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Advanced therapy medicinal products (ATMPs)

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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. The different strategies applied in gene therapies are presented, as well as, the methodologies to manufacture the vectors. Additionally a general overview is provided on 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 molecular strategies applied in the treatment of diseases

• Gene delivery systems: advantages and disadvantages

• Target diseases and clinical trials

• Vectors used and their manufacture

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

• Organ Bioengineering: Strategies for descelullarization and recullarization of tissues/organs

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 or essay of a given clinical trial or ATMP, chosen by the student from a list of themes timely provided.

Main Bibliography

  •  “Gene and Cell Therapy”, Nancy Smyth Templeton, CRC Press, 4th Edition, 2015.

  • “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.

  • “Regenerative Medicine 2006” (can be downloaded at http://stemcells.nih.gov/info/scireport/2006report.htm)

  • “Biopharmaceutical Processing: Development, Design, and Implementation of Manufacturing Processes”, Edited by Jagschies G, Lindskog E, Lacky K, Galliher P. Elsevier, 2018.

  • “Stem Cells to Model diseases”, Edited by Cell Press, 2012.

  • “Bioreactors for Stem Cell Expansion and Differentiation”, Edited by Joaquim M.S. Cabral, Claudia Lobato da Silva, CRC Press Taylor & Francis Group, Florida, 2018.

  • Recent research and review papers published in high impact peer-review journals (Nature Biotechnology, Trends in Biotechnology, Biotechnology and Bioengineering, Tissue Engineering, Journal of Biotechnology, Gene Therapy, Stem Cells, Cell Stem Cell, etc).

 

 

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