Introducing CASGEVY in Europe: Ethical and Regulatory Perspectives on the First CRISPR Gene Therapy

Abstract The first year of CASGEVY - CRISPR/Cas9-based gene-editing therapy for transfusion-dependent beta-thalassemia and sickle cell disease - marked a milestone in the application of genome editing technologies in European healthcare. On 9 February 2024, the European Medicines Agency (EMA) granted conditional marketing authorization, followed by the European Commission's approval on 13 February 2024. As a conditionally authorized product, CASGEVY must undergo further confirmatory studies to establish long-term safety and efficacy. This regulatory model, although enabling early access, raises ethical concerns regarding patient consent, risk communication, and the evolving definition of therapeutic standards. As a flagship example of precision medicine, CASGEVY targets disease at its genetic root, aligning with the paradigm's goal to deliver individualized treatment. However, the introduction of such therapies also reveals systemic tensions - including limited access, high cost, and disparities in regulatory implementation across Europe. A questionnaire-based study was conducted with experts from ten European countries across disciplines such as clinical research, laboratory genetics, molecular diagnostics, bioethics, and data governance. The study explored professional views on patient access, long-term monitoring, regulatory adequacy, and the broader societal impacts of gene-editing therapies. Findings indicated widespread concern over ethical inconsistencies, inequity in access, and the challenges posed by conditional approvals in high-stakes contexts. This study emphasizes the urgent need for harmonized ethical frameworks and equitable healthcare policies to ensure that the promises of precision medicine - embodied by therapies like CASGEVY - are realized in a just and socially responsible manner. Key messages • The conditional approval of CASGEVY reveals key ethical and regulatory challenges of CRISPR therapies, including informed consent, long-term safety, and uncertainty in clinical outcomes. • While CASGEVY shows the promise of precision medicine, its rollout reveals access disparities and regulatory gaps, highlighting the need for ethical harmonization and fair healthcare across Europe.