piggyBacTM Transposon System

Footprint-FreeTM Gene Editing

Description Non-viral vector for adding or removing genes Combines site-specific nucleases with piggyBacTM for seamless precision engineering
Potential Advantages
  • Safer vs. viral approaches
  • More cells transduced and higher expression levels
  • 10x less costly GMP production
  • No known cargo limit (>300kb)
  • Reversible – no footprint
  • The only therapeutic method capable of seamless excision of resistance or reporter genes
  • Increased efficiencies by utilizing selection enrichment for gene edits
  • Can edit as little as a single nucleotide with no unwanted mutations – the cleanest gene editing technology available
Therapeutic Applications
  • Autologous and allogeneic CAR-T therapies for hematological cancers and solid tumors
  • in vivo and ex vivo gene therapies for a variety of monogenic diseases (e.g., orphan liver, hematological, CV, and CNS diseases among others)
  • Infectious diseases (e.g., HIV, HBV, etc.)