Material Requirements to Support Gene Therapy Development

Manufacturing processes for gene therapy products are generally low yielding compared to production processes for other biologics. The material requirements to support analytical, comparability, and stability activities at all stages of development could exceed the requirements for clinical dosing. This talk will highlight some of the key material requirements at different stages of development and discuss options for maximizing use of available material.

Presented by Clare Blue, Director of Analytical Development at Biogen

Clare Blue holds a degree and PhD in Molecular Microbiology from the University of Glasgow, Scotland, UK. She has industry experience ranging from viral / vector manufacturing, biosafety testing, analytical development, QC and process development. Clare has been involved in AAV gene therapy for over 7 years, initially at Nightstar Therapeutics where she held senior roles overseeing the manufacturing and analytical strategy of phase II/III AAV products for treating retinal diseases.  In her current role as Director of Analytical Development, Biogen, she is involved in all aspects of clinical AAV product development. Clare is also an advocate for sustainable development.

Followed by James Wilkinson, VP of Sales and Customer Success at Refeyn

James studied a BA in Natural Sciences followed by a PhD in protein chemistry at the University of Cambridge studying the biophysical properties of ankyrin repeat proteins. After a short postdoc he joined NanoTemper Technologies in Munich in 2012 when the company was in its infancy. As one of the first application and sales specialists he stayed for 6 years developing the technology, launching new products and ended up running the UK subsidiary with responsibility for the UK and global distributor businesses. After working for a number of other start up companies he joined Refeyn in 2019 as the Sales Director, now as VP of Sales and Customer Success. Refeyn are an Oxford spin out company and have recently developed a methodology called mass photometry to analyse biomolecules. James is focussed on introducing the technology globally and supporting our rapidly growing customer base.

Sponsored by Refeyn

Refeyn, the mass photometry pioneer, offers new capabilities to characterise the function, structural composition and dynamics of biomolecules. Refeyn instruments measure the mass of individual molecules directly in solution, quickly and simply revealing the true behaviour of molecules in near-native environments. The diverse applications of mass photometry include quantitative analysis of sample purity and homogeneity, biomolecular complex assembly and disassembly, the strength and kinetics of molecular interactions, and more. Refeyn was spun out of Oxford University in 2018 to make mass photometry available globally. Its technology has now been rapidly adopted across academia and industry, where it is transforming biomolecular characterisation. The Two^MP, Refeyn’s mass photometer, brings the technology into everyday laboratory life, enabling molecular measurements with unprecedented speed and simplicity. It uses proprietary technology to detect the light scattered by single molecules in solution, delivering label-free mass measurements over a broad mass range with high precision and exquisite sensitivity.