- In January 2025, ViGeneron GmbH, a clinical-stage gene therapy company based in Munich, Germany, announced that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation for its gene therapy candidate VG901, targeting CNGA1-associated retinitis pigmentosa
- In February 2025, At Mater Hospital in Dublin, Ireland, a 31-year-old man blind for 13 years regained his sight after receiving Luxturna gene therapy. This groundbreaking treatment, administered by Professor David Keegan, marks the first such procedure in Ireland and demonstrates significant benefits even in adults with inherited retinal dystrophies
- In February 2025, Doctors at Great Ormond Street Hospital in London successfully restored vision in children with Leber congenital amaurosis (LCA) using gene therapy. The procedure involved injecting healthy copies of the AIPL1 gene into the retina, enabling the children to see shapes, recognize their parents, and, in some cases, read and write
- In October 2024, At the American Academy of Ophthalmology (AAO) 2024 meeting, Nanoscope Therapeutics presented promising results for their gene therapy MCO-010, targeting retinitis pigmentosa. The therapy showed significant visual acuity improvements in up to 50% of patients without the need for external devices, offering hope for those with advanced vision loss
- In September 2023, Genentech announced successful early-stage clinical trials for a new gene therapy targeting specific retinal dystrophies. This development has generated optimism for patient outcomes and heightened investor interest in the field of retinal therapies
