Molecular, gene and cellular therapies

- To understand the concepts of advanced, personalized, and targeted therapy and their implications in the drug R&D process and precision medicine.
- To identify the different types of molecular and gene therapies and to understand the rationale for their development.
- To know and understand the impact of immunotherapy in current therapeutics.
- To learn about the main current clinical applications of molecular and gene therapies and to envision their future potential.
- To know and understand the fundamentals of regenerative medicine, cell therapy, and tissue engineering.
- To know the experimental and clinical applications of regenerative medicine, cell therapy, and tissue engineering and their future perspectives.

A) Lectures (30h)

Section I. Introduction to advanced and personalized therapies (3h)
Unit 1. Fundamental concepts of personalized therapies and advanced therapies.
Unit 2. Introduction to the drug research and development process.

Section II. Molecular therapies (4h)
Unit 3. Introduction to personalized/targeted molecular therapies.
Unit 4. Clinical applications of personalized/targeted molecular therapies.

Section III. Gene therapy. Interference RNAs and other non-coding RNAs (5h)
Unit 5. History and fundamentals of gene therapy.
Unit 6. Gene therapy tools.
Unit 7. Therapeutic RNAs. Types and delivery strategies. Applications.
Unit 8. Clinical applications of gene therapy. Authorized applications in Europe and the USA and the evolution of clinical trials.

Section IV. Immunotherapy (8h)
Unit 9. Introduction to immunotherapy: from biologics to advanced cellular immunotherapy.
Unit 10. Immunostimulant and immunosuppressant drugs. Immune checkpoint inhibitors.
Unit 11. Vaccines. Technological evolution and current applications. Viruses as therapeutic agents.
Unit 12. Advanced cellular immunotherapy: the paradigmatic case of CAR-T cells.
Unit 13. Beyond CAR-T. Other immunotherapies based on T cells and other cells of the immune system.

Section V. Cell therapy (10h)
Unit 14. Introduction to cell therapy and regenerative medicine.
Unit 15. Stem cells. Types, sources, and applications.
Unit 16. Cellular reprogramming in regenerative medicine. Fundamentals and applications.
Unit 17. Tissue engineering and biomaterials. Fundamentals, strategies, and applications.
Unit 18. Ethical and legal considerations of stem cell research.

B) Seminars (3h)

S1. Regulatory aspects of the production and marketing of cell therapies. GMP manufacturing.
S2. Safety aspects in advanced and targeted therapies.
S3. Visit the Advanced Therapies Manufacturing Centre of Galicia.

Basic

Atala A., Lanza R., Mikos T. and Nerem R. (Eds.). Principles of regenerative medicine. 3rd ed. New York: Elsevier Academic Press; 2019. Online access.

Galli M. C., ed. Regulatory Aspects of Gene Therapy and Cell Therapy Products A Global Perspective. 2nd ed. Springer International Publishing; 2023. Online access.

Khan, F. Advances in the application of stem cells: from bench to clinics. Switzerland: Springer; 2021. Online access.

Nóbrega C., Mendonça L., Matos C. A. A Handbook of Gene and Cell Therapy. 1st ed. 2020. Springer International Publishing. Online access.

Slack, J.M.W. The science of stem cells. Hoboken: John Wiley; 2018. Online access.

Complementary
http://www.stembook.org/
http://www.eurostemcell.org/
https://stemcells.nih.gov/
https://learn.genetics.utah.edu/content/stemcells/
https://www.aemps.gob.es/
https://www.fda.gov/
https://www.ema.europa.eu/en/homepage

LEARNING OUTCOMES

Knowledge
Con01 - Identify and relate the structure and applications of polymeric materials and nanomaterials with biomedical and agri-food applications.
Con03 - Know the fundamentals and applications of the main advanced experimental methods and cutting-edge analytical and molecular techniques used in the field of study of Molecular Biosciences
Con06 - Understand the molecular techniques used in the diagnosis and therapeutic approach of various diseases, with emphasis on all those molecular, genetic, and cellular techniques, typical of the so-called personalized medicine.

Skills
H/D02 - Apply safe methods and technologies for the application of new developments in genomics for the study and analysis of molecular mechanisms of antimicrobial resistance.
H/D03 - Ability to design and carry out experiments using advanced instrumental techniques, applying regulations and techniques related to safety and hygiene, waste management, and quality of the results generated within the scope of work.
H/D05 - Apply the knowledge of the field of Immunology to any study of Molecular Biosciences by designing experiments and using the most advanced techniques and tools.
H/D09 - Properly apply the knowledge and techniques acquired to analyse a complex situation or concept and find a way to solve it

Competences
Comp01 - Develop the ability to properly organize and plan work, based on a synthesis and analysis that allow decisions to be made.
Comp04 - That students know how to apply theoretical and practical knowledge professionally and are competent in the approach/resolution of problems both in academic and professional contexts related to Molecular Biosciences
Comp05 - Collaborate in interdisciplinary teams in any work environment, based on knowledge of the environment and current legal regulations.
Comp06 - Develop the ability to learn autonomously with initiative and entrepreneurial spirit.
Comp08 - Be able to communicate conclusions and knowledge in a reasoned way to specialized and non-specialized audiences with clarity and precision, also in foreign languages, mainly English.

Lectures will be taught through master classes, with the support of the blackboard and audiovisual resources, in which reflection and participation of students will be encouraged. The seminars will address topics and carry out activities that complement the knowledge acquired in the master classes. There will be a study outing aimed at the knowledge of professional and working environments, where students learn in person about the reality and way of working in the biotechnology sector on a larger scale than that of the laboratory. Active-participative methodologies, in which students work individually and/or in groups guided by the teachers, will be used to promote autonomous learning. In addition, complementary activities will be carried out, based on problem-solving, gamification, or others, to reinforce learning. The participation and attitude of the students in the activities programmed during the course will be monitored. Tutoring activities will be carried out to guide learning and a public tutoring forum will be set up in the virtual classroom of the subject, as well as other tools for the resolution of doubts.

Regular attendance at lectures and seminars (at least 80%) is required to be evaluated. The knowledge, skills, and competencies acquired in lectures (Con01, Con03, Con06, H/D02, H/D05, H/D09, Comp01, and Comp06) will be evaluated through a final test with objective multiple-choice questions, which will represent up to 60% of the overall maximum grade. Continuous assessment will also be carried out to evaluate the skills and competencies acquired (H/D03, H/D05, H/D09, Comp01, Comp04, Comp05, Compo06, and Comp08), in which class attendance, attitude and participation, and the performance and content of all the activities programmed during the course will be assessed. Continuous assessment will account for up to 40% of the overall grade.

In cases of fraudulent completion of exercises or tests, the provisions of the Normativa de evaluación del rendimiento académico de los estudiantes y de revisión de calificaciones (Resolución del 15 de junio de 2011, DOG del 21 de julio de 2011) will apply.

The subject has an endowment of 4.5 ECTS credits, distributed as follows:
- Lectures: 30 face-to-face hours.
- Interactive (seminars): 3 face-to-face hours
- Tutoring in small groups: 1 hour.
- Exam: 2 hours
- Student's work: 76.5 hours.

It is recommended to carry out an active learning of the knowledge acquired along the development of the syllabus by elaborating organized schemes and making links between one's previous/own knowledge and new knowledge. Participative attendance to the classes is recommended, regular study of the subject with the support of class notes, consultation of the recommended texts or manuals, and tutoring activities for learning guidance and resolution of doubts. Students need to become aware of the importance of understanding the concepts, methods, and techniques described to develop skills and competencies that will be useful in the professional field.

To gain the maximum benefit from the course, it is recommended that students have previous knowledge of immunology, genetics, molecular biology, cell biology, and embryology, English for the management of bibliography/teaching resources, and skills in the use of information and communication technologies. Students will have at their disposal a virtual classroom of the subject in the Moodle platform, with the resources and communication channels it offers. In addition, the Microsoft Teams application and email can be used as a means of communication.