrophy (SMA) type 1 that led to the U.S. Food and Drug Administration (FDA) approval of AveXis’ Zolgensma in 2019. During most of that time, I was working as chief medical officer at AveXis and collaborated closely with Jerry on the Zolgensma clinical trial program. As one of only two gene therapies recently approved by the FDA, Zolgensma has treated hundreds of patients with SMA worldwide and has had a life-changing effect on patients and their families.

Jerry’s unsurpassed commitment to his patients and brilliant scientific mind touched my wife, Ann, and I so deeply that we have partnered with the American Society of Gene & Cell Therapy (ASGCT) to create an award in his name. The Jerry Mendell Award for Translational Science recognizes the extensive work required to bring gene and cell therapies to clinical trials. Ann and I are thrilled the first recipient of this award is Jerry himself.

The ASGCT award committee selected Jerry 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. Currently, Jerry is a professor in neurology, pediatrics, pathology, physiology, and cell biology in the Center for Muscle Health & Neuromuscular Disorders at The Ohio State University / Nationwide Children’s Hospital in Columbus. Jerry continues to work as a translational scientist conducting preclinical and clinical research using gene therapy to treat muscular dystrophy.

Despite the many advances made in the gene therapy field to date by Jerry and numerous others, patients with severe genetic diseases continue to need new treatments. I know firsthand the incredible opportunity gene therapy can bring, and that’s why I continue to leverage what I learned from Jerry in my work as president of R&D at Jaguar Gene Therapy. It is important to support the up-and-coming researchers and clinicians who are essential in further advancing innovation in the field, and my hope is to help encourage new life-changing treatment options through the Jerry Mendell Award for Translational Science.

With a commitment throughout our lives to give back and pay it forward, my wife and I are proud to be supporting, encouraging, and boosting the next generation of researchers and clinicians in gene therapy through the award. Ann and I have every expectation that Jerry and future award winners will use their expertise to bring gene and cell therapies to clinical trial, ultimately enabling new gene therapies to reach the patients who desperately need them.  

Suku Nagendran, M.D. is the president of R&D at Jaguar Gene Therapy and a member of ASGCT.