Navigating evolving regulatory CMC guidance in the AAV gene therapy field

Wednesday 09:00 PDT / 12:00 EDT / 16:00 GMT / 17:00 CET

Sponsor

Navigating evolving regulatory CMC guidance in the AAV gene therapy field

With the first AAV vector-driven gene therapy product approvals has come significant evolution in regulators’ guidance and expectations, particularly in the area of product characterization and quality control (QC). Analytical tool innovation is delivering a new depth of understanding of AAV vectors and the impact of elements such as full-empty capsid ratio. But with this new knowledge comes uncertainty, as regulators and developers alike struggle to keep pace with the speed of progress in what is a still nascent and relatively unstandardized field.

This roundtable discussion will feature regulatory, analytical and process experts who will examine some of the key developments in the CMC landscape for AAV-based gene therapy. The panel will dissect current, and likely future, regulatory guidance, offering advice to those seeking to avoid the setbacks that have hindered a number of products in late-stage development over recent times.

Attend this webinar to:

Better understand recent US and European regulatory guideline evolution impacting the AAV CMC area
Receive insights into the process and analytical tools and technologies that can support a robust gene therapy product characterization and CMC strategy
Appreciate what needs to be done in early development to avoid CMC-related pitfalls later on

Yan Zhi

Director, Cell and Gene Therapy Product Owner, Process Engineering, CSL Behring

Christine Le Bec

Head of CMC Gene Therapy, Sensorion

Christine Le Bec joined Sensorion Pharma in early 2020 as Head of CMC Gene Therapy. She is responsible for all CMC activities, including pre-clinical development, CMC transfer to CMOS, manufacturing and supplying of Phase 1 and 2 clinical trails. Before joining Sensorion Pharma, she worked for more than 20 years at Genethon in the field of Gene Therapy vectors (AAV, Lentivirus, Baculovirus) for rare diseases. She has a strong expertise in the development, qualification, validation of analytical methods for product characterization, release testing of gene therapy products and in stability studies. She has also a solid knowledge of International regulations and reviewing CMC documents for clinical trial applications.

Michael Brewer

Director, Global Principal Consultant, Regulatory, BioProduction Group (BPG), Thermo Fisher Scientific

Michael Brewer is the Director, Global Principal Consultant, Regulatory for the BioProduction Division (BPD) at Thermo Fisher Scientific. In this role, Michael is responsible for providing global support to BioProduction customers and serving as the regulatory thought leader and expert across all technology areas within BPD. Prior to moving to this role, he led the team responsible for product applications including Microbiology, Analytical Sciences and Quality control. The products are fully integrated, solutions for Glycan profiling, Bacterial and Fungal identification, Mycoplasma and Viral detection and host cell DNA and protein quantitation. Michael has over 30 years experience in the Biopharma industry, including, Scios, Synergen and Amgen in a variety of roles including Discovery Research, Analytical Sciences and Quality Control. Prior to joining Thermo Fisher Scientific, he led a group at Amgen that developed qualified, validated and implemented molecular methods for host cell DNA quantitation, contaminant (Mycoplasma, Virus and Bacteria) detection, contaminant identification, strain typing and genotypic verification of production cell lines.