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

Purpose and goal:

Gene therapy is perhaps one of the biggest paradigm shifts of the 21st century. The proposed project can address several critical sustainability issues in manufacturing (e.g. energy consumption, use of antibiotics, generation of large amounts of GM biohazardous waste). Thanks to the project and Vinnova´s support, Combigene hopes to be able to choose a more sustainable manufacturing process for its key project for neuropathic pain, a disease that causes massive, unmet and continuous suffering to more than one billion people worldwide.

Expected results and effects:

The project will evaluate whether plasmid DNA can be safely and reliably replaced by more sustainable synthetic DNA for the production of AAV vectors.

If successful, CombiGene will be able to choose a significantly more sustainable production method for its gene therapy project for chronic pain. Furthermore, supported by collaboration with KTH and, ideally, publication(s) and presentation(s) at international conferences, this route can be made more attractive to other biotech companies and contract manufacturers that cannot afford an evaluative study.

Approach and implementation:

As a first step of the project, suitable contract manufacturers (CDMOs) for viral vectors will be evaluated based on e.g: -cost -quality of the offer (the request for proposal has already been sent to 11 CDMOs) -ability and interest to successfully implement the CombiGene/KTH project plan (including analysis)

Ideally (cost dependent) 2-3 CDMOs will be selected.

The CDMOs will then perform the study (DOE= Design of experiment), perform analysis and send samples to KTH for sequencing with their methodology. Relevant results will be published and presented.