The field of genome editing is quickly gaining momentum, as demonstrated by the recent funding rounds of fledgling CRISPR-Cas9 companies: in November last year, Intellia Therapeutics raised a $15 million Series A, which was followed by a collaboration with Novartis in January to develop new cell therapies. In April, Caribou Biosciences raised $11 million in Series A funding to further develop their CRISPR technologies. On the same month, CRISPR Therapeutics secured $64 million in two rounds led by Celgene and the venture arm of GlaxoSmithKline to translate CRISPR-Cas9 technology into new therapies.

But recently, Editas Medicine received the largest financial investment yet in a CRISPR-Cas9 company: a $120 million Series B funding. The round was led by Boris Nikolic, Bill Gates’ former scientific advisor and managing director of the investment company Bng0 (Bill Gates himself is among the bng0 backers). Editas also announced that Boris joined the company’s board of directors.Editas Medicine, which was recently named a 2015 Technology Pioneer by the World Economic Forum, was founded in November 2013 in Cambridge by some of the most prominent inventors of the gene-editing system: Feng Zhang, a researcher at the Broad Institute, Jennifer Doudna and David Liu from Howard Hughes Medical Institute, George Church and J. Keith Joung from Harvard Medical School. With an initial investment of $43 million from Flagship Ventures, Polaris Partners and Third Rock Ventures, Editas will use the new $120 million funding to translate the CRISPR/Cas9 technology into genomic medicines to treat diseases including cancers, sickle-cell anemia, retinal disease, and Duchenne muscular dystrophy.

Harnessing the Human Immune System to Treat Diseases

Because the CRISPR-Cas9 system can be engineered to cut any DNA sequence at a precisely chosen location, Editas Medicine is working with Juno Therapeutics (which got $310 million last year before going public) to create therapies for cancer. This promising type of immunotherapy involves engineering the patients’ own immune cells (T-cells) to recognize and attack their tumors. This is how it works: they separate the T-cells from a patient’s blood sample, and engineer the cells (through CRISPR-Cas9 for example) to produce specific receptors on their surface (chimeric antigen receptors or CARs). The CARs allow the T-cells to recognize a certain protein (antigen) on tumor cells. The engineered CAR-T cells are then cultivated in the lab, and then infused into the patient. In the body, they multiply, recognize and kill cancer cells that harbor the antigen on their surfaces.

In addition to cancer therapies, Editas researchers have also taken retinal cells out of patients with LCA10 (a retinal disease) and edited them with CRISPR to make them express specific proteins in the lab. The company is testing its technology in patient cells, but clinical trials with CRISPR-edited cells are not yet scheduled to begin.