More sustainable & safer gene-therapy AAV upscale-manufacture with synthetic DNA- feasibility study

Important results from the project

The goal of the project was to evaluate the possibility to use synthetic DNA instead of plasmid DNA in the production of adeno-associated viruses for gene therapy. The results are still being evaluated but confirms the possibility to reach similar production levels, which enables more sustainable production of gene therapy. Furthermore, a method för DNA sequencing of gene therapeutic products was developed.

Expected long term effects

The project demonstrates the possibility of producing gen therapies with syntethic DNA instead of plasmid DNA, which could lead lower production prices. The developed sequencing method has the potential to improve quality control of gene therapeutic products, both routinely on production batches and during development. This can lead to better and safer gene therapies being developed and guaranteeing more uniform product quality.

Approach and implementation

The project was carried out as a collaboration between multiple partners with distinct tasks. Combigene (now Lärkberget) together with KTH and Zyneyro have been in charge of the project with different primary tasks and responsibilities, while several CDMOs have been contracted for production for evaluation and comparison by the main partners. Technology development and analysis have primarily been done at KTH with support from Combigene.