Gene-Edited Allogeneic Cell Therapies

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Allogeneic cell therapies can enable “off-the-shelf” treatment options. Conventional allogeneic cell therapy approaches can result in rejection, low potency, and in the case of allogeneic immune cell therapy, graft-versus-host disease.

Our scientists developed a technology that uses gene editing to address limitations of conventional approaches to allogeneic cell therapy5. This technology can be used to generate allogeneic cell therapies with high potency and enhanced targeting.

The Gene-Edited Allogeneic Cell Therapies technology is protected by a pending U.S. patent (with additional patents pending in other countries).

5Kopacz, M., 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)
  • Allogeneic engineered cell therapies (e.g., CAR-T, CAR-NK, stem cell-derived therapies, etc.)