Exploring key regulatory considerations to ensure patient safety in the gene therapy space

Leigh Shaw is VP, Regulatory Affairs and Quality Assurance, at SpliceBio and has 30 years of industrial experience in the development of gene therapies and therapeutic vaccines for a range of ophthalmic, neurological, and orphan indications. Over the years, he has managed applications at all stages of development in all major markets, including the EU, UK, US, Canada, Australia, and Japan. He has been a visiting lecturer on regulatory affairs at Cambridge University for more than 10 years and was previously a member of the Editorial Panel for Regulatory Rapporteur, the professional journal of The Organisation for Professionals in Regulatory Affairs (TOPRA).

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.

María Orío is the Director of Regulatory Affairs at Viralgen, where she leads regulatory strategy and compliance for AAV-based gene therapies. Prior to joining Viralgen, she held key regulatory roles at several companies, contributing to the development and approval of innovative therapies. María holds a degree in pharmaceutical sciences and specialized training from CESIF. Her extensive experience ensures alignment with global regulatory standards, supporting innovative treatments through every phase of development.

Nathalie Clement, Senior Vice President of Vector Development at Siren Biotechnology, San Francisco, CA, USA, has over 25 years of experience in the gene therapy field with leading expertise in AAV viral vector manufacturing. Nathalie has led AAV process development and manufacturing for R&D POC, IND-enabling toxicology, and IND studies and currently leads CMC operations to bring clinical candidates to patients.