As ATTR Amyloidosis becomes increasingly prevalent worldwide and contributes to comorbidities such as diabetes, cardiovascular disease, and certain cancers, the need for safer and more effective therapies continues to grow. DelveInsight reports that more than three pharmaceutical and biotech companies are actively advancing over three therapeutic candidates targeting ATTR Amyloidosis. These candidates are in various stages of clinical and preclinical development, reflecting significant innovation and dedication to addressing this major public health concern.
The “ATTR Amyloidosis Pipeline Insight 2025” report by DelveInsight provides a comprehensive strategic overview of the current R&D landscape, examining clinical trial progress, emerging therapies, mechanisms of action, competitive positioning, and key company initiatives. It is an essential resource for researchers, healthcare investors, and decision-makers seeking insights into the evolving ATTR Amyloidosis therapeutics market and the breakthroughs shaping its future.
Explore the Cutting-Edge Landscape of ATTR Amyloidosis Drug Development @ https://www.delveinsight.com/report-store/attr-amyloidosis-pipeline-insight?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr
Key Takeaways from the ATTR Amyloidosis Pipeline Report
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DelveInsight’s report on the ATTR Amyloidosis pipeline highlights a dynamic landscape, with over three active companies developing multiple therapeutic candidates for ATTR Amyloidosis.
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In March 2025, Alnylam announced FDA approval of AMVUTTRA® (vutrisiran), the first RNAi therapy shown to reduce cardiovascular death, hospitalizations, and urgent heart failure visits in adults with ATTR Amyloidosis with cardiomyopathy (ATTR-CM).
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Leading companies, including Intellia Therapeutics, YolTech Therapeutics, Alexion Pharmaceuticals, and others, are actively pursuing novel therapies to advance the ATTR Amyloidosis treatment landscape. Notable pipeline candidates in various stages of development include NTLA-2001, YOLT-201, and additional therapies.
ATTR Amyloidosis Overview:
ATTR-CM is a form of systemic amyloidosis characterized by the accumulation of misfolded transthyretin (TTR) protein in the heart, leading to impaired cardiac function. While light-chain (AL) amyloidosis can also affect the heart, transthyretin amyloidosis is the more common cause of cardiac involvement.
The disease can impact multiple organs, though in some cases the heart is the only affected site. Historically, diagnosing ATTR-CM has been difficult due to limited diagnostic tools. However, advances in bone scintigraphy and the development of new therapies have improved diagnostic accuracy. Consequently, ATTR-CM is now more frequently detected, particularly in patients with heart failure with preserved ejection fraction (HFpEF), a population previously not recognized as high-risk.
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ATTR Amyloidosis Pipeline Analysis
The ATTR Amyloidosis pipeline insights report 2025, provides insights into:
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Provides comprehensive insights into key companies developing therapies in the ATTR Amyloidosis Market.
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Categorizes ATTR Amyloidosis therapeutic companies by development stage: early, mid, and late-stage.
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Highlights major companies involved in targeted therapy development, including both active and inactive (paused/discontinued) projects.
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Reviews emerging ATTR Amyloidosis drugs under development based on:
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Stage of development
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ATTR Amyloidosis Route of administration
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Target receptor
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Monotherapy vs. combination therapy
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ATTR Amyloidosis Mechanism of action
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Molecular type
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Offers detailed analysis of:
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Company-to-company and company-academia collaborations
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ATTR Amyloidosis Licensing agreements
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Funding and investment activities supporting future ATTR Amyloidosis market advancement.
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Unlock key insights into emerging ATTR Amyloidosis therapies and market strategies here: https://www.delveinsight.com/report-store/attr-amyloidosis-pipeline-insight?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr
ATTR Amyloidosis Emerging Drugs
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NTLA-2001: Intellia Therapeutics
NTLA-2001 is an innovative CRISPR/Cas9-based therapy under development as a potential single-dose treatment for transthyretin (ATTR) amyloidosis. Its mechanism targets the TTR gene, silencing it to prevent the production of the misfolding-prone transthyretin protein that drives the disease. Importantly, NTLA-2001 represents the first experimental CRISPR therapy designed for systemic in vivo gene editing, allowing direct modification of genes within human cells throughout the body.
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YOLT-201: YolTech Therapeutics Co., Ltd
YOLT-201 Injection is an experimental therapy for transthyretin (ATTR) amyloidosis that uses lipid nanoparticles (LNPs) to deliver genetic material into the body. The formulation includes ionizable lipids and other components that encapsulate mRNA and single-guide RNA (sgRNA) to form LNPs.
After intravenous administration, Apolipoprotein E (ApoE) binds to the LNP surface in the bloodstream, allowing liver cells—which express the LDLR receptor—to take up the nanoparticles via endocytosis, creating intracellular compartments known as endosomes.
ATTR Amyloidosis Pipeline Therapeutic Assessment
ATTR Amyloidosis Assessment by Product Type
• Mono
• Combination
• Mono/Combination
ATTR Amyloidosis 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
ATTR Amyloidosis Assessment by Route of Administration
• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical
ATTR Amyloidosis Assessment by Molecule Type
• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
Download sample pages to get an in-depth assessment of the emerging ATTR Amyloidosis therapies and key ATTR Amyloidosis companies
Table of Contents
1. Report Introduction
2. Executive Summary
3. ATTR Amyloidosis Current Treatment Patterns
4. ATTR Amyloidosis - DelveInsight's Analytical Perspective
5. Therapeutic Assessment
6. ATTR Amyloidosis Late-Stage Products (Phase-III)
7. ATTR Amyloidosis Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. ATTR Amyloidosis Discontinued Products
13. ATTR Amyloidosis Product Profiles
14. ATTR Amyloidosis Key Companies
15. ATTR Amyloidosis Key Products
16. Dormant and Discontinued Products
17. ATTR Amyloidosis Unmet Needs
18. ATTR Amyloidosis Future Perspectives
19. ATTR Amyloidosis Analyst Review
20. Appendix
21. Report Methodology
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