About Matthew Lefkowitz

I am a translational scientist with a background in biology, specifically cell and molecular biology, with a focus on therapeutic development. My training has been shaped by a strong interest in rare disease biology and the challenge of connecting fundamental scientific insight to the discovery and development of effective therapies.

I recently completed my PhD in Biosciences at George Mason University, where my work focused on cell and molecular biology. I also serve as a PreDoc IRTA Fellow at the National Institutes of Health, where I have worked in the Neuromuscular and Neurogenetic Disorders of Childhood Section at NINDS and the Therapeutic Development Branch at NCATS.

My current research centers on alpha-dystroglycanopathies and uses AI-based machine learning and deep-learning tools to examine links between pathogenic variants, functional impairment, and clinical phenotype severity. This work reflects my broader interest in using analytical and translational approaches to better understand disease mechanisms and support therapeutic development.

In addition to disease-focused research, I have worked on both in silico and in vitro drug discovery questions. My prior work includes identifying potential activating compounds for human IspD, creating 3D drug-candidate models, running molecular docking studies, generating assay standard curves, and evaluating enzyme-substrate productivity experimentally.

Across my scientific training, I have built experience in computational analysis, pathway modeling, molecular biology, scientific communication, and cross-functional problem solving. I am especially interested in roles where disease biology, translational research, and therapeutic innovation intersect.