Editas Medicine, Inc. (NASDAQ:EDIT), a leading genome editing company, today announced results from a pre-clinical study evaluating multi-gene knockout and transgene knock-in using its proprietary engineered CRISPR-Cas12a (EngCas12a) in induced pluripotent stem cells (iPSCs) for the development of engineered cell immunotherapy medicines. The results of this study further reinforce Editas Medicine’s belief in the transformative potential of iPSC-derived natural killer (iNK) cells as off-the-shelf engineered cell medicines for the treatment of solid tumor cancers. The Company reported these data today in an oral presentation at the 23rd Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) being held virtually.
Induced pluripotent stem cells (iPSCs) offer a renewable source of highly characterized cells that can be differentiated into an array of immune effector cells, including, but not limited to, iPSC-derived natural killer cells (iNKs). Edited iPSC clones can then be screened and selected to contain only the desired edits, ensuring a pure and edited final population of iNKs. Allogenic NK cells are an effective cancer cell therapy without evidence of graft versus host disease.
In this study, CRISPR-Cas12a was used to make highly edited iPSC clones. The iPSCs were then differentiated into functional iNK cells. The iNKs derived from the edited iPSC clones had enhanced tumor killing activity relative to iNKs from unedited iPSCs, demonstrating the utility of an edited iPSC platform. This data supports the continued development of off-the-shelf engineered cell medicines for people with solid tumor cancers.
“We have made significant progress in our engineered cell medicine program for the treatment of oncologic diseases, and this data further supports our belief that using CRISPR technology to make differentiated medicines can make a meaningful impact in the treatment of cancer,” said Charles Albright, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. “We are focused on solid tumors given the high unmet need and will pursue multiple cell types to create a portfolio of innovative medicines. We believe using innate immune cells, specifically NK cells, has the potential to yield transformational medicines and potentially overcome the challenges of treating solid tumors.”
Editas Medicine is also advancing programs using CRISPR editing for the potential treatment of both solid and liquid tumors. The Company initiated IND-enabling studies for EDIT-201, a healthy donor NK (HDNK) engineered cell medicine for solid tumors and is advancing its engineered gamma-delta T cell program. In addition, the Company is advancing oncology therapies with engineered alpha-beta T cells in collaboration with Bristol Myers Squibb.