On July 23, Science and Retraction Watch broke the tragic news that a 6-year-old girl in China had died days after being given an experimental gene therapy. The investigation reported that the researchers, led by Zilong Qiu at the Songjiang Research Institute, had received over $800,000 in funding from the girl’s family to develop the experimental therapy. A year after the girl’s death, in March of 2025, the research team published evidence that their approach worked in mice and monkeys but did not disclose the death. Gene therapy experts and bioethicists interviewed by Science and Retraction Watch who reviewed the preliminary safety data in nonhuman primates questioned whether the study should have gone ahead.
The South China Morning Post reports that the researcher’s university is launching a comprehensive investigation into both the paper and the clinical study. Whatever the outcomes of any investigations, the tragedy leaves another dark stain in the history of gene therapy development, a history that is unfortunately filled with other potentially avoidable deaths.
In 1999, 18-year-old Jesse Gelsinger died in a clinical trial meant to test the safety of an adenovirus vector in support of a potential gene therapy treatment for ornithine transcarbamylase deficiency, a genetic liver disease. The US Food and Drug Administration investigated and found that the researchers had broken several rules of conduct.
The story of Jesse Gelsinger set back gene therapy development by a decade. A few years after Gelsinger’s death, the FDA shut down its Institute for Human Gene Therapy. And while fundamental research in support of gene therapies continued, it wasn’t until 2014 that the US ran another clinical trial on a gene therapy.
In 2026, we must not let this latest incident set us back. The promise of gene therapy is far too great, and to realize that promise requires a bold leap forward that will protect patients and the science.
At their best, gene therapies offer potential one-time, permanent cures for debilitating genetic disease. And unlike the earliest days of gene therapy, tools like CRISPR, base editors, and a bevy of vectors have proven track records of success in human gene therapy.
The FDA has already approved gene therapies that cure sickle cell anemia, metabolic disease, and even vision loss, among others. The story of baby KJ Muldoon, for whom a personalized gene therapy was developed and administered, showed the world that curing genetic disease was possible.
In fact, the FDA is even relaxing its standards for getting gene therapies approved—at least in very specific cases. It may seem counterintuitive to make administering experimental gene therapies easier when pushing too soon can result in the worst possible outcome. But that’s only in the context of treating gene therapies like any other drug.
Gene therapies are not like any other drug. It’s true that death is a, thankfully, rare part of some drug trials. But gene therapy’s greatest advantage—its permanence—is also a great weakness in a clinical trial: you can’t stop once you’ve begun. If a patient has a negative reaction to a more typical drug, the overseeing clinician can stop a subsequent administration.
Typical drug trials have also become routine. Institutional review boards generally know what to look for before approving a trial. And often the drugs being tested now are built on several generations of drugs tested before. Our knowledge and experience create a safety net.
But gene therapy is a new business. The number of people who have received an in vivo base editor, for example, is in the dozens. Some therapies are designed to work in a single individual only. The therapy itself fundamentally alters a patient’s DNA. None of that is routine. We are still learning what the risks and requirements are at every stage of gene therapy development.
Therefore, it’s time for regulatory agencies to wholly separate the approval process of gene therapies from other drugs. Gene therapies in development should require very specific standards and go through an enhanced review process overseen by an independent panel of gene therapy experts before dosing in humans. Patients should meet strict requirements before they can participate in a trial. And unique budgets should be set in place to provide public funding for gene therapy research, a budget separate from that from other drugs. This would mitigate the drive for researchers to resort to unconventional, possibly unethical tactics to secure funding.
It’s naive to think that these approval measures would eliminate risk in gene therapy development entirely—failure is an unfortunate reality we face in pursuit of new medicines. But if the right people ask the right questions before gene therapies move to funding for trials, it’s possible we may prevent the worrying combination of desperation and overzealousness that can lead to preventable tragedies.
This editorial is the result of collective deliberation in C&EN. For this editorial, the lead contributor is Max Barnhart.
Views expressed are not necessarily those of ACS.
