Tuesday, 1 September - 8.00-11.00AM
Developing Relevant In Vitro Potency Assays: Walking Through Case Studies
Developing a relevant set of potency assays is challenging due to the high level of molecular complexity for gene therapies with their mechanisms of action (MoA) not always being fully understood. Potency is considered a critical quality attribute (CQA) and is required to assess product quality and consistency. and the implementation of relevant assays is one of the main challenges for gene therapy developers and regulators throughout product development.
By attending this workshop, you will:
- Review case studies from different products potency assay development
- Explore alternative approaches for showcasing potency
- Streamlining development of functional potency assays and exploring optimised validation timing
Workshop Leader to be confirmed
Tuesday, 1 September -11:30-2.30PM
A Deep Dive into Lentiviral-Specific Analytical Techniques
The analytical development challenge associated with lentiviral vectors differ and therefore optimised analytical methods are essential for novel lentiviral vector-based gene therapeutics manufacturing throughout development. This workshop will provide an overview of the assays used to ensure full lentiviral vector characterisation, quality control and stability testing, and preparing CMC components for regulatory filings.
Attend this workshop to:
- Discuss the relative advantages and disadvantages of lentiviral and AAV vector approaches in a variety of indications
- Understand how analytical strategies differ in the context of different viral vectors
- Review case studies detailing optimised approaches for lentiviral vector characterisation, quality control and stability testing
Workshop Leaders: Tushar Patel, Scientist I, Vector Characterization, Bluebird Bio and Jennifer Lin, Senior Scientist, Orchard Therapeutics
Tuesday, 1 September - 3.00-6.00PM
Determining Comparability between Analytical Methods in a Development Phase-dependent Manner
Whatever stage of development you’re currently at, progressing to the next phase and adapting your analytical strategy is a key challenge you will need to navigate. This is all in the context of increasing regulatory scrutiny and uncertainty over the analytical and comparability strategies required to satisfy the regulators.
Attend this workshop to discuss these topics in more detail:
- Dissecting the transitions between early and late stages and developing effective bridging studies
- Dispelling uncertainties around the changes that can be made to processes while maintaining analytical comparability to satisfy regulatory agencies
- Understanding how analytical methods need to evolve and improve as development reaches later stages
Workshop Leader: Sushmita ‘Mimi’ Roy Executive Director, Analytical Sciences BioMarin and Christopher Bravery, Founder, Consulting on Advanced Biologicals Ltd