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The first part of this lecture describes the design and operation of a production facility for adeno associated viral vectors used for in-vivo gene therapy. The second part will provide an introduction into the “Good Manufacturing Practice” framework covering the entire pharmaceutical product…
Like any new drug, gene and cell therapeutics must undergo both safety studies and a product development phase before they can be used in humans for the first time. The preclinical safety studies include various in-vitro and in-vivo animal studies to assess potential risks such as toxicity,…
This lecture highlights current ethical debates in stem cell research, including the use of human embryonic stem cells for research, perspective of cloning animals or humans, human germ line editing, the increasing costs in personalized medicine, human animal chimeras, and synthetic embryos.
Advanced Therapy Medicinal Products (ATMPs) are a specialized category of medicinal products for human use that are based on genes, tissues or cells and offer groundbreaking new opportunities for the treatment of disease and injury.
This lecture will give an overview on the rapidly evolving field of Gene and Cell Therapies (GCT) with its challenges for manufacturer and opportunities for patients.
This lecture explores the clinical use and therapeutic potential of CAR T-cell therapy.
Following a brief definition of the term “Gene Therapy,” the lecture provides an overview of the vector systems commonly employed for gene transfer into target cells. The remaining lecture focuses primarily on retroviral vectors, with particular attention given to the retroviral replication cycle…
This lecture offers a comprehensive introduction to the field of stem cell biology. It begins by outlining fundamental characteristics, including the capacities for self-renewal and differentiation, and subsequently addresses the classification of stem cell subtypes according to their developmental…
In this lecture, life science entrepreneur and investor Marius Rosenberg shares his experience of commercializing life science innovation via the creation of one’s own startup.