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CRISPR gene editing to take on inherited blindness in U.S. study

A new study in the United States will use CRISPR gene-editing technology to treat Leber congenital amaurosis (LCA), a retinal condition that is a major cause of inherited blindness. The study, which will take place later in 2019, is currently open for would-be patients to enroll.

LCA affects roughly one in 80,000 people, making it the most common form of inherited sight loss among children. The condition causes the light-sensing photoreceptor cells at the back of the eye to stop functioning correctly. Symptoms can involve poor peripheral vision, night blindness, and faulty pupil reactions. Over time, these conditions can worsen; leaving patients able to only detect the faint outlines of shapes or even totally blind.

The test will evaluate a gene therapy named AGN-151587 as a treatment for LCA. The study will assess safety, tolerability, and efficacy of this treatment in 18 patients. Patients in the study will receive a single dose of AGN-151587 administered using a subretinal injection in the eye.

“We are very proud of our continued commitment to developing innovative treatments for unmet needs in eye care,” David Nicholson, Chief R&D Officer at Allergan, said in a statement. “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 LCA10.”

This is a significant advance in the usage of CRISPR gene-editing technology. Since bursting onto the scene a decade ago, CRISPR has opened up a slew of new possibilities — from the creation of malaria-resistant mosquitoes to, as this study could help confirm, life-changing medical advances. CRISPR got a bad rap last year when it was used as part of a controversial Chinese experiment in which gene-editing was used on embryos or infants. Unlike that widely condemned experiment, this work involves fully grown consenting adults.

“Now that enrollment is underway, we are one step closer to delivering a transformative medicine to LCA10 patients,” Charles Albright, Chief Scientific Officer of Editas Medicine, one of the other companies involved with the study, said in a press statement. “The team at Editas looks forward to continuing to collaborate with our partners at Allergan, patient advocacy organizations, and the inherited retinal diseases community as we develop this and other durable experimental medicines for patients with devastating ocular diseases.”

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