Cardiac Amyloidosis Pipeline: Advancing Innovation with 20+ Companies Developing Cutting-Edge Therapies | DelveInsight

The Cardiac Amyloidosis market is rapidly advancing and is fueled by groundbreaking research and innovative therapies from companies such as Eidos Therapeutics, Intellia Therapeutics, Novo Nordisk, Alnylam Pharmaceuticals, and Precision Biosciences. These industry pioneers are transforming treatment strategies and redefining the future of Cardiac Amyloidosis, bringing new hope to patients worldwide.

DelveInsight's "Cardiac Amyloidosis Pipeline Insight, 2025" comprehensively analyzes the current clinical landscape and growth prospects in the Cardiac Amyloidosis market. The report covers disease insights, treatment guidelines, and a detailed pipeline assessment from preclinical to marketed stages. It includes drug mechanisms, clinical studies, regulatory progress, and key developments such as collaborations, mergers, funding, and designations.

For emerging Cardiac Amyloidosis drugs, the Cardiac Amyloidosis pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.

Key Takeaways from the Cardiac Amyloidosis Pipeline Report
• DelveInsight's Cardiac Amyloidosis Pipeline analysis depicts a robust space with 20+ active players working to develop 20+ pipeline drugs for Cardiac Amyloidosis treatment.
• The leading Cardiac Amyloidosis companies include Eidos Therapeutics, Intellia Therapeutics, Novo Nordisk, Alnylam Pharmaceuticals, Precision Biosciences, Prothena, Caelum Biosciences, Zentalis Pharmaceuticals, Attralus, and others are evaluating their lead assets to improve the Cardiac Amyloidosis treatment landscape.
• Key Cardiac Amyloidosis pipeline therapies in various stages of development include Acoramidis, NTLA 2001, PRX 004, Vutrisiran, ALN TTRsc04, Birtamimab, Anselamimab, ZN d5, Iodine (124I) evuzamitide, and others.
• In March 2025, Alnylam Pharmaceuticals (Nasdaq: ALNY) announced moderated poster presentations of new data from the HELIOS-B Phase 3 trial, evaluating vutrisiran for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM) in a patient population reflective of current cases.
• In January 2025, Life Molecular Imaging secured fast-track designation from the U.S. FDA for its amyloid PET radiotracer, used to visualize cardiac amyloid light-chain (AL) and amyloid transthyretin-related (ATTR) amyloidosis.
• In November 2024, Ultromics' EchoGo® Amyloidosis, an AI tool for echocardiography aimed at early cardiac amyloidosis detection, received FDA clearance. It is the first device in the FDA's Total Product Lifecycle Advisory Program (TAP) to gain marketing authorization.

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Cardiac Amyloidosis Overview
Cardiac amyloidosis is a leading cause of restrictive cardiomyopathy, often manifesting as rapidly progressive diastolic dysfunction in a non-dilated ventricle. Despite its clinical significance, it remains an underdiagnosed condition. A high index of suspicion is essential for diagnosis, with cardiovascular imaging playing a crucial role in detecting the disease.
Amyloid deposition contributes to heart disease through multiple mechanisms.

Direct interstitial infiltration results in increased ventricular wall thickness, stiffness, and subsequent diastolic dysfunction. In AL amyloidosis, amyloid deposits in arterioles can lead to angina and, in rare cases, myocardial infarction. Atrial infiltration causes structural changes that promote atrial fibrillation, while even in its absence, atrial amyloidosis heightens the risk of atrial thrombosis and thromboembolism. Additionally, light chains can induce direct myocardial injury through oxidative stress mechanisms.

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Cardiac Amyloidosis Treatment Analysis: Drug Profile
Acoramidis (AG10): Eidos Therapeutics
Eidos Therapeutics is developing Acoramidis (AG10), a small molecule engineered by Stanford University researchers to effectively stabilize transthyretin (TTR). Acoramidis mimics the structure of the T119M TTR variant, which is known to confer resistance to ATTR by forming stabilizing bonds that enhance protein stability approximately 40-fold compared to its native form. Preclinical studies indicate that Acoramidis binds to TTR tetramers at the same central sites where the T119M variant establishes stabilizing interactions, suggesting that this binding mechanism contributes to its strong TTR-stabilizing effects.

NTLA-2001: Intellia Therapeutics
NTLA-2001 is the first CRISPR/Cas9-based therapy candidate to be systemically administered for precise gene editing in humans. It is designed to inactivate the TTR gene in liver cells, thereby reducing the production of misfolded TTR protein, which accumulates in tissues and leads to the severe and often fatal complications of ATTR amyloidosis.

Key Cardiac Amyloidosis Therapies and Companies
• Vutrisiran: Alnylam Pharmaceuticals
• Acoramidis: Alexion Pharmaceuticals/Eidos Therapeutics/Bridgebio
• Eplontersen: Ionis Pharmaceuticals/AstraZeneca
• NTLA-2001: Intellia Therapeutics

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Cardiac Amyloidosis Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

By Stage
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

By Route of Administration
• Oral
• Intravenous
• Subcutaneous

By Molecule Type
• Small molecule
• Cell Therapy
• Peptides
• Polymer
• Small molecule
• Gene therapy

Scope of the Cardiac Amyloidosis Pipeline Report
• Coverage: Global
• Key Cardiac Amyloidosis Companies: Eidos Therapeutics, Intellia Therapeutics, Novo Nordisk, Alnylam Pharmaceuticals, Precision Biosciences, Prothena, Caelum Biosciences, Zentalis Pharmaceuticals, Attralus, and others.
• Key Cardiac Amyloidosis Pipeline Therapies: Acoramidis, NTLA 2001, PRX 004, Vutrisiran, ALN TTRsc04, Birtamimab, Anselamimab, ZN d5, Iodine (124I) evuzamitide, and others.

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Table of Contents
1. Introduction
2. Executive Summary
3. Cardiac Amyloidosis Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Cardiac Amyloidosis Pipeline Therapeutics
6. Cardiac Amyloidosis Pipeline: Late-Stage Products (Phase III)
7. Cardiac Amyloidosis Pipeline: Late-Stage Products (Phase III)
8. Cardiac Amyloidosis Pipeline: Mid-Stage Products (Phase II)
9. Cardiac Amyloidosis Pipeline: Early Stage Products (Phase I)
10. Therapeutic Assessment
11. Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Key Companies
14. Key Products
15. Unmet Needs
16. Market Drivers and Barriers
17. Future Perspectives and Conclusion
18. Analyst Views
19. Appendix

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This release was published on openPR.