Renasant Bio has officially emerged out of stealth to advance next-generation disease-modifying treatments for autosomal dominant polycystic kidney disease (ADPKD), which happens to be the leading cause behind genetic cause of end-stage renal failure.
According to certain reports, the company is developing a pipeline of oral small molecule corrector and first-in-class potentiator therapies that, on their part, directly target the underlying biology associated with ADPKD. Renasant is basically doing so to restore the function of key polycystin proteins across multiple different mutations, eventually halting disease progression.
More on the same would reveal how, to achieve the given objective, Renasant has raised a sum of $54.5 million in seed funding. Co-led by founding investor 5AM Ventures, the round saw participation coming from various other leading life sciences investors including Atlas Venture, OrbiMed, and Qiming Ventures. This particular seed financing will tread up a long distance to support further progression of the company’s lead corrector program and ongoing discovery efforts for its first-in-class potentiator program.
“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.”
Talk about the given disease on a slightly deeper level, autosomal dominant polycystic kidney disease is understood to be a chronic, progressive genetic condition, where we see progressive formation of fluid-filled cysts in the kidneys, often leading to end-stage renal failure. As for what causes this condition in the first place, the answer relates to mutations in the PKD1 and PKD2 genes that encode polycystin proteins PC1 and PC2, both critical regulators of kidney cell function.
Making treatment even more challenging is the fact that genetic mutations underlying ADPKD are extremely heterogeneous, with no single mutation found in more than 2% of patients.
“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.”
Expanding upon Renasant’s approach towards treating ADPKD, it focuses on restoring the normal function of PC1 and PC2. You see, the company would leverage its leading expertise in ADPKD biology, including electrophysiology and polycystin channel physiology proprietary, to develop assays that can assess protein trafficking and other key mechanisms for effectively facilitating precision-guided development of novel therapies to treat ADPKD.
In that context, the company lead program includes a small molecule corrector, which is designed to work across a wide range of disease-causing mutations, stabilizing and properly folding PC1/2 to promote protein trafficking and restoring kidney function.
While the stated program is currently in preclinical development, Renasant is also scaling discovery efforts towards a potentiator to enhance ion flux through the polycystin channel and support anti-cystogenic signaling.
For better understanding, correctors and potentiators can act as stand-alone therapies, or they can be seamlessly 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 at Renasant Bio. “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.”
