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Jerry Mendell’s Courage Inspires ASGCT Member to Turn Award into Endowment

Devin Rose - June 05, 2023

ASGCT Member Suku Nagendran, MD, and his wife, Ann, have funded the endowment in perpetuity to help the next generation of scientists develop more gene therapies.

ASGCT Member Suku Nagendran, MD, says it’s a dream of his that genetic medicines will be a “drug of choice” for conditions that are now life threatening. Soon, he hopes—as soon as 25-to-30 years. Dr. Nagendran makes this prediction because he has seen firsthand the impact that gene therapy can have in humans, which he would not have witnessed without Jerry Mendell, MD.

“I call him the Godfather of Genetic Medicine in the clinic,” Dr. Nagendran says. “He’s done this for 45 years and I think a lot of us who are in the field owe him something.”

Dr. Mendell is the namesake for a Society award that Dr. Nagendran and his wife, Ann, began supporting in 2021 to recognize translational science in cell and gene therapy. Dr. Nagendran has an extensive background in drug development and collaborated with Dr. Mendell on the regulatory process of the Zolgensma clinical trial. Now, Dr. Nagendran has funded the endowment in perpetuity as a way to give back to ASGCT and to help the next generation of scientists develop more gene therapies.

“This is my way of not only repaying [Dr. Mendell], but also hoping that the money I’ve given ASGCT will last long term to encourage young scientists and physicians to develop life-saving products for horrible diseases.”

Dr. Nagendran says he’s been through the spectrum of moving a trial from animal models to humans, but he has never seen outcomes like those resulting from Zolgensma to treat spinal muscular atrophy. Kids who couldn’t sit up or lift their heads up before receiving the gene therapy were walking, running, and throwing balls afterwards.

“That was magical…and it was amazing to see the gratefulness of the parents and the families to Jerry for what he has done,” Dr. Nagendran said, adding it takes a lot of courage to actually dose the patients.

“I think it's one thing to develop the construct, but it's something else to do the trial, worry about the patient 24/7, take the calls from the parents when something doesn't go right.”

His experience with Zolgensma gives Dr. Nagendran hope that more gene therapies can be developed to treat common diseases in humans, but scientists need resources and opportunities to do so, which is where ASGCT comes in.

ASGCT is “the premier scientific organization when it comes to gene therapy and basic science research, and I’ve watched the organization grow rapidly,” says Dr. Nagendran, a member since 2016.

He hopes that a decade from now, research conducted using the endowment fund will result in breakthrough therapies, particularly for children who would not survive long without them.

“If these kids are gone at two years of age, you have no idea what their potential level was. All of a sudden, these kids are surviving…they’re functional, their brains are working. Who knows what they can contribute to society, right?”

Dr. Nagendran wraps up by emphasizing that the overall goal is getting treatments to patients faster.

“We hope that encouraging translational research in gene therapy enables faster bench to bedside work that results in more therapies being available to patients sooner, which will satisfy the FDA need for warp speed in drug development and gene therapy when appropriate!”

The Jerry Mendell Award for Translational Science is given out at the Annual Meeting each spring. The first two honorees were the award’s namesake Jerry Mendell, MD in 2021 and Kathy High, MD in 2022. The award was shared this year by Mark Dudley, PhD; Bruce L. Levine, PhD; and Isabelle Riviere, PhD. ASGCT is grateful to the Nagendrans for their generous gift allowing ASGCT to provide this award in perpetuity.

Nominations for the 2024 Jerry Mendell Award for Translational Science will open in fall 2023.

Devin Rose is ASGCT's communications manager.

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