Concerns about the safety of gene therapy treatments have intensified following the death of a female pediatric patient from systemic hyperinflammatory syndrome shortly after receiving gene therapy.

This tragic incident recalls previous cases, including the death of a 27-year-old man during a Duchenne muscular dystrophy trial in 2022 and the fatalities of four boys in trials conducted in 2020 and 2021.

Current safety monitoring in gene therapy involves multiple stakeholders, but the lack of data sharing due to commercial secrecy severely hampers effective risk management.

While it is essential to protect commercial interests, there is a strong call for voluntary transparency that would allow regulators to share critical safety event details with key stakeholders.

Improved sharing of safety data among gene therapy stakeholders is crucial for better risk management and the prevention of adverse events.

Open cooperation between sponsors, investigators, and regulators could facilitate quicker risk-reducing actions and more informed decision-making regarding trial pauses.

To enhance safety protocols, the authors propose a standardized reporting mechanism to notify stakeholders of significant adverse events.

Clear communication about adverse events in understandable terms is vital for fostering trust and ensuring informed consent among participants.

Greater transparency and cooperation among all parties involved in gene therapy research are essential for safeguarding participants and advancing the field effectively.

The FDA's capacity to analyze safety events is hindered by companies' reluctance to share information, even when similar gene therapy vectors are used across multiple trials.