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Scale-­up of human pluripotent stem cell-­based therapies for age-­related macular degeneration

Roberts, IT; (2015) Scale-­up of human pluripotent stem cell-­based therapies for age-­related macular degeneration. Doctoral thesis , UCL (University College London). Green open access

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Abstract

This doctoral thesis was in collaboration with a research group from the Institute of Ophthalmology at Moorfields Eye Hospital and the London Project to Cure Blindness. The group was in late stage pre-clinical development creating a pluripotent stem cell (PSC) derived therapy to produce retinal pigment epithelium cells (RPE) for the treatment of Age Related Macular Degeneration (AMD). Creating effective treatments for AMD is vital given that it is the leading cause of blindness in the developed world with around 500,000 people in the UK afflicted with the disease, half of which are registered as visually impaired. In addition, for the dry form of the disease, which represents 85-90% of total cases, there is no effective treatment. The current lab based manufacturing protocols intended for use during Phase 1 trials are not feasible for subsequent larger scale clinical trials or for the commercial manufacture of an affordable mass-produced therapy. The aim of this thesis was to explore the feasibility of applying methodologies and technologies from traditional biotechnology production to cell therapy manufacturing. Specifically use of bioreactors to scale up cell culture (Chapter 2), the application of online monitoring and control to PSC culture (Chapter 3), the use of a design of experiments (DoE) approach to PSC differentiation (Chapter 4) and cell-cell purification (Chapter 5). Taken as a whole it is clear that the application of traditional biomanufacturing technologies and approaches have much potential when applied to cell therapies, and specifically a PSC derived cell therapy to treat blindness. However much more work is needed to reduce variability in the process to better understand the impact of process parameters on critical quality attributes and how best to approach these in an environment where there is little clinical experience to define the target product profile.

Type: Thesis (Doctoral)
Title: Scale-­up of human pluripotent stem cell-­based therapies for age-­related macular degeneration
Event: UCL (University College London)
Open access status: An open access version is available from UCL Discovery
Language: English
UCL classification: UCL > Provost and Vice Provost Offices
UCL > Provost and Vice Provost Offices > UCL BEAMS
UCL > Provost and Vice Provost Offices > UCL BEAMS > Faculty of Engineering Science
UCL > Provost and Vice Provost Offices > UCL BEAMS > Faculty of Engineering Science > Dept of Biochemical Engineering
URI: https://discovery.ucl.ac.uk/id/eprint/1470390
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