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E1 - Nonviral Therapeutic Gene Delivery and Synthetic/Molecular Conjugates

1717: A Novel, Synthetic DNA for Cell and Gene Therapy Application

Type: Poster Session

Poster Board Number: 1717
Presentation Details
Session Title: Friday Posters: Nonviral Therapeutic Gene Delivery and Synthetic/Molecular Conjugates

In the last decade, the field of cell and gene therapy has undergone a remarkable expansion with unprecedented growth in research, clinical trials, and therapeutic applications. Central to this transformative development is the increased recognition of DNA as the essential input material. As demand for advanced therapies continues to rise, there is increasing need for efficient and scalable platforms for manufacturing DNA constructs that are used directly as therapeutic agents or as input material for therapeutic products.
We have developed a novel optimized synthetic linear double-stranded DNA with customizable hairpin-ended structures (e.g. synthetic and natural inverted terminal repeats) for cell and gene therapy applications. Our proprietary one-pot, enzymatic and cell-free production process has a small footprint, seamless scalability and allows production of synthetic DNA constructs of variable size and sequence complexity without residual bacterial backbone sequences. Additionally, as the generation of the hairpin ends is not sequence specific, the DNA platform allows application-oriented customization of the hairpin structures on both DNA ends.
Here, we show that our cell-free, enzymatic DNA production process offers high product purity and flexibility coupled with faster production times and minimal footprint requirements compared to plasmid DNA production process. Additionally, we show the broad versatility of our synthetic DNA for cell and gene therapy applications by demonstrating its use as input for mRNA, recombinant adeno-associated virus, and lentivirus vector production, as well as for use in transgene expression in vitro and in vivo. Our synthetic DNA is well-positioned to replace plasmid DNA as key starting material for cell and gene therapy modalities.

Plain Language Summary
We have developed a novel synthetic DNA that could replace plasmid DNA as a method of choice for Cell and Gene Therapy applications. We have a one-pot, enzymatic, and cell-free production process that is easily scalable, quick and has a small footprint. The resulting DNA has high purity and fidelity. In this work we show its use in adeno-associated virus, lentivirus vector, and mRNA production as well as for the expression of transgenes in vivo and in vitro.

Alexander Pekarsky, Luca Distefano, Marco Guarrera, Ivana Pastierikova, David Wilson, Martin Cusack, Anna Krutyholowa, Roxanne Lourman, Fabian Trick, Gustavo Lou, Andreia M. Silva, Ileana Guerrini, Nicolas Meier, Jorge Omar Yanez Cuna, Joel de Beer

Anjarium Biosciences AG, Schlieren, Switzerland"

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