Shanelle Mendes

Age-related macular degeneration (AMD) is one of the major causes of blindness among those over 50 in the United States, and affects over 170 million people worldwide. Over 1 million people in the United States suffer from neovascular AMD (NVAMD), which causes blindness at a higher rate than other forms of AMD. Existing therapies for NVAMD include antibody therapeutics like Avastin and Lucentis, which aim to eliminate the overgrowth of blood vessels in the retina through inhibition of vascular endothelial growth factor A (VEGFA). However, these therapies involve monthly intravitreal injections, which can be both time-consuming and uncomfortable for patients. An alternative approach to direct administration of antibody therapeutics is to deliver nucleic acids coding for VEGFA inhibitors. This gene delivery approach would allow for a more sustained production of VEGFA inhibitors, thus avoiding repeated treatments. In 2017, the FDA approved Luxturna, a retinal gene delivery drug. However, Luxturna costs $425,000 per eye. Furthermore, recombinant adeno-associated virus (rAAV) vectors like Luxturna have several drawbacks including a limited gene cargo capacity and risk of pre-existing immunity in patients. Non-viral, polymer-based vectors for gene delivery have much larger gene cargo capacities and have a lower chance of inducing an immune response. Here, we have designed a novel, non-toxic, and highly efficient, non-viral gene delivery vehicle for delivery to retinal cells.

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