LCA patients experience dramatic improvements in vision following gene therapy

US researchers have described the results of gene therapy in a small group of Leber congenital amaurosis (LCA) patients.

Writing in The Lancet, researchers described how a group of 15 LCA patients received unilateral subretinal injections of the gene therapy, ATSN-101.

The study tested different dosage levels of the gene therapy. For the first phase of the study, three groups of participants received different dose levels: low, mid and high.

A second phase of the study involved only administering the high dosage levels to three adults with LCA and three children with LCA.

Among the patients who received high dose gene therapy, there was a 100-fold improvement in vision – with two patients experiencing a 10,000-fold improvement in vision.

Lead author, Dr Artur Cideciyan, of Perelman School of Medicine at the University of Pennsylvania, shared: “That 10,000-fold improvement is the same as a patient being able to see their surroundings on a moonlit night outdoors as opposed to requiring bright indoor lighting before treatment.”

“One patient reported for the first time being able to navigate at midnight outdoors only with the light of a bonfire,” he added.

There were no drug-related serious adverse events reported during the 12-month trial.

The authors highlighted that the favourable safety profile and improvements in retinal sensitivity support a randomised, controlled trial to further investigate the treatment.

“ATSN-101 has the potential to be a first-in-class treatment for LCA1, a blinding condition with no approved treatment,” they emphasised.