Targeting TTR amyloidosis at its source.

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Transthyretin (TTR) amyloidosis is a rare, devastating disease with no FDA-approved treatments. Eidos Therapeutics is working to change that by developing a novel therapeutic that addresses the root cause of the condition.


TTR amyloidosis presents in two forms: a cardiomyopathy causing progressive heart failure and a polyneuropathy leading to degeneration of sensory and motor function. Both forms of the disease generally occur later in life, but can progress rapidly. Current treatment options focus on managing symptoms, but do not address the underlying cause of the disease.


People Worldwide
Suffer From TTR Cardiomyopathy

TTR cardiomyopathy

TTR cardiomyopathy is a progressive, fatal disease caused by the accumulation of misfolded TTR amyloid in the heart. These deposits are toxic to cardiac muscle cells and limit the heart’s ability to fill completely during relaxation, leading to heart failure.

More than 200,000 people worldwide suffer from TTR cardiomyopathy, though the patient population is likely underestimated due to missed diagnoses. Most patients are diagnosed after age 50, when the disease is already advanced and may progress rapidly.

Some patients are predisposed to TTR cardiomyopathy due to mutations in the gene that encodes TTR. The most common of these mutations, V122I TTR, is found in roughly 4% of African Americans.

Liver and heart transplantation remain the only disease-modifying treatments for TTR cardiomyopathy.

TTR polyneuropathy

TTR polyneuropathy is a progressive, fatal disease caused by the accumulation of misfolded TTR amyloid in the peripheral nervous system. These deposits can damage patients’ ability to move and feel, while also impairing normal cardiovascular and digestive function.

Approximately 10,000 people worldwide suffer from TTR polyneuropathy, including regions of Portugal, Japan and Sweden where disease-causing TTR mutations are endemic. The disease generally onsets later in life, after age 60, but can progress rapidly.

There are no FDA-approved treatments for TTR polyneuropathy.


People Worldwide
Suffer From TTR Polyneuropathy


Eidos Therapeutics is developing a drug candidate, AG10, to treat both forms of TTR amyloidosis. AG10 is an orally-administered, small molecule designed to potently stabilize tetrameric TTR, thereby halting at its outset the series of molecular events that give rise to ATTR. Our approach to the treatment of ATTR mimics a naturally-occurring genetic rescue mutation that protects high-risk individuals from developing ATTR by stabilizing the TTR tetramer.


TTR normally exists as a 4-part molecule.


This molecule can destabilize and dissociate into individual monomers.


These monomers are unstable, and can misfold and aggregate as amyloid fibrils that cause disease.


AG10 binds to the 4-part TTR molecules, stabilizing the structure and preventing the formation of disease-causing amyloid fibrils.

What is TTR?

Transthyretin (TTR) is a relatively abundant protein in the blood, named after its role in transporting thyroxine and retinol. Due to a genetic mutation or environmental factors, the protein can become unstable and misfold, causing it to accumulate as toxic amyloid aggregates in the heart or peripheral nerves.

Eidos aims to halt TTR amyloidosis by treating the root cause of the disease, destabilized and misfolded TTR. Inspired by a natural TTR mutation that protects carriers from the disease, Eidos is developing a small molecule (named AG10) that binds and stabilizes TTR in the blood. Pre-clinical studies have shown promising data for safety and efficacy, paving the way for clinical testing.

Selected Literature

About Us

Eidos Therapeutics is a clinical stage biopharmaceutical company focused on addressing the large and growing unmet need in diseases caused by transthyretin, or TTR, amyloidosis, or ATTR. Eidos seeks to treat this well-defined family of diseases at their collective source by stabilizing TTR.

Eidos is led by a team of industry veterans who are responsible for developing over 30 molecules through IND applications and more than 10 approved drugs. Together with patients and physicians, the company aims to bring a safe, effective and disease-modifying treatment for ATTR to market as quickly as possible.



Neil Kumar, Ph.D.

Chief Executive Officer

Jonathan Fox, M.D., Ph.D., FACC

President and Chief Medical Officer

Uma Sinha, Ph.D.

Chief Scientific Officer

Christine Siu

Chief Financial Officer

Board of Directors


Eidos is a member of the BridgeBio family

BridgeBio is a clinical-stage biotech company developing novel, genetically targeted therapies to improve the lives of patients. The BridgeBio approach combines a traditional focus on drug development with a unique corporate model, allowing rapid translation of early stage science into medicines that treat disease at its source.

Founded in 2015 by a team of industry veterans, the company has built a robust portfolio of nine transformative drugs ranging from pre-clinical to late stage development in multiple therapeutic areas including oncology, cardiology, dermatology, and rare disease. BridgeBio holds each drug candidate in a distinct subsidiary company to maximize management focus and financial flexibility.

BridgeBio’s focus on scientific excellence and rapid execution aims to translate today’s discoveries into tomorrow’s medicines.


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