Stephen Hughes

Background Stephen Hughes New Platform Development Group Introduction Platform using nanoDNA for hospital-based self-transduction of ultrapersonal episomal or integrated gene therapies From the beginning of the gene and cell therapy revolution in 2017, the FDA has been pushing for a safe hospital-based ultrapersonal route. The route must be useful for both ultrapersonal gene editing and addition integrating gene therapy and also for ultrapersonal euchromatin chromosome episomal replicative cell therapy. This paradigm should involve self-assembly without use of plasmids or E. coli for production, and it should not involve expensive disposable production processes for plasmid production nor expensive viral production. The process should be low cost and produce large numbers of doses and should be able to be carried out in the hospital’s gene and cell therapy areas. Goal Integrated Synthetic Chromosome Technologies Inc. and GenScript nanoDNA gene therapy production collaboration team with Rutgers Cancer Institute of NJ, Penn Health Princeton, Universal Machine and Engineering, and Calvary Robotics will produce a microfluidic device that self assembles ultrapersonal gene therapie chromosomes for episomal or euchromatin integrated routes. The microfluidic platforms will use Integrated Synthetic Chromosome Technologies Inc. equipment and GenScript reagent packs at the hospital for undefeated error-free nanoDNA particles for ultrapersonal therapeutic use. This unit will self-assemble the nanoDNA particle for a gene therapy that can be used with or without the patients’ cells. That is the ultrapersonal stem cell or patient cell for therapy can be removed and self- transduced with this nanoDNA, or the nanoDNA can be directly self-transduced at the site in the patient’s tissue. No plasmid, or plastic disposables, or oligos will be used in the unit. Gene therapy platform for manufacturing nanoDNA therapeutic product for targeted cell- specific gene expression or editing