Exploring and overcoming the challenges of cell and gene-modified cell therapy CMC

Tuesday 08:00 PDT / 11:00 EDT / 16:00 BST / 17:00 CEST

Sponsor

Exploring and overcoming the challenges of cell and gene-modified cell therapy CMC

Cell and gene-modified cell therapies hold immense potential for transforming healthcare, but they come with unique complexities in Chemistry, Manufacturing, Controls (CMC) that can result in setbacks, such as manufacturing comparability failure, clinical holds, product approval delay, and even manufacturing issues during post-market surveillance. As more therapies are being developed, it is crucial to explore and overcome the CMC challenges that can impede program and market progress.

Attend this webinar to:

Understand the importance of establishing a robust supply chain for cell and gene-modified cell therapies.
- Learn about key considerations in sourcing starting and raw materials, including quality control measures and regulatory compliance.
Gain insights into phase-appropriate and product-specific analytical development for cell and gene-modified cell therapies.
- Discover strategies for designing and implementing analytical methods to ensure comprehensive control of critical quality attributes throughout the product lifecycle.
Explore effective scaling-up techniques for cell and gene-modified cell therapies.
- Learn about best practices for process optimization, technology transfer, and manufacturing scale-up to ensure successful commercialization while maintaining product quality, safety, and efficacy.
Acquire expert guidance on developing a potency assay framework for complex advanced therapies.
- Understand the challenges associated with assessing potency in advanced therapies and learn how to design and validate robust potency assays to support product characterization and clinical development.

Julia Sable

Associate Director Project Management, Viral Vector Process Design (PDe), Kite

Research and Process Development Lead for early and late phase Gene Therapy (U Penn and Columbia) and Cell Therapy (Legend Biotech and Tmunity). Upstream and Downstream PD lead for Oncolytic Virus (OV) Therapies (Merck) High Throughput (HTP) virus characterization and potency assay development. Analytical Development for stability and release. End-to-end bioprocessing for lentivirus (LVV), retrovirus, adeno-associated virus (AAV), g-deleted rabies (RABVdG) platforms for preclinical to IND and BLA filings. Viral Vector purification, process optimization, process validation for adherent, fixed-bed, microcarrier, and suspension Bioreactors, Clarification (TFDF), Chromatography, TFF, and sterile filtration. Expert in advanced microscopy (Confocal, TIRF, STED, 2-P, SLM and E-Phys setups).Lab Manager and Senior Research Scientist for Dr. Michael P. Sheetz (Lasker Winner); Dr. Charles Rice (Lasker and Nobel Prize winner) and non-tenure-track faculty member at Columbia University as Director of Virology for the Zuckerman Institute.

Larry Bellot

Scientific Advisor, Cell & Gene Therapy, Charles River

Larry Bellot is a Scientific Advisor for the Charles River Cell and Gene Therapy CDMO business, having held previous positions as Analytical Sciences and Method Validation Manager and Senior Principal Scientist within the Manufacturing, Science & Technologies (MSAT) group. Prior to Cognate BioServices’ acquisition by Charles River in 2021, Larry also gained experience as MS&T IV, QC Tech Transfer Scientist, and QC Analytical Scientist at the established cell therapy CDMO. A dedicated coach and mentor, Larry received his PhD with honors from The University of Texas Medical Branch at Galveston and has foundational degrees in Industrial Biotechnology and Microbiology and has a vast and varied industrial biotechnology experience.

Karen Doucette

Cell and Gene Therapy Navigator, Charles River

Karen Doucette currently serves as Cell and Gene Therapy (CGT) Navigator at Charles River Laboratories. Karen plays a critical role in partnering with clients to map the development journey from discovery through commercial manufacturing, with a focus on providing process development guidance to avoid common, time-consuming pitfalls. Karen has over 20 years of experience with biopharmaceutical CROs and CDMOs, spanning pre-clinical and clinical research, development and manufacturing. She has been instrumental in collaboratively driving expansion of CGT service portfolios and aligning research innovation with customer need. Karen received her BS in Animal Science from the University of Delaware and completed her MBA at Goldey-Beacom College.