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.