12 Oct Promising Study Supports Gene Therapy For Wet Macular Degeneration
MedicalResearch.com Interview with:
Professor P. Elizabeth Rakoczy
Centre for Ophthalmology and Visual Sciences
The University of Western Australia
Head of Department – Molecular Ophthalmology
Lions Eye Institute Australia
Medical Research: What is the background for this study?
Prof. Rakoczy: Wet age related macular (wet-AMD) is the major cause of blindness in the developed world. It is treated with frequent anti-VEGF injections into the eye. Our preclinical studies demonstrated that following the subretinal injection of a recombinant adeno-associated vector (rAAV) carrying a natural inhibitor of neovascularization (sFlt-1), leaky new, abnormal vessels can be controlled and retinal anatomy improved. The rAAV.sFlt-1 based Ocular Biofactory™ platform has potentially significant advantages over existing technologies as it is designed to provide sustained production of a naturally occurring antiangiogenic agent, sFlt-1, in situ in the eye. In this trial we investigated the safety of rAAV.sFlt-1 in patients diagnosed with wet-AMD.
Medical Research: What are the main findings?
Prof. Rakoczy: What are the main findings?
- sFlt-1 was safe and well tolerated.
- Subretinal injection of rAAV.sFlt-1 is a viable approach for drug delivery.
- No gene therapy-related serious adverse events were noted.
Medical Research: What should clinicians and patients take away from your report?
Prof. Rakoczy: Clinicians and Patients should take away that the study drug was safe and well tolerated. Further, note the hopeful concept that the Ocular Biofactory™platform, based on secretion gene therapy, may provide a viable long term treatment for wet-AMD and other chronic eye disease.
Medical Research: What recommendations do you have for future research as a result of this study?
Prof. Rakoczy: The results of this safety study support the concept that ocular gene therapy might be a viable long-term treatment option for wet age-related macular degeneration. Growing the body of evidence supporting the viability of intraocular gene therapy to treat retinal disease seems indicated.
Professor P. Elizabeth Rakoczy (2015). Promising Study Supports Gene Therapy For Wet Macular Degeneration