Editas Medicine, a biotechnology startup, predicts that we will begin human testing with CRISPR within two years. Editas CEO Katrine Bosley spoke at the EmTech conference in Cambridge, saying that the company is looking to begin a clinical human trial to treat a rare form of blindness using the groundbreaking gene-editing technology in 2017. The rare eye disease in question is called Leber congenital amaurosis, and it affects the retina’s light-receiving cells. The disease is suitable for the treatment because the exact gene error is known and the eye is easily accessible for genetic treatments. “It feels fast, but we are going at the pace science allows,” says Bosley.
Editas will conduct the procedure as gene therapy, and it will involve an injection into the retina. The injection will deliver a soup of viruses that are loaded with DNA instructions to manufacture CRISPR components, along with a protein that precisely cuts the gene. The therapy will delete 1,000 DNA letters from a gene in the patient’s photoreceptor cells. CRISPR was invented three years ago and has already been used in China to modify human embryos.