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Marina Cavazzana awarded 2026 Taylor Prize for advances in gene therapy
By Patrick Morley
The internationally renowned physician-scientist has helped turn the promise of gene therapy into life-saving treatments
By Patrick Morley
When Dr. Marina Cavazzana began treating children with gene therapy more than three decades ago, the premise was as ambitious as it was unproven: remove a patient's own stem cells, introduce a working copy of the faulty gene in the laboratory and return the modified cells to the body.
Now, Cavazzana, a professor of hematology at Université Paris Cité, is the 2026 recipient of the J. Allyn Taylor International Prize in Medicine, recognizing her important contributions to the field.
Established in 1985, the Taylor Prize honours internationally recognized researchers for outstanding achievements in basic and clinical research. This year’s $50,000 prize acknowledges the power and potential of gene therapy.
“Gene therapy is changing the outlook for patients with some rare genetic diseases that once had few effective treatment options,” said Robert Bartha, PhD, vice dean of research and innovation. “Dr. Cavazzana’s work has been instrumental in advancing the field, from some of its earliest successes to the development of safer, life-saving treatments.”
The promise of gene therapy
The families who came to Cavazzana were often seeking treatment for children with severe genetic diseases and few remaining options.
"They wanted their children cured, and they were ready to accept a new treatment that no other child had tried before," Cavazzana said. "We understood the fears of the families, but we also let them know that there was hope."
Among those patients were children born with severe combined immunodeficiency (SCID), a rare genetic disorder that leaves babies with little or no functioning immune system. A bone marrow transplant can provide a cure, but a suitable donor is not always available.
Gene therapy offered another approach: use the patient's own cells.
In this case, blood-forming stem cells are collected from the patient and, in the laboratory, a working copy of the faulty gene is introduced into them. The modified cells are then returned to the patient, where they can give rise to healthy immune cells.
For Cavazzana, some of the earliest evidence the treatment was working came through blood tests.
"The stem cells generated new cells containing the right information," she said. "We followed the patient each week and after three months, the T-cells appeared in the blood. It was incredible."
Children who had been born without functioning immune systems were developing their own immune cells.
"We could cure a child with severe combined immunodeficiency disease literally within the first year of life," Cavazzana said. "It was a great step forward for the medical field."
A duty to continue
The early trials also exposed risks that researchers had not anticipated.
Several years after receiving treatment, some of the children developed leukemia. Researchers determined that the viral vector used to deliver the working gene had, in some cases, inserted it near genes that can promote cancer, inadvertently activating them.
"This was the most dramatic moment in my medical career," Cavazzana said. "I was completely shocked."
Emotionally exhausted and questioning her future in medicine and research, Cavazzana spoke with the mother of one of the children who had developed leukemia.
Rather than expressing anger, the mother encouraged Cavazzana to continue.
"She told me that I had to continue to fight. That I had a duty to continue," Cavazzana recalled. "She said that I had saved her child at birth and that she'd had a life with him as a result."
For Cavazzana, the exchange changed the course of her career. The complications could not be dismissed, but neither could the years of life the treatment had made possible.
"I thought, ‘I have no right to stop,’" she said. "This mother gave me the courage and the force to decide that 'yes, I will continue.'"
A field transformed
After the leukemia cases, Cavazzana and researchers around the world worked tirelessly to find out what went wrong. They discovered a new generation of viral vectors that delivered the healing gene without the powerful genetic switches that had activated cancer-causing genes in the early patients.
Cavazzana played a vital role in studying the complications and in further clinical research that used these safer approaches. The experience also brought more rigorous long-term monitoring of patients – lessons from a painful setback that helped make later gene therapies considerably safer.
For Cavazzana, there are also more personal reminders of what the work has meant. Every Christmas, she receives a text message from the family of a child she helped treat for sickle cell disease. Other families whose children she treated years ago still keep in touch.
"These families have been another important part of my career and inspire me to keep working," she said.
Cavazzana has had a front-row seat to the evolution of gene therapy, from some of the earliest successful treatments for immune deficiencies to advances in treating inherited blood disorders.
The Taylor Prize, which will be presented to Cavazzana at a special celebration on November 19, recognizes the collective effort behind that progress: the scientists and clinicians who spent decades building the field, and the patients and families who faced considerable uncertainty as participants in its early trials.
"Maybe we had the privilege of putting the pieces of gene therapy together," she said. "But it is not a single discovery. It is built from the work of many incredible people over many decades. This award is for all."