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Kidney disease, or renal disease, refers to when the kidneys are damaged and unable to filter blood effectively, leading to a buildup of waste and extra fluid in the body. It can be chronic, meaning it develops slowly over time, or acute, resulting from a sudden event. Early detection and treatment are crucial to prevent further damage and potential kidney failure. Fortunately, there are several biotech companies actively working on developing innovative treatments, whether it be gene therapies or small molecules, for various types of kidney disease. In this article, we take a look at six kidney disease companies trying to make a difference to patients’ lives.
Table of contents
Judo Bio
- Technology: Oligonucleotide therapies
- Approach: Delivering siRNA conjugates to kidney cells to target/silence genes
- Recent news: Raised $100 million in initial financing
In October last year, Judo Bio launched with $100 million in initial financing to develop oligonucleotide therapies delivered to the kidney, which has historically been a challenging organ to access for genetic medicines, for the treatment of systemic diseases and renal diseases. It does this with the help of its STRIKE (Selectively Targeting RNA Into KidnEy) platform.
The company’s lead pipeline programs use megalin receptors for the intracellular delivery of ligand-small interfering RNA (siRNA) conjugates to specific kidney cells to target and silence genes. The company calls these drugs megalin-STRIKERs. They are designed to silence mRNA, resulting in the reduction of specific solute carrier proteins that contribute to the homeostasis of circulating substances, including amino acids, electrolytes, glucose, and other metabolites. Targeting these proteins is an established approach for the treatment of various systemic diseases.
Judo Bio also said that data suggests that the megalin family of receptors is expressed on podocytes and cysts in polycystic kidney disease, which may open opportunities to use megalin-STRIKERs to treat a broad range of kidney diseases, too.
The company has already completed preclinical studies that successfully demonstrate receptor-mediated delivery of oligonucleotides to the kidney with its ligand-siRNA conjugates.
Maze Therapeutics
- Technology: APOL1 inhibitor
- Lead kidney disease candidate: MZE829
- Recent news: Dosed the first patient in a phase 2 trial of MZE829
As a small molecule drug discovery company, Maze Therapeutics is working on harnessing the power of human genetics to develop small molecule precision medicines. It is particularly focused on understanding genetic drivers of common diseases – an area that has traditionally been challenging in drug discovery – with its main focus areas currently being renal and cardiometabolic indications. To help it with this task, Maze’s platform, called Compass, identifies gene variants that are associated with disease and unravels what those variants do in order to identify new targets and drugs.
Maze’s lead candidate, called MZE829, is an oral APOL1 inhibitor designed to treat APOL1-mediated kidney disease. This type of kidney disease – primarily affecting individuals of African descent – is caused by inheriting two risk variants of the APOL1 gene, leading to an increased risk of chronic kidney disease and kidney failure. MZE829 is being tested in a phase 2 trial, in which the first patient was dosed in February 2025. This came after the candidate’s positive phase 1 results, which showed that it was well-tolerated at single doses up to 480 mg and multiple doses up to 350 mg, and all treatment-related adverse events were reported as mild, with no severe or serious adverse events occurring.
Maze also has another candidate in development, called MZE782, for the treatment of chronic kidney disease. The first participants were dosed in a phase 1 trial for the candidate in October 2024. MZE782 is an oral small molecule targeting the solute transporter, SLC6A19.
Since it launched with $191 million in 2019, the kidney disease company has raised a significant amount of money. In 2022, it bagged another $190 million, before bringing in $115 million in a series D round in December last year and $140 million in an initial public offering (IPO) in January this year.
Purespring Therapeutics
- Technology: Gene therapy
- Lead kidney disease candidate: PS-002
- Recent news: European Medicines Agency granted orphan drug designation to PS-002 in IgAN
Purespring Therapeutics is focused on developing gene therapies to halt or prevent kidney disease. According to the company, it is the first to successfully treat kidney disease models by directly targeting the podocyte – a specialised cell implicated in approximately 60% of renal diseases – through its proprietary adeno-associated viral (AAV) gene therapy platform.
The kidney disease company has a pipeline of programs in development that includes its lead asset for the treatment of IgA Nephropathy (IgAN) and other complement-mediated kidney diseases. In October 2024, Purespring presented new preclinical data at the American Society of Nephrology Kidney Week; it showed that mice given its gene therapy, PS-002, had lower levels of complement deposited in their kidney cells and less protein in their urine, along with healthier kidney morphology.
Also in October last year, Purespring announced that it had raised $105 million in series B funding, which it said would be used to support its pipeline, including the initiation of a phase 1/2 clinical trial for IgAN. Furthermore, in April this year, the company announced that the European Medicines Agency (EMA) had granted orphan drug designation to PS-002 in IgAN.
Revelation Biosciences
- Technology: TLR4 agonist
- Lead kidney disease candidate: Gemini
- Recent news: Raised $4 million in a public offering
Revelation Biosciences is focused on developing Gemini, an intravenously administrated formulation of phosphorylated hexaacyl disaccharide (PHAD) – a type of selective toll-like receptor 4 (TLR4) agonist – that reduces the damage associated with inflammation by reprogramming the innate immune system to respond to stress (trauma, infection, etc.) in an attenuated manner.
Gemini is being developed for multiple indications, including as a pre-treatment to prevent or reduce the severity and duration of acute kidney injury, as a pre-treatment to prevent or reduce the severity and duration of post-surgical infection, and as a treatment to stop or slow the progression of chronic kidney disease.
In June 2024, Revelation announced that a phase 1 study of Gemini, which enrolled 40 healthy individuals between 18 and 55 years old, met its primary safety endpoint and showed statistically significant dose dependent upregulation of key biomarkers, demonstrating the immunostimulatory preconditioning effect of Gemini. More recently, in February 2025, the company dosed the first patients in a phase 1b trial of Gemini in patients with stage 3 and stage 4 chronic kidney disease.
The kidney disease company also managed to close a $4 million public offering in March this year, which it said it would use to further develop its candidates, including completing the phase 1b trial.
Travere Therapeutics
- Technology: Endothelin and angiotensin II receptor antagonist
- Lead kidney disease drug: Filspari
- Recent news: FDA accepted supplemental New Drug Application for the approval of Filspari for the treatment of FSGS
Rare disease company Travere Therapeutics – previously called Retrophin before changing its name in 2020 – is advancing a pipeline of kidney and metabolic disease therapies in an effort to set new standards of care and bring hope to patients with rare disorders of this kind. The company already has two approved medications: Thiola for the treatment of cystinuria, a rare inherited kidney condition that causes a buildup of an amino acid called cystine in the urine, resulting in an increased risk of developing kidney stones, and Filspari for the treatment of IgAN.
IgAN is usually treated using angiotensin-converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs), but Filspari’s accelerated U.S. Food and Drug Administration (FDA) approval in February 2023 made Travere’s therapy the first non-immunosuppressive therapy to treat the disease. It works by blocking the endothelin-1 and angiotensin II receptors, two critical pathways in the progression of IgAN, and is intended for patients who are at risk of rapid disease progression that will likely result in end-stage renal disease. The accelerated approval for the drug was granted due to the fact that it reduced proteinuria, which in turn reduces the risk of kidney failure.
Furthermore, in May 2025, the kidney disease company announced that the FDA had accepted its supplemental New Drug Application for the approval of Filspari for the treatment of focal segmental glomerulosclerosis (FSGS), another rare kidney condition where scarring occurs in the glomeruli, the tiny filters in the kidneys.
In November 2024, Travere also managed to complete a $143.8 million public offering.
Vera Therapeutics
- Technology: Recombinant fusion protein
- Lead candidate: Atacicept
- Recent news: Entered into a new credit facility providing for up to $500 million of term loans
Focused on developing treatments for serious immunological diseases, Vera Therapeutics’ lead candidate, called atacicept, is a recombinant fusion protein that is self-administered as a subcutaneous injection once a week. It works by blocking the cytokines B-cell activating factor (BAFF) and A PRoliferation-Inducing Ligand (APRIL), both of which stimulate B cells to produce autoantibodies contributing to certain autoimmune diseases, including IgAN.
Earlier this month, Vera announced that a phase 3 trial of the drug candidate for the treatment of IgAN met its primary endpoint of reduction in proteinuria at week 36, with participants receiving atacicept achieving a 46% reduction from baseline and a 42% reduction compared to placebo at week 36. The company said it now plans to meet with the FDA in the coming weeks to discuss these results and the regulatory pathway, and it plans to submit a Biologics License Application (BLA) for accelerated approval in the fourth quarter of this year. Additionally, the phase 3 trial will continue, with two-year results expected in 2027.
Also earlier this month, the kidney disease company announced that it had entered into a new credit facility providing for up to $500 million of term loans with its current partner, Oxford Finance. Vera said that this refinancing enhances its ability to generate cash and manage its capital structure efficiently, while providing additional working capital flexibility to support commercial launch and strategic initiatives.
Chronic kidney disease market set for growth
The market for kidney disease drugs is set for substantial growth, particularly when looking specifically at the chronic kidney disease market – it is is expected to grow at a 19.6% compound annual growth rate (CAGR) from $2.2 billion in 2023 to $13.5 billion in 2033 in the seven major global markets (the U.S., France, Germany, Italy, Spain, the U.K., and Japan). This is largely driven by pipeline approvals of varying mechanisms of action and an influx of newly diagnosed patients. It is expected that the U.S. will contribute the most to this growth due to the higher prevalence of chronic kidney disease in the country, as well as the substantially higher cost of prescription medications in the U.S. compared to Europe and Japan.