Touchlight Genetics and National Physical Laboratory (NPL) have secured funding from Innovate UK under their Innovative Technologies: Nucleic Acid Medicines Manufacture, Round 2 call to develop a circular, single-stranded genome editing template known as MegaBulb DNA (mbDNA).
Increasing clinical activity with genome edited products is leading to significant demand for safe, efficient and manufacturable non-viral nucleic acid templates for targeted genome insertions, as this will replace the need for viral vectors.
mbDNA's place in the industry
Touchlight’s mbDNA shows low cellular toxicity and achieves high gene-length knock-in efficiencies, outperforming other non-viral technologies and rivalling viruses. mbDNA’s enzymatic manufacture, which leverages Touchlight’s established technologies & capabilities, allows for a robust and scalable process suitable for rapid development towards GMP production – and addresses a major bottleneck for non-viral gene therapy (NVGT) development.
This funding will enable Touchlight, with the support of NPL, to comprehensively characterise mbDNA, develop relevant analytical methods and rapidly scale up manufacturing procedures.
The project will inform specific metrics for the performance attributes of mbDNA and its manufacturing, which will be assessed using NPL’s reference materials and procedures. The project results will facilitate a mechanism for more efficient ssDNA production through improving mbDNA’s manufacturability, as well as increasing the productivity and flexibility of Touchlight’s already established GMP manufacturing process.
Karen Fallen, Chief Executive Officer of Touchlight, said: “We are excited to partner with NPL to further develop our innovative mbDNA technology. mbDNA’s superior knock-in efficiency and low toxicity, combined with our established GMP synthetic DNA manufacturing expertise, has the potential to address current supply limitations for long single-stranded DNA, strengthening the UK's market position in the nucleic acid manufacture market for gene editing.”