Overview

RNA molecules can be used to express proteins in cells, both ex vivo and in vivo. Conventional approaches using linear RNA molecules can result in exonuclease degradation, which can lead to low protein expression.

Our scientists developed a technology for synthesizing circular RNA molecules that does not require splints or ribozymes9. This technology can enable durable protein expression in cells and can be used, for example, to generate low-dose RNA vaccines and for in vivo epigenetic reprogramming.

Splint and Ribozyme-Independent Circular RNA Synthesis is protected by a pending U.S. patent (with additional patents pending in other countries).

9Svihla, A., et al. Mol Ther, Vol 29, No 4S1, 2021.

Example Applications

  • Ultra-high efficiency editing of T cells, fibroblasts, keratinocytes, and pluripotent stem cells
  • Ultra-high specificity gene editing
  • Virus-free and DNA-free gene editing
  • Gene repair using a DNA-repair template
  • Donor sequence insertion into a target genomic locus (e.g., TRAC, AAVS1 safe harbor, etc.)
  • Gene-editing therapies (ex vivo and in vivo)
  • Autologous and allogeneic engineered cell therapies (e.g., CAR-T, CAR-NK, stem cell-derived therapies, etc.)
  • Low-dose RNA vaccines