Audrey Lapinaite Awarded RPB Career Advancement Award for Innovative AMD Genome Editing Project

Dr. Audrey Lapinaite has been awarded the Research to Prevent Blindness (RPB) Career Advancement Award for her project entitled “Modulation of Angiogenic Factors via Precision Genome Editing in AMD.” Her research focuses on developing a durable, one-time therapeutic strategy for neovascular age-related macular degeneration (nAMD) by targeting the molecular drivers of pathological blood vessel growth.

In nAMD, overproduction of vascular endothelial growth factor A (VEGF-A) leads to the formation of leaky and damaging blood vessels beneath the retina, resulting in progressive vision loss. Dr. Lapinaite’s proposal leverages next-generation genome editing technologies to address this disease mechanism at its source. Using high-fidelity CRISPR nucleases and base editors, her work aims to simultaneously and permanently reduce the expression of both VEGFA and ANGPTL4 genes in relevant human and mouse cells.

This dual-targeting strategy is designed to overcome redundancy in angiogenic signaling pathways that limits the effectiveness of current therapies. In addition, Dr. Lapinaite will reprogram the transcriptional activity of hypoxia-inducible factor 1 alpha (HIF1A) by precisely editing its protein–protein interaction and DNA-binding domains. This approach seeks to weaken pathological gene expression while preserving HIF1A’s essential physiological functions.

Together, this work aims to restore balanced regulation of angiogenic and metabolic processes involved in disease progression, offering a more comprehensive and lasting approach to treating nAMD. Insights from this research may also help inform therapeutic strategies for other ocular disorders.

The RPB Career Advancement Award, established in 2020, supports early- to mid-career vision researchers who have received their first independent National Institutes of Health (NIH) R01 grant and are generating new data to apply for a second R01. This stage has been identified as a critical gap in the vision research funding pipeline. To date, only 16 Career Advancement Awards have been granted to vision research scientists at universities across the United States.