Human CRISPR Trial

The United States Food and Drug Administration (FDA) recently granted permission for the first in-body clinical trial of a CRISPR-Cas9-based gene-editing therapy. The study will include patients with Leber’s congenital amaurosis type 10, a hereditary form of retinal dystrophy and the most common cause of inherited childhood blindness.

The agent is designed to correct a mutation that disrupts the structure of light-sensing photoreceptors in the retina through genome editing. It will be delivered by subretinal injection into one eye. The patient population will include between 10 and 20 adult and pediatric patients. The term of the study is three years, although some data will likely become available sooner.

In contrast to the ethically and scientifically dubious work recently reported from China claiming use of CRISPR methods to “customize” babies (e.g., through preselection of eye color, athletic or intellectual prowess, etc.), this and other proposed studies remain focused on treating and preventing human disease.