Accelerating gene therapy development: from concept to clinic

Thursday 08:00 PST / 11:00 EST / 16:00 GMT / 17:00 CET

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Accelerating gene therapy development: from concept to clinic

As gene therapy pioneers begin to tackle systemic diseases and those with a large patient cohort, their focus must increasingly be set on the twin goals of exceptional AAV yields and quality, while constantly scanning the horizon for future technologies to help improve targeted delivery, improve process robustness and decrease cost of goods. In this case study, we present OXGENE’s work with Innervate Therapeutics, who are developing gene therapies for Parkinson’s and Motor Neuron Disease, that will be delivered directly to the brain via convection-enhanced delivery (CED). This is an emerging therapeutic strategy for the treatment of neurological disorders; when delivered via CED, biologics and chemotherapeutics can achieve homogeneous distribution within a targeted brain structure while avoiding systemic immunogenicity and off-target side effects. OXGENE has been supporting Innervate Therapeutics to develop their lead candidate, a gene therapy for motor neuron disease, making use of OXGENE’s transient expression platform. This combines proprietary XAAV™ plasmids and a GMP-banked clonal suspension HEK293 cell line, optimised for maximum viral titre, high per cell rAAV productivity and a high percentage of full capsids.

In this webinar, we demonstrate how OXGENE’s molecular biology and process development services supported Innervate Therapeutics’ pre-clinical research, accelerating their progress from laboratory research towards clinical trials. We then introduce the advantages of our GMP plasmid and viral vector manufacturing platforms, before discussing novel technologies under development to further improve cell-specific delivery of gene therapies, optimise transgene expression and improve scalability and robustness of AAV production, while reducing manufacturing costs.

Steven Gill

Founder and Director at

Qian Liu

Head of Biomanufacturing Services at OXGENE

Dr Qian Liu has a background in cell and molecular biology. She joined OXGENE in July
2017 as a Cell Line Engineering Scientist. She joined OXGENE in July 2017 as a Cell Line
Engineering Scientist, and now leads all of OXGENE’s biomanufacturing services. Prior to
joining OXGENE, Qian was a postdoctoral researcher in the field of Regenerative Medicine,
involved in bioartificial liver development and investigating the mechanisms of stem cell
differentiation in Loughborough University and the University of Nottingham, respectively.

Ryan Cawood

Chief Scientific Officer at OXGENE

Ryan founded Oxford Genetics in 2011, after earning a first class degree in genetics and a PhD from Oxford University. The idea behind the company was to simplify and standardise the process of DNA engineering using a proprietary DNA plasmid platform called SnapFast™ that allowed researchers – for the first time – to assemble complex sections of DNA as simply as ‘molecular Lego’. Ryan used his background in genetic engineering and virology to guide and grow the business through a series of strategic changes that explored how further development of the SnapFast™ platform through in house research and development could help overcome multiple challenges in the development of new biologics.
This culminated in a rebrand to OXGENE in 2019, as the company redefined itself as a leading solutions provider, using a combination of proprietary technologies to address multiple pinch-points on the journey through design, discovery, development and manufacture of a novel biologic.