December 3, 2013 – A Cambridge, Massachusetts startup, Editas Medicine, is on a mission to develop life-changing treatments of diseases through a method called genome editing. This involves modification of the human genome to treat diseases at a genetic level.
Founded by five leading researchers in the field of genomics, the company has developed and patented gene editing technology. It uses CRISPR (clustered, regularly interspaced short palindromic repeats)/Cas9 (CRISPR-associated protein 9) and TALENs (transcription activator-like effector nucleases) to edit defective genes. The Cas9 protein binds to RNA molecules which then guide it to the gene allowing for repair treatment. And rather than being limited to a specific disease the therapy can be used to treat a wide range of defective genes.
Editas just received $43 million from venture capital firms in the U.S. to help it deliver on the promise that is this technology. Although the company doesn’t talk about specific diseases it hopes to tackle, it is pretty clear that among those known to be triggered by dysfunctional genes are cystic fibrosis, sickle-cell anemia, Huntington’s, Marfan syndrome, hemochromatosis, Alzheimer’s, cancer, obesity, heart disease, Down syndrome, and so many others. The ability to do corrective molecular modification of such a wide range of diseases will truly be an enormous advancement for gene therapy and spark a revolution in medical science.