Revolutionary gene therapy eyedrops restore boy’s sight, open door for similar treatments 

Newly developed gene therapy eyedrops restored a boy’s sight, and a similar treatment may help millions, according to the Associated Press.

The treatment was administered by Dr. Alfonso Sabater to Antonio Vento Carvajal, who was legally blind for much of his 14 years due to a rare genetic condition called dystrophic epidermolysis bullosa. The condition causes blisters to form over the body and in the eyes.

Carvajal and his family came to the U.S. from Cuba in 2012 to seek treatment for his condition. A clinical trial testing the world’s first topical gene therapy improved his skin, prompting Sabater to contact drugmaker Krystal Biotech to see if it could be reformulated for the boy’s eyes.

After two years of development, including testing the drug in mice, the FDA granted “compassionate use” approval to the team, which also received permission from university and hospital review boards.

The success of the treatment has opened the door for similar therapies that have the potential to treat millions of people with other eye diseases, including common ones.