Drug Discovery
We’re initially focused on cardiomyopathies and myocardial fibrosis, aiming to build the most advanced drug discovery engine for tackling this critical unmet need.
Cardiomyopathies
Cardiomyopathies are a group of diseases that affect the structure and function of the heart muscle, often leading to progressive heart failure, arrhythmias, and sudden cardiac death. These disorders can arise from genetic mutations, systemic diseases, or environmental stressors, and they are typically classified into several subtypes — including dilated, hypertrophic, restrictive, and arrhythmogenic cardiomyopathy.
What unites all cardiomyopathies is a fundamental disruption of the heart’s ability to contract and relax effectively. Over time, this leads to maladaptive remodeling of the myocardium, impaired cardiac output, and increasing clinical burden. Many patients develop symptoms such as breathlessness, fatigue, and fluid retention — and in severe cases, may require implantable devices or heart transplantation.
Despite advances in diagnosis and patient management, cardiomyopathies remain an area of significant unmet medical need. Treatments today are largely supportive — aimed at alleviating symptoms or slowing disease progression — but they do not target the underlying molecular or cellular drivers of disease.
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~60% have a genetic basis.
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~40% are thought to have a genetic cause.
- Marian, Ali J., and Braunwald, Eugene. “Hypertrophic Cardiomyopathy: Genetics, Pathogenesis, Clinical Manifestations, Diagnosis, and Therapy” Circulation Research, (2017). 21(7):749-770.
- McNally, Elizabeth M., and Mestroni, Luisa. “ Dilated Cardiomyopathy: Genetic Determinants and Mechanisms” Circulation Reseach. (2017). 121(7):731-748.
- Corrado, Domenico et al. “Arrhythmogenic Cardiomyopathy”. Circulation Research. (2017). 121(7):784-802
Myocardial Fibrosis
Myocardial fibrosis is a pathological process characterized by the excessive accumulation of fibrotic tissue in the heart muscle. It occurs when the normal balance between collagen production and degradation is disrupted, typically following injury, inflammation, or sustained cardiac stress. Over time, this fibrotic remodeling leads to stiffening of the myocardium, impaired electrical conduction, and reduced contractile function — ultimately contributing to the progression of heart failure.
Fibrosis is particularly common after myocardial infarction (MI), where damaged heart tissue is replaced by non-functional scar tissue. While some degree of scar formation is necessary for structural stability, excessive or unresolved fibrosis compromises the heart's ability to pump effectively. In chronic conditions, such as hypertensive heart disease or cardiomyopathies, diffuse interstitial fibrosis can develop gradually, often undetected, leading to diastolic dysfunction and arrhythmias.
Despite being a central driver of heart failure progression, myocardial fibrosis remains largely untargeted by existing therapies. Current treatments focus primarily on managing symptoms or underlying risk factors, such as hypertension or ischemia, but do not directly reverse or halt the fibrotic process.
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individuals
of HFpEF patients
of HCM patients
- Savarese, Gianluigi, et al. "Global burden of heart failure: a comprehensive and updated review of epidemiology." Cardiovascular research 118.17 (2022): 3272-3287.
- Bozkurt, Biykem et al., “HF STATS 2024: Heart Failure Epidemiology and Outcomes Statistics An Updated 2024 Report from the Heart Failure Society of America.” Journal of Cardiac Failure. (2025). 31(1):66-116.
- Mewton et al., “Assessment of Myocardial Fibrosis With Cardiovascular Magnetic Resonance”, JACC, (2011). 57 (8): 891-903.
- Habib et al, “Progression of Myocardial Fibrosis in Hypertrophic Cardiomyopathy” JACC: Cardiovascular Imaging (2021). 14 (5): 947-58.
- Hahn et al., “Endomyocardial Biopsy Characterization of Heart Failure With Preserved Ejection Fraction and Prevalence of Cardiac Amyloidosis”, JACC: Heart Failure (2020). 8 (9):712-24.
Discovery Strategy
We use our proprietary human disease models to uncover novel, disease-relevant biological targets. Once identified, we conduct target-based screens to find small molecules or nucleotide-based modulators that can specifically influence these targets. This enables us to start from a mechanistic understanding of disease biology and build a rational path toward therapeutic intervention.
We run unbiased phenotypic screens in our human cardiac disease models to identify compounds that produce meaningful, disease-modifying effects, without necessarily relying on prior knowledge of the target. This approach allows us to capture complex biology and discover first-in-class compounds that might be missed in traditional target-driven approaches.
We actively in-license promising lead compounds from external sources. We focus on assets with a strong mechanistic rationale, preliminary in vitro proof-of-concept data, and a validated target hypothesis. These assets are then further validated and optimized using our platform to accelerate their path toward clinical development.
Partnering Strategy
We seek strategic partnerships with pharma and biotech companies focused on developing novel small molecule or nucleotide therapies for heart failure. By integrating our human-centric drug discovery platform with industry leaders’ therapeutic expertise, complementary technologies, and development capabilities, we aim to accelerate breakthrough treatments and deliver them more efficiently.
We engage with leading academic innovators working on next-generation therapeutics for heart failure. Through co-development or in-licensing opportunities, we seek to translate pioneering research into clinically viable drug candidates—leveraging our platform to de-risk early-stage assets and streamline their progression toward the clinic.
We partner with technology providers that enhance our platform across key areas such as assay development, high-throughput compound screening, data analytics, and AI-driven drug discovery. Specifically, we’re looking for collaborators offering capabilities in target prediction, chemo-informatics, and molecular design to strengthen our ability to generate and optimize novel small molecule therapies.
Cooperation Partners
Partner with us
