Dr. Bala Ambati’s career has focused on understanding the basis of ocular vascular demarcations and leveraging those insights to develop novel protein & gene therapeutics which promise to transform the care of blinding diseases such as AMD, diabetic retinopathy, and corneal injury. His Nature paper served as the foundation for the following key subsequent developments:
a. Dr. Ambati’s development of intraceptors, subunits of VEGFR-1 which sequester VEGF intracellularly, complementing the existing arsenal of anti-VEGF agents which all work outside cells. His group has developed exciting ways of delivering these agents via targeted nanoparticles and AAV vectors.
b. The culmination of 3 clinical trials by different pharmaceutical companies of AAV therapy to express sVEGFR-1 for the chronic therapy of neovascular age-related macular degeneration, also known as wet AMD.
In addition to Dr. Ambati’s trenchant contributions to understanding and exploiting the potential of sVEGFR-1, his team made several other innovative advances. Dr. Ambati’s laboratory discovered soluble VEGF receptor-3 (completing the suite of soluble VEGF receptors) and its key role in ensuring corneal alymphaticity. Dr. Ambati and his team have developed implants for long-acting inflammatory control after cataract surgery (enabling drop-free cataract surgery), a morpholino that shifts alternative splicing of VEGFR-1 towards the soluble isoform, and most recently demonstrated that diabetic neurovascular degeneration can be stopped and reversed using a combination of AAV.COMP.Ang1 and vascular regenerative endothelial colony forming cells. Each of these will be invaluable to the care of ophthalmic patients.