Cambridge Healthtech Institute ’s 4th Annual
Gene Therapy CMC and Analytics
Regulation and Analysis of Vector-Based Gene Therapies
August 26-27, 2020
One of the biggest challenges for AAV and lentivirus gene therapy products is establishing an appropriate CMC and analytical strategy to support product manufacture, release, stability, comparability and characterization at different stages of development.
Cambridge Healthtech Institute’s Gene Therapy CMC and Analytics meeting uncovers the practical challenges facing the analysis, characterization, and quality of viral vector-based gene therapies for clinical and commerical supply, with dedictated sessions on CMC strategy, regulatory requirements, orthogonal analytical strategies, new analytical methods, particle detection, stability, and the impact of machine learning on advancing vector safety and quality.
Cover will include, but is not limited to:
- CMC and analytics
- CMC requirements from Phase I-Phase III
- Analytical development of gene therapies
- Potency assay development and validation
- Gene therapy analysis and characterization – MS, NMR, HPLC
- Particle sizing
- Plasmid production, aggregation, and purity
- Stability testing – ancillary materials, lipids, carbon source of producer
- Comparability following process change
- Formulation, stability, and delivery
- Gene editing/CRISPR techniques in gene therapy
- Gene editing and delivery
- Vector design, selection, and development
- Delivery strategies for gene therapies – viral and non-viral delivery systems
- Determining whether the vector is full or empty
- Molecular biology – RNA, DNA, Synthetic, Peptide
* The program is subject to change without notice, due to unforeseen reason.