13 February, 2025
5 years of Luxturna – The first gene therapy for an IRD
ERN-EYE hosted a webinar in late January 2025 to mark five years since the first gene therapy for an inherited retinal disease (IRD), Luxturna.
ERN-EYE is a European Reference Network dedicated to rare eye diseases. The webinar explored what clinicians have learned over the past 5 years, insights from clinical trials, the genetic diagnosis process and if there anything to be learned from molecular genetics, and a patient perspective.
Webinar agenda
- Introduction of the speakers, Pr Katarina Stingl, University Tübingen, Germany.
- What have clinicians learned in 5 years?, Pr Katarina Stingl, University Tübingen, Germany.
- Insights from clinical trials, Pr Bart P. Leroy, Ghent University Hospital, Belgium.
- Can anything be improved in molecular genetics?, Pr Elfride de Baere, Center for Medical Genetics, Ghent University Hospital & Dept of Biomolecular Medicine, Ghent University, Belgium
- Patient perspectives, Mr João Paulo Lacerda, father of a child with Retinitis Pigmentosa.
Webinar Speakers
- Professor Katarina Stingl, MD is a professor for Ophthalmology and head of the Center for Rare Eye Diseases and the Clinical Unit for Inherited Retinal Degenerations of the University Tübingen.
- Professor Leroy is professor of Ophthalmology, Ophthalmic Genetics and Visual Electrophysiology at Ghent University and the Head of the Ophthalmology department at Ghent University Hospital, Ghent, Belgium.
- Professor Elfride De Baere, MD, PhD, is Professor at Ghent University and Head of Lab at the Center for Medical Genetics Ghent (CMGG) of the Ghent University Hospital. Her lab offers genetic testing for rare eye diseases such as inherited retinal diseases (IRD), cataract, developmental eye diseases, corneal dystrophies, glaucoma. She chairs the TWG6 ‘Genetic Diagnostics’ of ERN-EYE. She is member of ocular ClinGen working groups and co-chairs the ABCA4 Variant Curation Expert Panel.
- Mr João Paulo Lacerda is an experienced Software Engineer. In 2020, his son Enrique was diagnosed with Retinitis Pigmentosa. Due to a job offer, his family moved from Brazil to Germany at the end of 2021. In 2023, his son received genetic treatment with Luxturna and has been showing excellent progress since then.
