 | | Advanced therapy medicinal products (ATMPs) Coordinators
Margarida Serra
Ana Sofia Coroadinha Objectives
This Curricular Unit provides an in-depth education in the cutting edge and rapidly developing field of Advance Therapy Medicinal Products (ATMP). It covers all aspects of this subject, including the basics in biomedical science, current and developing technologies and applications of Gene and Cell Therapies. This unit also aims to
introduce the principles and applications of stem cells and biomaterials science, engineering and biology related to Tissue Engineering and Regenerative Medicine and provide a general overview of the legal and ethical restrictions placed by the regulatory agencies for the development, production and delivery of novel ATMPs. Syllabus
Definition of ATMPs
Gene therapy
• Gene therapy: the concept
• Principles and challenges in manufacturing and delivery of gene therapy products
• Gene delivery systems: advantages and disadvantages
• Target diseases and clinical trials
Stem Cells and Cell Therapy
• Stem cell types, sources and characterization
• Principles and challenges of cell therapies: autologous versus allogeneic
• Stem Cell Bioprocessing
• Innovative approaches for stem cell isolation, expansion, differentiation, purification and cryopreservation
• Case studies and clinical trials
Biomaterials and Tissue Engineering
• Materials for biomedical applications
• Tailoring biomaterials and scaffolds for tissue engineering
• Design of smart biomaterials and carriers for the controlled release of bioactive agents
Regulatory Procedures and Ethical Challenges of ATMPs
• EMA and FDA regulatory guidelines for development of ATMP
• Case studies of approved ATMPs Evaluation
The evaluation procedure will be performed at the end of the curricular unit and includes a short presentation of a given clinical trial followed by discussion, chosen by each group from a list of themes timely provided. Main Bibliography - “Gene and Cell Therapy”, Nancy Smyth Templeton, CRC Press, 3rd Edition, 2009.
- “Tissue Engineering”, Palsson B.O. and Bhatia S.N., Pearson Prentice Hall Bioengineering, 2004.
- “Handbook of Stem Cells”, Lanza R. et al., Elsevier Academic Press, 2004.
- “Stem Cell: Scientific Progress and Future Research Directions” (can be downloaded at http://stemcells.nih.gov/info/2001report/2001report)
- “Regenerative Medicine 2006” (can be downloaded at http://stemcells.nih.gov/info/scireport/2006report.htm)
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