Caroline Hendry, PhD, MBA
Research ScientistCards
About
Non nobis solum nati sumus
(Not for ourselves alone are we born - Marcus Tallius Cicero)
Research
Overview
My research interest is in how genetic discoveries become diagnoses and therapies for patients, and the translational and regulatory pathways that get them there.
In genetic therapies, I am a Co-Investigator on the NIH-funded program developing the STEP non-viral CRISPR delivery platform for neurogenetic disease, where I share responsibility for regulatory strategy, manufacturing and pre-IND research. My recent article with Jiangbing Zhou and Yong-hui Jiang (Nature Genetics, 2026) sets out how the FDA’s plausible mechanism pathway applies across the rare disease landscape.
In diagnosis and population health, I am interested in how genetic testing can be scaled beyond the specialist clinic and delivered within a health system. I am a Co-Investigator on the Yale Diagnostic Center of Excellence for Undiagnosed Diseases, part of the NIH Undiagnosed Diseases Network, and on the Yale Population Genomic Screening Pilot. I also partner with Yong-hui Jiang to guide one of the Dean’s precision medicine pilots, which tests this model in familial hypercholesterolemia, using AI to identify at-risk patients in the electronic health record.
I have also written about how academic departments can structure scientific leadership (Cell, 2023).