EpyQ® AAV Production System

In AAV production, traditional triple transfection (3 plasmids) is expensive and suffers from inherent limitations that can directly impact productivity and product quality. The EpyQ® AAV Production System is a two-plasmid transfection platform using one custom plasmid and one off the shelf plasmid to lower cost and complexity. In application, users can expect more rapid development of more robust AAV manufacturing processes with improved yields and reduced product-related impurities.

EpyQ® AAV Production System licensing

Clients manufacturing at Ascend in Munich, Germany or Alachua, Florida do not require a license
Clients manufacturing outside Ascend require licensing

GOI/capsid Plasmid

Where the client’s valuable IPR lies

  • Annual License Fee
    • $25K for innovators & low use
    • $100,000 for Biotech-Pharma
  • Can be used for all GOI’s, capsids, indications (no additional licences)
  • No milestones or royalties
  • Transferable under same terms

AdHelper/rep plasmid used for manufacturing with EpyQ® & GMP plasmid supply

  • EpyQ® licensees must use this plasmid
  • Provided off the shelf by Ascend as needed under supply agreements
  • $300,000 for 250mg for 200L production run = Cost of Goods =$18,250 & CM of $281,000

The EpyQ® AAV Production System uses two plasmids vs. three

  • Proven in multiple clinical AAV programs to enhance safety via reduced plasmid sizes
  • Only one bespoke plasmid lowers COGs
  • More full capsids & less DNA contaminants with higher productivity (vg/mL upstream)
  • All customer IP on one licensed plasmid
  • Rapid supply of GMP grade AdHelper/rep plasmid
  • Lower chances of replication competent AAV formation
  • Fewer & smaller plasmids may be used for any serotype, clinical program or scale
  • ITR’s (responsible for gene delivery & persistence) are 100% stable with our design
  • Lowers costs at larger scale
  • Simple migration to 2-plasmid system

Traditional 3 plasmid systems have higher DNA costs (by almost double!)

  • Clients must source 3 plasmids (1 or 2 be$poke)
  • Innovators must purchase, modify & clone 2 plasmids independent of the product or serotype developed
  • Lower efficiency transfection & rAAV yields
  • Fewer fulls & more DNA contaminants
  • Higher input costs (MCB generation, GMP manufacturing)
  • 3 plasmid systems suffer from functional co-transfection efficiency & AAV impacting yield & quality
  • Innovators pay higher costs for multiple plasmids
  • Many systems have lower % ITR sequence (less intact) with partial deletions/modifications & the potential to destabilize during amplification
  • Significant increase in the risk of rcAAV (replication) using 3 plasmid systems