Correctors and Potentiators for Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Transforming the Treatment of ADPKD
At Renasant Bio, we are pioneering a new approach to treating autosomal dominant polycystic kidney disease (ADPKD), a devastating genetic disorder with no disease-modifying therapies and the leading genetic cause of end-stage renal failure. Our mission is to develop small molecule correctors and potentiators that target the root cause of the disease, restoring the function of polycystin 1 and 2 (PC1/2) to halt disease progression.
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About ADPKD
Autosomal dominant polycystic kidney disease (ADPKD) is one of the most common inherited kidney disorders, affecting an estimated 12 million people worldwide. Characterized by the progressive development of fluid-filled cysts in the kidneys, ADPKD leads to organ enlargement, chronic pain, hypertension, and ultimately kidney failure in the majority of patients.
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Despite its prevalence and impact, the current standard of care remains limited—focusing primarily on symptom management and blood pressure control. Only one FDA-approved treatment exists, with modest effects on slowing disease progression. For most individuals living with ADPKD, there are no approved therapies that address the underlying cause or halt the relentless decline in kidney function.
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At Renasant Bio, we aim to change that.
Our Science
Targeting the Biology Behind ADPKD
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ADPKD is driven by mutations in the PKD1 and PKD2 genes, which encode PC1 and PC2—key proteins responsible for kidney function. When these proteins are misfolded or dysfunctional, cysts form in the kidneys, leading to progressive loss of renal function. Our approach restores the normal function of PC1/2 with two classes of small molecules:
Correctors
​Small molecule chaperones that stabilize PC1/2, restoring proper folding and trafficking to the cilia surface where they are needed for normal function.
Potentiators
​Small molecules that hold the polycystin channel open, enhancing ion flux and restoring crucial signaling pathways.
Correctors and potentiators can act as stand-alone therapies, or they can be combined for synergistic effect.
Reversing Disease Progression
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In preclinical studies, restoring PC1/2 function not only stops the progression of ADPKD but actively reverses cyst growth and improves kidney function[1]. By developing therapies that work across a broad range of mutations, we aim to provide a treatment option for the largest possible number of patients.
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A Precision Approach for a Complex Disease
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ADPKD is highly heterogeneous, with no single mutation found in more than 2% of patients. Disease‑modifying strategies that correct or potentiate polycystin biology must overcome this diversity—making therapies that work broadly across mutations a key treatment advancement for ADPKD. Current therapies do not directly address the underlying protein dysfunction and are limited in their applicability and tolerability.
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We are developing a pipeline of oral small molecule corrector and first-in-class potentiator therapies that directly target the underlying biology of ADPKD, with the goal of restoring the function of key polycystin proteins across different mutations to halt disease progression.
1. Dong, K et al., Nature Genetics 2021.
Leadership Team
We are led by a world-class team with deep expertise in ADPKD biology and a proven track record in building biopharma companies.
Board of Directors
Scientific Advisory Board
Jeremy Reiter, MD, PhD
Scientific Co-Founder, Renasant Bio;
Professor, Biochemistry & Biophysics,
University of California, San Francisco
Markus Delling, PhD
Scientific Co-Founder, Renasant Bio;
Associate Professor, Physiology,
University of California, San Francisco
Stefan Somlo, MD
Professor of Medicine & Genetics, Yale University
Peter Harris, PhD
Director, Mayo Clinic Polycystic Kidney Disease Center;
Professor of Biochemistry, Molecular Biology and Medicine, Mayo Clinic
Sorin Fedeles, PhD, MBA
Executive Director, PKD Outcomes Consortium; Adjunct Faculty, Yale University
Bob McDowell, PhD
Chief Scientific Officer, Kardigan;
Former SVP of Research, Bristol Myers Squibb
Neera K. Dahl, MD, PhD
Professor of Medicine, Mayo Clinic Alix School of Medicine, Division of Nephrology and Hypertension;
Associate Director, Mayo Clinic Translational PKD Center;
Director, Mayo Clinic Center of Excellence in PKD
Investors
Latest News
Careers
We’re seeking teammates who are energized by challenges and driven to make a meaningful impact on the lives of the patients we serve. If you value creativity and candor, thrive in the face of obstacles, and are passionate about empowering others to succeed, we invite you to join us. Together, we are advancing our mission to discover therapies for polycystic kidney disease.
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