{
  "abstract": "Background Recent advancements in novel gene therapies (GTx) for sickle cell disease (SCD), including Casgevy and Lyfgenia, are notable therapeutic milestones. However, the clinical implementation of GTx is hindered by health infrastructure limitations, ethical considerations, and financial constraints, particularly in regions with an increased disease burden, such as sub-Saharan Africa.Methods This literature review incorporated PRISMA guidelines and the PICO framework to identify key clinical implementation barriers. Synthesising clinical trial data, peer-reviewed studies, and grey literature, including policy reports, enabled the critical review of implementing GTx for SCD across various healthcare settings.Results A total of 23 studies met the inclusion criteria, revealing three interdependent themes: equity, cost-effectiveness, and implementation feasibility in low-resource settings. Limitations in manufacturing capacities and the shared clinical infrastructure between treating SCD and other conditions with GTx highlight the importance of developing equitable deployment frameworks. Further, traditional cost-effectiveness analyses lack applicability to transformative GTx, which deliver broader societal benefits.Conclusion The implementation barriers question whether GTx for SCD are truly curative if they remain inaccessible to most patients globally. To fulfil the clinical potential of GTx across diverse settings, future advancements must transcend developing novel therapeutic mechanisms and prioritise sustainable and accessible strategies for clinical implementation.",
  "authors": [
    {
      "affiliations": [
        "University of Cambridge, Cambridge"
      ],
      "name": "Nahian Ahmed"
    },
    {
      "affiliations": [
        "PHG Foundation, Cambridge"
      ],
      "name": "Chantal Babb De Villiers"
    }
  ],
  "title": "P34 Evaluating the challenges of clinically implementing novel gene therapies for sickle cell disease",
  "uid": "b5374e35-cce6-594e-83de-a9e3154a2b6b"
}