Resumo

HENDRICKS, C L et al. Progress in advanced cellular and gene therapies in South Africa and barriers to patient access: A National Consortium paper on behalf of the BloodSA Cell and Gene Therapy working party. SAMJ, S. Afr. med. j. [online]. 2025, vol.115, n.5, pp.21-25. ISSN 2078-5135.  https://doi.org/10.7196/SAMJ.2025.v115i5.3070.

The fields of molecular and cellular medicine have, in recent years, witnessed a great deal of progress globally, particularly in understanding disease pathogenesis and through the development of advanced cellular therapy products and gene therapies. Despite the transformative potential of these new therapies, low- and middle-income countries face significant barriers to their access. Advanced cellular therapy legislation in South Africa (SA) has not kept up with this fast-advancing field, and requires a fast-tracked renewal. Furthermore, the prohibitive cost of commercial therapies, including chimeric antigen receptor (CAR) T-cell products, and the lack of infrastructure, manufacturing and research capacity, must be addressed to make equitable patient access an achievable goal in our setting. To this end, a national cell and gene therapy consortium, comprising clinicians, clinician-scientists, scientists, legal experts, postgraduate students and representatives from industry, the national blood service and the pharmaceutical industry, was initiated. The mandate of this group is to aid the progression of advanced cellular therapies in SA, and the purpose of this article is to outline the progress that has been made. We will highlight the gaps in each core field of practice within this space, and provide a proposal for making these therapies more accessible in SA.

Palavras-chave : advanced cellular therapy products; cellular therapy regulation; gene therapy regulation; equitable access; health equity; cell and gene therapy access; South African health regulation; chimeric antigen receptor (CAR)-T cell therapy; haemophilia gene therapy.

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