Renasant Bio launches with $54.5M to tackle rare kidney disease

Renasant Bio raised $54.5 million in seed funding to develop treatments for autosomal dominant polycystic kidney disease (ADPKD), a rare and progressive kidney disorder that also happens to be the most common genetic cause of renal failure.

Why it matters: ADPKD affects roughly 12 million people worldwide but has limited treatment options. Renasant’s approach, inspired by cystic fibrosis therapies, could lead to transformative new drugs targeting the disease at its biological root.

Backstory: The only FDA-approved drug for ADPKD, Otsuka’s Jynarque, was cleared in 2018 but only slows down the progression of the disease. Sanofi, which also developed a drug for the condition, halted its program in 2021 due to poor trial results. Recently, interest has been rekindled by Novartis and its acquisition of a promising candidate from Regulus Therapeutics for $800 million in April.

Big picture: Renasant is building on the success of cystic fibrosis drugs such as Vertex‘s Trikafta, which work by fixing broken ion channels in lung cells. Renasant is applying the same idea to kidney cells affected by ADPKD, aiming to correct the faulty PKD1 and PKD2 proteins that disrupt fluid flow and cell signaling.

Zoom in: Renasant’s lead candidate is a small molecule “corrector” currently in preclinical testing. A second compound, a “potentiator,” is also in development to enhance treatment efficacy. Clinical trials are planned for the coming years.