Exploring the future of gene therapy: Professor Simon Waddington visits AGTRU
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We were thrilled to host Simon Waddington alongside IDORI (Infectious Diseases and Oncology Research Institute).
Prof Waddington presented a compelling seminar titled "Gene Therapy, from Hollywood to the bench to the clinic."
Prof Waddington from UCL (University College London), a leading figure in the field of gene therapy, has conducted work spanning rare diseases, viral vectors as research tools, gene delivery, mRNA in lipid nanoparticles, and gene delivery for immunisation. His seminar addressed where gene therapy is today: remarkable breakthroughs, persistent scientific challenges, and the urgent need to make these therapies accessible to low and middle-income countries.
Notably, Prof Waddington has a special interest in Spinal Muscular Atrophy (SMA) Type 1. Once fatal, a single-dose gene replacement therapy can transform clinical outcomes. It is a defining example of modern gene therapy's potential.
In addition, Prof Waddington described the expanding applications of gene-based interventions such as CAR-T cell therapy for cancer and DNA-encoded monoclonal antibodies.
But with prices ranging from USD 1–3 million per dose, accessibility is extremely limited. Producing good manufacturing practice (GMP) quality viral vectors is technically demanding and costly.
Wits/SAMRC Antiviral Gene Therapy Research Unit is hoping to develop local capacity and contribute to a broader ecosystem, including GMP capabilities. This can address issues around access.