Gene and Cell Therapy Progresses Unevenly Despite Breakthroughs
Cell and gene therapy (CGT) has changed modern medicine over the past three decades. Once considered experimental, these treatments now offer real hope for diseases that were once untreatable. A major study by Kyoto University and Arthur D. Little Japan tracked this progress by analysing over 160,000 research papers from 1989 to 2023.
The findings reveal a field in rapid motion—but with uneven success across different therapies.
The study highlights two clear leaders in CGT adoption: hematopoietic stem cell transplantation (HSCT) and ex vivo gene therapies. These approaches have seen steady growth and now stand closest to mainstream clinical use. Between 2015 and 2023, regulators approved five key therapies: Strimvelis for ADA-SCID (2016), Kymriah for B-cell leukaemia (2017), Luxturna for inherited blindness (2017), Yescarta for lymphoma (2017), and Tecartus for mantle cell lymphoma and leukaemia (2020). Each relies on modifying cells outside the body before reinsertion.
Yet not all CGT methods have advanced at the same pace. Mesenchymal stem cell therapy and in vivo gene therapy—where genetic material is altered directly inside the patient—have lagged behind. The study also maps global research trends, showing the US and China as dominant forces. Their high-impact studies often emerge from cross-border partnerships, shaping innovation worldwide. Japan, while prolific in cell therapy research volume, has yet to match this influence in setting global benchmarks.
Collaboration remains critical to progress. Networks of researchers, clinicians, and funders create ecosystems that accelerate discovery. But the study warns that geopolitical tensions and unstable funding could disrupt these partnerships. The bibliometric analysis further pinpoints active research hubs and rising subfields, offering a detailed snapshot of where the science is headed.
The study confirms CGT's potential to revolutionise treatment for previously incurable conditions. With five therapies now approved and more in development, the field is moving from promise to practice. Yet its future depends on maintaining strong international cooperation—and ensuring that breakthroughs reach patients beyond just a few leading nations.
