Renasant Bio Launches to Pioneer Next-Generation Disease-Modifying Small Molecule Treatments for ADPKD
'ADPKD is a devastating genetic disorder that affects more than 12 million people globally and still lacks truly disease-modifying therapies,' said Emily Conley, Ph.D., chief executive officer of Renasant. 'Our platform of differentiated correctors and first-in-class potentiator therapies are designed to directly address the underlying cause of ADPKD across its broad mutation spectrum, offering the potential to benefit the vast majority of patients.'
Next-Generation Corrector and Potentiator Programs for ADPKD
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Autosomal dominant polycystic kidney disease is a chronic, progressive genetic condition marked by the progressive formation of fluid-filled cysts in the kidneys, often leading to end-stage renal failure. The condition arises from mutations in the PKD1 and PKD2 genes, which encode polycystin proteins PC1 and PC2—critical regulators of kidney cell function. The genetic mutations underlying ADPKD are extremely 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.
Addressing the Underlying Biological Drivers of ADPKD
Renasant's therapeutic strategy is centered on restoring the normal function of PC1 and PC2. Through its leading expertise in ADPKD biology, including electrophysiology and polycystin channel physiology, Renasant has developed proprietary assays to assess protein trafficking and other key mechanisms, enabling precision-guided development of novel therapies for ADPKD.
The company's lead program is a small molecule corrector designed to work across a wide range of disease-causing mutations to stabilize and properly fold PC1/2, promoting protein trafficking to restore kidney function. The program is currently progressing through preclinical development. In addition, Renasant is advancing discovery efforts toward a potentiator to enhance ion flux through the polycystin channel and support anti-cystogenic signaling—a first-in-class approach in ADPKD. Correctors and potentiators can act as stand-alone therapies, or they can be combined for synergistic effect.
'Our corrector program is designed to work across a broad range of mutations, and our potentiator represents a novel mechanism of action in ADPKD,' said Gus Gustafson, Ph.D., chief scientific officer. 'These programs hold the potential to reshape the treatment landscape. We are very encouraged by our preclinical data and committed to rapidly advancing these programs for patients in urgent need.'
Team of Leading Experts in ADPKD
Renasant is led by a world-class team with deep expertise in ADPKD biology and a proven track record in building biopharma companies. The team includes researchers who have shaped the field of polycystin biology and electrophysiology, seasoned drug developers, and biotech executives experienced in advancing cutting-edge scientific approaches.
'5AM Ventures founded Renasant around a bold idea: that we could change the trajectory of ADPKD,' said Deborah Palestrant, Ph.D., partner at 5AM Ventures. 'ADPKD is one of the most complex diseases in renal medicine, but Renasant has assembled the right team, with years of research experience in polycystic disease that has informed the right scientific approach. We're proud to have supported the company from the start and are energized by its progress and commitment to delivering truly disease-modifying treatments for ADPKD patients.'
Renasant's executive team includes:
Emily Conley, Ph.D., chief executive officer
Gus Gustafson, Ph.D., chief scientific officer
B. Barry Touré, Ph.D., senior vice president, drug discovery
Rachel Gallagher, Ph.D., vice president, biology
Michelle Ho Huey, Ph.D., vice president, strategy & operations
Renasant's board of directors includes:
Natalie Holles, chair of the board
Kevin Bitterman, Ph.D., partner, Atlas Venture
Evan Caplan, M.D., principal, OrbiMed
Emily Conley, Ph.D., chief executive officer
Anna French, D. Phil, managing partner, Qiming Venture Partners USA
Charlotte McKee, M.D., chief medical officer, Sionna Therapeutics
Deborah Palestrant, Ph.D., partner, 5AM Ventures
In addition, Renasant has assembled a scientific advisory board consisting of preeminent leaders in ADPKD genetics, biology, and both preclinical and clinical drug development.
About Renasant Bio
Renasant Bio is a biopharmaceutical company pioneering disease-modifying oral small molecule treatments for autosomal dominant polycystic kidney disease (ADPKD), the leading genetic cause of end-stage renal failure. Renasant is advancing a pipeline of oral small molecule corrector and potentiator programs that target the underlying biology of ADPKD to restore the function of key proteins to halt disease progression. Renasant was founded by and incubated within the 4:59 Initiative, the company creation engine of 5AM Ventures, and is based in Berkeley, California. For more information, please visit us at www.renasantbio.com and follow us on LinkedIn and X.
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