New article calls for urgent action to safeguard gene therapy data

New article calls for urgent action to safeguard gene therapy data

Gene therapy has advanced from pioneering trials in the 1990s to several recent regulatory approvals in the U.S.A. and Europe. These approvals were made possible because of the courage of the 395 people with hemophilia who stepped forward to participate in gene therapy clinical trials and share their data, fully aware of the risks, the uncertainty, and the impossibility of re-dosing. They did so not only for their own health, but to advance science and benefit the broader community. Today, their contributions are at risk of being lost. A new article—“The Critical Need to Consolidate All Gene Therapy Data in Haemophilia”—calls for urgent action to safeguard gene therapy data.

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Some companies are exiting or scaling back gene therapy programs, while others continue to invest in their development and commercialization. Yet critical trial data from across all programs remain fragmented across treatment centres, registries, and corporate databases. Preserving these data is not just a scientific necessity—it is an ethical responsibility. Maintaining long-term safety data is essential to monitor risks, protect future patients, and ensure the responsible development of new therapies.

A recent article published in Haemophilia, the official journal of the WFH, calls for urgent collaboration to consolidate clinical trial data. The World Federation of Hemophilia (WFH) Gene Therapy Registry (GTR)—endorsed by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP)—provides the solution: a single global platform for preserving and analyzing data across trials and commercial programs. Consolidation of clinical trial data is both a scientific imperative and an ethical obligation to honour patient contributions.

To read the article, “The Critical Need to Consolidate All Gene Therapy Data in Haemophilia”, please click here.

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