Retinal Dystrophy

The loss of UBAP1L gene function appears to be associated with nonsyndromic retinal dystrophy, according to research published in JAMA Ophthalmology.

The FDA cleared an investigational new drug application from HuidaGene Therapeutics for a clinical trial of HG004 for the treatment of RPE65 mutation-associated inherited retinal dystrophies, according to a press release.

Avista Therapeutics and Roche will partner to develop adeno-associated virus gene therapy vectors for ocular diseases, according to a press release.

Optogenetic stimulation of the retina with a treatment combining injection of photoactivatable channelrhodopsin protein and light-stimulating goggles showed the ability to partially restore vision in late-stage retinitis pigmentosa.

FDA approval of the first gene therapy in December 2017 marked the beginning of a new chapter in medical practice.

NHS England will fund Luxturna gene therapy treatment for inherited retinal dystrophies starting in January 2020, according to a press release.