Development of stem cell based-gene therapy for immune deficiencies

Recent clinical trials using patient’s own corrected hematopoietic stem cells (HSCs), such as for primary immunodeficiencies (ADA-SCID, X-SCID, CGD, WAS), have yielded promising results in the clinic; endorsing gene therapy to become  standard therapy for a number of diseases. However, the journey to achieve such a successful therapy is not easy and several challenges occur along the way. In my presentation, I will address diverse challenges in the development of gene therapy for immune deficiencies using our own experience with RAG1-SCID as example. We will discuss product development (targeting of the therapeutic cells and choice of a suitable vector and delivery method), the proof-of-concept (in vitro and in vivo efficacy, toxicology, and safety) and the final release steps to the clinic (scaling up, GMP procedures/protocols and regulatory hurdles). 

Author(s): Frank J.T. Staal

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