The first trial using CRISPR within the human body is to begin this fall to treat a form of blindness, according to the Associated Press.
Editas Medicine and Allergan plan to enroll 18 people with Leber congenital amaurosis, a genetic form of blindness that affects the retina, in their study. According to the AP, Leber congenital amaurosis is the most common cause of inherited childhood blindness.
The condition can arise from more than a dozen genetic mutations, and Editas and Allergan are focusing on individuals whose disease is caused by homozygous or compound heterozygous mutation affecting intron 26 of the CEP290 gene, according to ClinicalTrials.gov. They are targeting that CEP290 gene alteration in patients' retinas with gene editing machinery delivered via injection, and the AP adds that the companies hope it will be a one-time treatment of the disease.
Recruitment to the study is underway at Massachusetts Eye and Ear Infirmary and is to expand to three other sites, according to ClinicalTrials.gov.
"Beginning patient enrollment in the AGN-151587 clinical trial with our partners at Editas is an important step toward our goal of developing a game-changing, transformative, CRISPR-based medicine for people with" this form of Leber congenital amaurosis, David Nicholson, the chief research and development officer at Allergan, says in a statement.