Gene-based treatment provides sustained AMD therapy
The first clinical trial of lentiviral gene therapy in ophthalmology met its primary objective, demonstrating safety and tolerability of subretinal administration of a lentiviral vector expressing anti-angiogenic proteins in patients with advanced exudative age-related macular degeneration.
Reviewed by Andreas K. Lauer, MD
Portland, OR-A novel gene-based therapy shows promise as being safe and well tolerated-with signs of clinical benefit in patients with advanced neovascular age-related macular degeneration (AMD), said Andreas K. Lauer, MD.
Such findings are from the results of a dose-escalating phase I study investigating subretinal administration of a lentiviral vector expressing endostatin and angiostatin (RetinoStat, Oxford BioMedica).
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In addition, assays of aqueous humor obtained through anterior chamber taps showed the treatment resulted in dose-dependent, significant, and persistent increases in expression of the endostatin and angiostatin genes.
“The phase I study of RetinoStat is the first clinical trial in ophthalmology investigating lentiviral gene therapy, and this construct offers a distinct advantage for the treatment of wet AMD as it has the potential for a much longer duration of effect than current therapies,” said Dr. Lauer, Kenneth C. Swan Professor of Ophthalmology and chief, Vitreoretinal Division, Casey Eye Institute, Oregon Health and Science University, Portland, OR.
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“The phase I study is also the first time therapeutic gene expression has been directly measured in an ophthalmology clinical trial,” Dr. Lauer said. “Notably, persistence of gene expression is being maintained during long-term follow-up that so far extends beyond 4 years.”