Sorin Fedeles, PhD, MBA

My work sits at the intersection of discovery science and translational medicine, with a long-standing focus on inherited kidney and liver disorders — particularly Autosomal Dominant Polycystic Kidney Disease (ADPKD), ARPKD, and ADTKD. Over the course of my career, I have operated at the intersection among academia, biotech start-ups, and non-profit organizations, driven by the conviction that the distance between a laboratory finding and a meaningful therapy should be as short as possible.

At the heart of my research is the question of what drives PKD — and what we can do about it. My lab has developed several genetic ADPKD mouse models, including work that produced one of the seminal papers in the field, defining how PC1 dosage shapes disease pathogenesis. That insight has since become a cornerstone of drug discovery programs across the pharmaceutical industry. Building on that foundation, we have explored chaperone therapies using hypomorphic ADPKD models, deployed whole-genome CRISPR/Cas9 forward screens coupled with RNAseq to uncover novel therapeutic targets, demonstrated for the first time the potential of in vivo base editing to slow ADPLD progression, and developed a proof-of-concept model probing ER protein folding in the context of PKD1 missense mutations. This body of work has yielded five patents published or filed, a method-of-use patent issued, and productive collaborations — including an ongoing partnership with a bioengineering group at MIT.

As a co-founder of academic spin-out companies rooted in Yale research, I lead scientific, pre-clinical, and business development strategy with the goal of turning promising biology into real treatments. As Principal Investigator, I have secured more than $10 million in competitive extramural funding from the NIH, Department of Defense, PKD Foundation, European ERDERA program, and the FDA.

I currently serve as Executive Director of the Polycystic Kidney Disease Outcomes Consortium at the Critical Path Institute, where I lead an interdisciplinary international team working to develop clinical biomarkers, PRO/COA instruments, and clinical trial simulator tools for ADPKD, ARPKD, and ADTKD. It is work that connects everything I care about — rigorous science, patient relevance, and the practical demands of getting new therapies across the finish line.

My research has been published in Nature Genetics, JCI, PNAS, Molecular Therapy, JASN, and other leading journals, with more than 75 publications, abstracts, and presentations to date. I also remain deeply committed to mentorship and to building the kind of teams — across academia, venture, and non-profit settings — that can take on hard problems and see them through.