ADPKD

ADPKD (Autosomal Dominant Polycystic Kidney Disease) is our lead indication, spearheading our pipeline of mRNA-upregulating small molecules.
In this portal you'll find an overview of the disease, market maps, and insights into the ecosystem around it.

ADPKD at a glance. Disease: an inherited disease in which fluid-filled cysts progressively enlarge the kidneys, eventually leading to kidney failure. A normal kidney is fist size; a polycystic kidney grows up to football size. About 140K diagnosed in the US, about 12M affected worldwide, and over 50% develop kidney failure by age 60. Systemic: ADPKD affects multiple organs, not just the kidneys. About 10% have brain aneurysms (4X the general population), about 50% high blood pressure (2X the general population), and about 80% liver cysts. Also associated with chronic pain, heart valve problems, hernias, and pancreatic cysts. Market and unmet need: Novartis acquired Regulus for $1.7B (2025): $800M upfront after phase 1 plus up to $900M in milestones, for an ASO (bi-weekly injection) that blocks miR-17 and increases PKD1/2. Tolvaptan peak sales were $1.6B (2024). It is the only approved drug, it is not disease modifying, it has severe side effects, and generics have been available since 2025. Market is open. Patients are waiting.

Market maps

Active Programs

Every ADPKD program in development, by modality and stage

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ADPKD History

Active and discontinued programs, from first evidence to today

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Cell Map

Therapeutic programs mapped onto a cyst-lining kidney cell

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Ecosystem

Investors

Who backs companies with ADPKD programs, round by round

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Pharma

Big pharma's ADPKD programs, deals, investments and trials

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Foundations

Funding, data, events and partnering support for ADPKD research

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Researchers

US-based researchers and their ties to companies with ADPKD programs

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Insights

How the ADPKD maps started

From a conference tag to a market map, its history and a cell model

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Who invests in ADPKD

167 investors, 54 rounds: who backs ADPKD companies, what they back and when

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What big pharma is doing in ADPKD

21 of the top 50 pharma, 48 activities: who moved in ADPKD, when, what and how much

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