Course Outline

Course Information

• The course begins at the time stated at the top of the page
• The course is broken up into modules outlined below
• There will be breaks between modules

Session 1 | Day 1

20 May 2024

8 October 2024

150 mins | 1:00 PM UK

Overview of the CGT industry

• Brief intro to history of gene therapy and recent approvals
• Viral vectors and their importance in gene therapy
• Brief intro to history of cell therapy and recent approvals
• CGT/ATMP manufacturing process and analytics
• CGT/ATMP clinical development and bioanalytics

Bioanalytical basics and challenges of CGTs

• Overview of main methodology used to measure PK: flow and PCR
• Immunogenicity of gene therapies
• Special focus on pre-existing antibodies to viral vectors
• Unwanted insertion events of gene therapies and how to monitor
• Tracking of cellular therapies
• Regulatory guidance on bioanalytical assays in the CGT space

Session 2 | Day 2

21 May 2024

9 October 2024

150 mins | 1:00 PM UK

Analytical basics and challenges of CGTs

• Product Release assays in the CGT space
• Special focus: Potency assays and the importance of mode of action
• The challenges of multiple mode of actions of cellular therapies
• Regulatory guidance on potency assays in the CGT space

Special focus: CAR T cells

• The rise of the CAR T cell and the need for future generations
• Clinical impact of CAR T cells with regards to patients’ safety
• CAR T cell future trends such as the off the shell CAR T cell
• Biomarker analysis

Dr. Melody Janssen is an expert in bioanalysis and immunogenicity testing of biopharmaceuticals with in-depth experience as a project manager, trainer and senior expert in biotech/CGT companies. She has worked on numerous biopharmaceutical products and is an expert on all areas of biopharmaceutical development and manufacturing.
Dr. Janssen has extensive experience of the regulatory frameworks (EMA, FDA etc.) for biopharmaceutical development/manufacturing. Ultimately Melody is aware of the many challenges biopharmaceutical companies face when developing a therapeutic product.

She is frequently invited to speak at conferences on immunogenicity and bioanalytical testing, where she also leads conference specific workshops and seminars. She has shared her knowledge through trainings for over 10 years focusing on assay validation for bioanalytical, potency and immunogenicity assays.

For the last years, Dr. Janssen has been working as a freelancer for projects including non-clinical and clinical immunogenicity assessment, leading various late pre-clinical candidates to FIH, production of vaccines and biologics and teaching bioanalytical method validation workshops.

Her educational background is in infectious diseases and immunology, with a PhD in pharmaceutical sciences investigating the effect of protein aggregates on immunogenicity.