Express Pharma

Touchlight introduces mbDNA: A circular single-stranded DNA (ssDNA) platform enabling superior gene editing

mbDNA’s design supports 1-20kb gene insertions and aims to offer more knock-in efficiency across diverse cell types

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Touchlight announce the commercial launch of their new technology for non-viral gene editing, mbDNA (megabulb DNA). Its novel circular single-stranded DNA (ssDNA) structure features a customisable stem region and a fully user-defined sequence free of phage or bacterial elements.

mbDNA’s design supports 1-20kb gene insertions and aims to offer more knock-in efficiency across diverse cell types, in comparison to double-stranded DNA (dsDNA) and competitor ssDNA formats, whilst maintaining high cell viability. 

CRISPR-nuclease technologies have transformed gene editing by enabling highly precise genetic corrections, unlocking unprecedented therapeutic possibilities and redefining how we approach disease targeting and treatment. Nonetheless, there are still challenges to be addressed in translating this technology into viable new products.

Historically, AAV (Adeno-Associated Virus), has been the vector of choice for HDR gene-editing applications. However, several AAV limitations are becoming increasingly evident, including restricted payload capacity, high manufacturing costs and lengthy timelines. Non-viral DNA platforms offer greater design flexibility and with manufacturing processes that can scale without excessive complexity.

Touchlight’s novel mbDNA platform is a single-stranded and circular, offering reduced cytotoxicity and highly improved editing efficiencies. Indeed, mbDNA can consistently achieve knock-in (KI) rates of 75 per cent in primary human T cells, with GMP coming soon.

mbDNA is compatible with multiple delivery methods, including electroporation, nucleofection, and lipid nanoparticle formulations, underscoring its versatility and making it an ideal choice for a wide range of gene-editing workflows. This new DNA architecture can also be used as an episomal expression vector in transient non-viral gene therapy (NVGT) settings with the promise of improved tolerability due to its single-stranded nature.

 

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