GCT Media Library

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. 

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, carcinogenicity, immunogenicity etc. as well as the fate in an organism and a possible dose to ensure patient safety. In the product development phase, the reproducibility of defined specifications of the clinical product is ensured by establishing a sufficient understanding of materials and processes. Both the preclinical safety studies and the product development program result from the product-specific risk assessment and the results obtained during the studies.

Principles of Good Manufacturing Practice (GMP)

GMP is a set of guidelines for the manufacture of medical-pharmaceutical products that are administered in or on humans. The aim is to ensure the defined quality and safety of a product in a reproducible and consistent manner for an approved therapy or a therapy under development. The regulations consist of nine different areas that cover all aspects of production. GMP is a global standard that is certified and continuously monitored by authorities on a product-specific basis. Each manufacturer and its management is and remains responsible for compliance with GMP standards and the manufactured product. GMP is a continuous process that also contributes to the continuous improvement of the quality and efficiency of manufacturing.
References

Preclinical safety Guidance

1. ICH S Guidelines (S1-S13)
2. ICH M3 (R2)
3. ICH M6
4. ICH M7
5. OECD Guidelines for testing of chemicals, section 4
6. A comprehensive guide to toxicology in non-clinical drug development. 2nd Edition (2017) Ed: Faqi, A.S. Academic Press. ISBN: 978-0-12-803620-4    

Product and process development Guidance

1. ICH Q8 (R2)
2. ICH Q9
3. ICH Q10
4. Guidelines on Good Manufacturing Practice for Advanced Therapy Medicinal Products

GMP Guidelines  

1. Eudralex, Vol 4: Good Manufacturing Practice
2. ICH Q Series (Q1-Q14)
3. ICH Q8(R2)
4. ICH Q9
5. ICH Q10
6. Guidelines on Good Manufacturing Practice for Advanced Therapy Medicinal Products