CombiGene and KTH have jointly received a grant from Vinnova of SEK 1 million to investigate the possibilities of replacing plasmid DNA with synthetic DNA, which would provide significant environmental and safety benefits, including that antibiotics and resistance genes can be avoided.
Gene therapy is a new paradigm shift in drug development, but so-called AAV vector manufacturing (based on adeno-associated virus) is a very complex and costly process, which leaves room for innovation.
The project, run by CombiGene and KTH together, will evaluate whether synthetic DNA can replace plasmid DNA as starting material in CombiGene’s project on chronic pain.
“If we succeed, it can provide significant benefits from a sustainability and product safety perspective. The environmental impact is also reduced by avoiding the use of antibiotics and antibiotic-resistant genes,” says Sebastian Bauer, Director CMC at CombiGene and the person responsible for the overall coordination between CombiGene, KTH and the company selected for the manufacturing.
“There are many advantages to synthetic DNA. Among other things, only one-tenth of the production volume is needed, energy consumption is lower, and production is significantly faster – five days compared to several weeks for traditional fermentation. In addition, the risk of E. coli contamination is reduced, which improves product safety and enables a more accurate DNA sequence. Even the safety of the final drug product can be improved,” explains Sebastian.
New technology optimizes gene therapy production
The main purpose of the project is to show that this new technology provides the same or better quality as with traditional production. The project is a spinoff from GeneNova, a research initiative led by Professor Johan Rockberg at KTH, which includes CombiGene and several other gene therapy companies. It is the first concrete spin-out project between CombiGene and KTH.
“The goal of the GeneNova project is to, with the help of academic and industrial excellence, jointly research, develop and validate platforms for the sustainable production of gene therapy drugs. This is exactly what is happening now in a concrete collaboration, part-financed by Vinnova, with a successful Swedish gene therapy company,” says Johan Rockberg, Professor in Antibody Engineering and Directed Evolution at KTH Royal Institute of Technology in Stockholm.
In addition to Sebastian, Annika Ericsson, CombiGene’s Chief Scientific Officer, will also be involved in the project, which is expected to be completed in a year.
“Innovations that contribute to reducing costs without compromising on sustainability and product safety, but perhaps improve these parameters, are obviously attractive to companies that will further develop a technology into a finished product and then start scaling up production. One of CombiGene’s strengths is that we have the ability to combine innovative ideas with thinking all the way to the market, and thus understand the needs that arise later in the chain. We are hopeful that in the project we will be able to show that this new technology can contribute to both better and more cost-effective gene therapy”, concludes Sebastian.