Transthyretin (TTR) amyloidosis (ATTR) is a rare, underdiagnosed, and life-threatening disease with limited treatment options that can damage the heart and/or nervous system. BridgeBio’s mission is to improve the morbidity and mortality of patients with ATTR through the discovery and development of a novel therapeutic through its affiliate, Eidos Therapeutics.

Our Research

transthyretin (TTR)

TTR is a highly evolutionarily conserved protein that has been found to circulate in the blood of all vertebrates. As a circulating protein primarily made in the liver, TTR plays a key role in the transport of thyroxine and vitamin A and is thought to have a role in preserving cognitive function.1,2

transthyretin amyloidosis (ATTR)

ATTR cardiomyopathy, or ATTR-CM, is a form of amyloidosis caused by destabilization of TTR and subsequent accumulation of misfolded TTR protein in the myocardium. Approximately 13 to 19% of all patients with heart failure with preserved ejection fraction, commonly referred to as HFpEF, may have ATTR-CM, though it is currently significantly underdiagnosed.3 ATTR-CM often dramatically impairs the quality of life, functional independence, and life expectancy of patients, as well as impacting caregivers due to the progressive nature of the disease. If left untreated, life expectancy from diagnosis is approximately four years.

ATTR can be caused by genetic variants, which can be associated with either destabilization or increased stabilization. Science has found:

  • In more than 130 destabilizing ATTR-causing variants, the severity of disease is associated with the level of destabilization caused by each variant.
  • Individuals who inherit stabilizing variants like T119M are partially or completely protected against the development of ATTR.

presentations & publications

ISA 2024 – Early increase in serum transthyretin level is an independent predictor of improved survival in ATTR cardiomyopathy 05.29.2024
ISA 2024 – Acoramidis treatment-related increase in serum TTR is associated with lower cardiovascular mortality in ATTR-CM 05.29.2024
ISA 2024 – Acoramidis treatment-related increase in serum TTR is associated with a lower risk of cardiovascular hospitalization in ATTR-CM patients 05.29.2024
ISA 2024 – Acoramidis achieves early reduction in cardiovascular death or hospitalization in transthyretin amyloid cardiomyopathy (ATTR-CM) 05.29.2024
ISA 2024 – Higher risk of mortality in previously hospitalized patients 05.29.2024
ISA 2024 – Rationale & design of ACT-EARLY 05.29.2024
ESC Heart Failure 2024 – ITT Sensitivity Analysis and Sub-Analysis Comparing Acoramidis and Placebo in Stage 4 CKD in ATTRibute-CM 05.11.2024
ESC Heart Failure 2024 – Improved Health-Related Quality of Life in Acoramidis-Treated Patients with ATTR-CM, Demonstrated by Improvements in KCCQ Scores 05.13.2024
ESC Heart Failure 2024 – EQ-5D Analysis from the ATTRibute-CM Study 05.13.2024
ESC Heart Failure 2024 – NT-proBNP Insights from the ATTRibute-CM Study 05.13.2024
ACC 2024 – CMR Imaging Substudy from ATTRibute-CM Phase 3 Study 04.07.2024
The New England Journal of Medicine, Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy 01.10.2024
AHA 2023 – Clinical Outcome Improvements 11.12.2023
HFSA 2023 – 4 Year Update from OLE Phase 2 Study 10.08.2023
ATTRibute-CM Detailed Results From ESC Congress 2023 08.28.2023
ESC 2023 – ATTRibute-CM-Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy 08.27.2023
ESC 2023 – Acoramidis Produces Near-Complete TTR Stabilization in Variant ATTR Patients that is Greater than that Achieved with Tafamidis 08.26.2023
ATTRibute-CM Phase 3 Topline Results 07.17.2023
ISA 2022 – updated analysis ongoing phase 2 ole 08.12.2022
ACC 2022 – updated analysis ongoing phase 2 ole 04.02.2022
ACC 2022 – unmet needs among patients with attr 04.03.2022
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  1. Liz MA, et al. Neurol Ther. 2020;9(2):395-402.
  2. Vieira M, Saraiva MJ. Biomol Concepts. 2014;5(1):45-54.
  3. AbouEzzeddine OF, Davies DR, Scott CG, et al. Prevalence of Transthyretin Amyloid Cardiomyopathy in Heart Failure with Preserved Ejection Fraction. JAMA Cardiol. 2021;6(11):1267-1274. doi:10.1001/jamacardio.2021.3070