AUG 19, 2015
Genome editing company Editas Medicine has raised $120 million to develop new treatments for cancer, sickle-cell anemia and Leber congenital amaurosis.
“With this financing, we have a strong foundation from which we can broadly develop our genome editing platform and advance multiple new therapies toward clinical trials,” said Katrine Bosley, the company’s CEO.
Editas uses the gene-editing technology, CRISPR (Clustered, Regularly Interspaced Short Palindromic Repeats)/Cas9 (CRISPR-associated protein 9).
Recent advances in CRISPR/Cas9 genome editing have made it possible to modify almost any gene in the human body by turning on or off or editing disease-causing genes. This technology has the potential to address diseases untreatable with traditional gene therapy, gene knock-down or other genome modification techniques.
CRISPR is particularly promising in making precise changes in cells. T cells taken from a patient could be edited and then infused back into a patient’s body. Or cells could be fixed and put back in the blood of a patient with sickle-cell anemia.
Such treatments, however, are in their early days. Editas is not yet running clinical trials involving CRISPR-edited cells. “We don’t want to get out there so fast with patients when we’re not ready, we don’t have the programs yet,” said Bosley.
In addition to using T cells in treating blood cancers, a second major project involves treating Leber congenital amaurosis. Editas scientists have taken retinal cells out of patients with LCA10 and used CRISPR to give those cells proteins that function—in the lab, for now.