H5
SynTra – Synthetic Transposons for Non-Viral Gene Transfer
Dr Michael Fichtner, Max Delbrück Center Berlin
Max Delbrück Center Berlin
Challenge
Personalised gene therapies represent a promising treatment option against previously untreatable diseases such as monogenic disorders and cancer. However, standard methods for producing gene therapy vectors come with serious limitations. Lentiviral vectors—commonly used to deliver genetic material into target cells—carry a significant risk of insertional mutagenesis, which can lead to severe side effects, including tumour formation. Moreover, lentiviral systems are complex to manufacture and face stringent regulatory hurdles, making production both time-consuming and extremely costly. Despite their clinical successes, the lengthy manufacturing processes and high production costs—reaching up to €500,000 per dose—still stand in the way of making these therapies widely accessible. Non-viral technologies, like the Sleeping Beauty Transposon systems or CRISPR/Cas, can reduce the complexity of the manufacturing process, significantly, thus lowering the costs associated with it. Nonetheless, for truly personalised therapies, in which every patient receives a tailor-made therapeutic gene, the production of the initial DNA vector remains a major roadblock. Non-viral gene delivery technologies such as the Sleeping Beauty transposon system and CRISPR/Cas can substantially reduce the associated costs of gene therapy manufacturing by simplifying production workflows. However, the shift toward fully personalised therapies—where each patient receives an individual therapeutic gene—still faces a key technical hurdle: the efficient, scalable, and regulatory-compliant generation of the initial DNA vector.
Technology
We’ve engineered a fully synthetic, automated platform for cell-free manufacturing of circular DNA vectors, eliminating the need for cell-based production. Our process delivers custom-designed vectors in under three days, dramatically accelerating development timelines. The SynTra technology integrates these synthetic vectors with the Sleeping Beauty transposon system, creating a scalable foundation for truly personalised cell therapies, while substantially reducing both production costs and time-to-patient.
Commercial Opportunity
The market for personalized gene therapies is projected to grow from approximately USD 6 billion today to more than USD 30 billion by 2033. Within this space, T-cell receptor (TCR) therapies—representing a more personalized and precise approach than CAR-T therapies—are expected to see particularly strong expansion, reaching over USD 4 billion by 2035 with an estimated CAGR of 50%. This trajectory underscores the substantial mid- to long-term commercial potential of our technology platform.
Development Status
Our production process is set-up and we have a first proof-of-concept. We were able to show, that our synthetic circles perform evenly well as the current gold-standard vectors (Minicircles and Nanoplasmids) in T-cells. We are open to collaborate to produce personalised vectors for interested parties.
Patent Situation
The Sleeping Beauty platform is patented in Europe and the US (EP2160461, US9228180). A patent application for the SynTra technology is currently being developed.
Further Reading
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