Promising Study Supports Gene Therapy For Wet Macular Degeneration

Professor P. Elizabeth Rakoczy Centre for Ophthalmology and Visual Sciences The University of Western Australia Head of Department - Molecular Ophthalmology Lions Eye Institute Australia 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 Biofactoryâ„¢ 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 Biofactoryâ„¢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.

 

Citation:

Gene therapy with recombinant adeno-associated vectors for neovascular age-related macular degeneration: 1 year follow-up of a phase 1 randomised clinical trial

Rakoczy, Elizabeth P et al.

The Lancet Published Online: 29 September 2015

DOI: http://dx.doi.org/10.1016/S0140-6736(15)00345-1

 

Professor P. Elizabeth Rakoczy (2015). Promising Study Supports Gene Therapy For Wet Macular Degeneration