CRISPR Therapeutics to launch first gene-editing clinical trial
Swiss genetic engineering firm CRISPR Therapeutics has sought permission from European regulators to begin its first-ever clinical trials early next year on CTX001, a gene therapy treatment for beta thalassemia. The company is also set to apply to the US Food and Drug Administration for approval to use it against sickle cell disease. CTX001 stimulates production of foetal haemoglobin, which the body usually cuts off at the age of around six months, and has resulted in an editing efficiency of 80%, with no reported off-target effects.

ISTOCK, BUBAONELast Thursday (December 7), CRISPR Therapeutics submitted an application to European regulatory authorities seeking permission to begin clinical trials for CTX001, an investigational CRISPR treatment for patients with sickle cell disease and β thalassemia.

CRISPR Therapeutics, the company cofounded by Emmanuel Charpentier—one of the developers of CRISPR gene editing technology—plans to start a Europe-based Phase 1/2 trial for patients with β thalassemia in 2018. “I think it’s a momentous occasion for us, but also for the field in general,” Samarth Kulkarni, CEO of the company, tells Wired. “Just three years ago we were talking about CRISPR-based treatments as sci-fi fantasy, but here we are.”

The firm also plans to apply for US Food and Drug Administration approval to use the treatment for sickle cell disease early next year.

Patients with sickle cell disease and β thalassemia possess mutations in a gene that produces a subunit of hemoglobin,...