Naples, gene therapy restores sight to ten children

in 2006 The gene therapy (voretigene neparvovec of Novartis) was the subject of a phase 1 trial conducted in collaboration between Vanvitelli University, the Telethon Foundation and Children’s Hospital of Philadelphia.

Naples Center of Excellence – The success of the treatments makes Naples a national reference point for the treatment of rare diseases of the retina, and the University emphasizes, on the effectiveness of the collaboration between the public and private sectors, access to the most innovative treatments for patients who arrive in recent times who did not have the possibility of treatment.

The ten patients were treated – explains Francesca Simonelli, Professor of Ophthalmology and Director of the Ophthalmology Clinic at the University of Campania Luigi Vanvitelli – “Today they can write, read and move independently. The results we obtained for expanding the visual field and increasing vision in near, far and low light conditions have profound scientific and clinical value. They also testify that in degenerative pathology, early treatment is a winning method.”

In 2019, commissioned by Aifa, “We treated the first two children in ItalyToday, after nearly two years of treatment, we can confirm the absolute stability of results and a good safety profile. These are the data that make us confident that what has been achieved in terms of visual ability
The expert added.

‘unusual result’, says the president of the University of Vanvitelli, Gianfranco Nicoletti. Ten visually impaired children ‘restored their sight, I must thank the Campania region that has invested in our gene therapy and ophthalmology for the excellent work done which puts us between
The first centers in Europe for the treatment of children.

Treating a patient with hereditary retinal atrophy With gene therapy it requires a very complex preparatory course and a management process that requires a long preparation. The Eye Clinic of the University of Vanvitelli in Naples is the first center in Italy to be accredited to administer gene therapy. “We now have a one-time treatment that allows us to offer real hope to patients and their families,” notes Professor Simonelli.