Gene Editing through CRISPR Considered in Human Models

MIT Technology Review reports that gene editing through use of the CRISPR/Cas9 complex will be evaluated by the National Institutes of Health (NIH) early next week, and it will be the first time it will be considered in human models. Although earlier CAR-T treatments have relied on traditional gene editing techniques, gene editing using CRISPR will reportedly be more precise and less costly. The use of the gene editing system will be discussed by the NIH’s Recombinant DNA Advisory Committee Meeting.

Although it appeared that Editas Medicine’s CRISPR-based eye treatment may be the first in human trials (slated to begin in 2017), a team led by CAR-T pioneer Carl June may be closer to the finish line. June plans to edit two genes in a T-cell immunotherapy protocol to better target myeloma, melanoma, and sarcoma tumor cells. One of the genes June will propose to remove codes for immune checkpoint inhibitor PD-1, which can bind with PD-L1 on tumor cells and helps the tumor cells evade destruction by the immune system. Many monoclonal antibodies on the market or currently in development are engineered to bind to PD-L1 on tumors or to PD-1 on T cells so that T cells may work freely to recognize and fight cancer cells.

June will propose his use of CRISPR to NIH in a presentation entitled, “Updates in manufacturing of CTL019” during the NIH meeting. CTL019 is the Novartis-backed therapy being developed by the Perelman School of Medicine at the University of Pennsylvania.