Take-home message: The very first medical trial of lentiviral gene therapy in ophthalmology met its primary objective, showing safety and tolerability of subretinal administration of the lentiviral vector indicating anti-angiogenic proteins in patients with advanced exudative age-related macular degeneration.

“The phase I study can also be the very first time therapeutic gene expression continues to be directly measured within an ophthalmology medical trial,Inches Dr. Lauer stated. “Notably, persistence of gene expression has been maintained during lengthy-term follow-up that to date stretches beyond four years.Inches

Additionally, assays of aqueous humor acquired through anterior chamber taps demonstrated the therapy led to dose-dependent, significant, and chronic increases in expression from the endostatin and angiostatin genes.

 

 

Such findings come from the outcomes of the dose-increasing phase I study looking into subretinal administration of the lentiviral vector indicating endostatin and angiostatin (RetinoStat, Oxford BioMedica).

Portland, OR—A novel gene-based therapy shows promise to be safe and well tolerated—with indications of clinical benefit in patients with advanced neovascular age-related macular degeneration (AMD), stated Andreas K. Lauer, MD.

“The phase I study of RetinoStat may be the first medical trial in ophthalmology looking into lentiviral gene therapy, which construct provides a distinct advantage to treat wet AMD as her possibility of a significantly longer time period of effect than current treatments,” stated Dr. Lauer, Kenneth C. Swan Professor of Ophthalmology and chief, Vitreoretinal Division, Casey Eye Institute, Or Health insurance and Science College, Portland, OR.

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