ATTR Amyloidosis Clinical Trials Analysis 2025: RNA-silencing, TTR Stabilizers, and One-time Gene-Editing Approaches Aim to Halt Amyloid Deposition and Restore Organ Function | DelveInsight

DelveInsight's "ATTR Amyloidosis - Clinical Trials Analysis, 2025" describes a crowded, fast-moving development landscape focused on two complementary goals: (1) suppress hepatic TTR production or stabilize circulating TTR to stop further amyloid deposition, and (2) reverse organ dysfunction (cardiac and neuropathic) and improve survival, functional capacity, and quality of life. Ongoing programs span TTR stabilizers, RNA-silencing/ASO agents for transthyretin reduction, repeat-dose oligonucleotides, and emerging in-vivo gene-editing candidates that could offer one-time durable benefit.

Late-stage and registration-enabling efforts include TTR stabilizers (tafamidis and newer small molecules), RNA-targeting drugs such as vutrisiran and eplontersen showing meaningful cardiac and neuropathy signals, and investigational in-vivo CRISPR/Cas9 gene-editing programs (NTLA-2001 / Nexiguran) advancing toward pivotal evaluation - a potential paradigm shift if safety and durable TTR knockdown are confirmed. Trial endpoints prioritize mortality/CV events, 6-minute walk distance, cardiac biomarkers (NT-proBNP), neuropathy scores/mBMI, and quality-of-life measures.

Combination strategies and real-world comparative studies are increasing (e.g., RNAi ± TTR stabilizer), and regulatory momentum has accelerated with multiple product approvals and high-profile pivotal readouts that are reshaping standard of care in ATTR-CM and ATTR-PN. Biomarker-guided enrollment and cardiac/neurologic outcome composites are now standard in late-phase trials to demonstrate both organ benefit and clinical meaningfulness. Upcoming data and regulatory decisions over the next 12-24 months will determine whether newer silencers and gene-editing approaches become first-line options alongside or ahead of traditional stabilizers.

Explore the full pipeline analysis for ATTR Amyloidosis and uncover key opportunities @ https://www.delveinsight.com/report-store/attr-amyloidosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Key Takeaways from the ATTR Amyloidosis Pipeline Report
• DelveInsight's ATTR amyloidosis pipeline analysis depicts a strong space with 10+ active players working to develop 10+ pipeline drugs for ATTR amyloidosis treatment.
• The leading ATTR amyloidosis companies include Alnylam Pharmaceuticals, Novo Nordisk, Sirnaomics, Arbor Bio, and others, who are evaluating their lead assets to improve the ATTR amyloidosis treatment landscape.
• Key ATTR amyloidosis pipeline therapies in various stages of development include Vutrisiran, NN-6019, ALN-TTRsc04, STP152G, ABO-102, and others.
• In May 2025, AccurEdit Therapeutics announced that its in vivo gene editing therapy, ART001, received FDA Regenerative Medicine Advanced Therapy (RMAT) designation. ART001 is the first gene editing product from China to earn this designation, supporting its expedited development for treating ATTR amyloidosis.
• In March 2025, Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced that WAINZUA (eplontersen), developed in collaboration with AstraZeneca, has been approved in the European Union for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR-PN or ATTRv-PN) in adult patients with stage 1 or stage 2 polyneuropathy. WAINZUA is the only approved treatment in the EU for ATTRv-PN that can be self-administered monthly via an auto-injector.
• In March 2025, Alnylam Pharmaceuticals announced moderated poster presentations of new data from the HELIOS-B Phase III trial, evaluating vutrisiran for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM) in a patient population representative of today's cases.
• In November 2024, Alnylam Pharmaceuticals (ALNY) announced that the FDA has accepted its supplemental new drug application (sNDA) for review, seeking label expansion of Amvuttra (vutrisiran) to treat ATTR amyloidosis with cardiomyopathy (ATTR-CM).

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ATTR Amyloidosis Overview
Transthyretin amyloidosis (ATTR), also known as cardiac amyloidosis or ATTR-CM, is a condition where misfolded transthyretin (TTR) protein accumulates in tissues, particularly the heart. The liver normally produces TTR and carries retinol and thyroxine, but structural changes in TTR lead to misfolding and aggregation. These deposits primarily affect the heart and peripheral nerves, causing a range of symptoms, including nerve damage that leads to loss of movement and temperature sensitivity. The onset of hereditary ATTR (hATTR) symptoms typically occurs between the mid-20s and mid-60s and worsens over time. Diagnosis is confirmed through tissue biopsy with histopathology and immunohistochemistry, though extracardiac tissue samples may have unreliable sensitivity.

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ATTR Amyloidosis Treatment Analysis: Drug Profile
Vutrisiran: Alnylam Pharmaceuticals
Vutrisiran is an investigational RNAi therapeutic, administered subcutaneously, designed to treat both hereditary and wild-type ATTR amyloidosis. It targets and silences specific messenger RNA to block the production of transthyretin (TTR) protein, potentially preventing the formation of TTR amyloid deposits. Administered quarterly or biannually, vutrisiran may help reduce amyloid accumulation and restore function in affected tissues. It uses Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, offering increased potency and metabolic stability for infrequent injections. The safety and efficacy of vutrisiran have not yet been evaluated by regulatory authorities, and it is currently in Phase III development.

NN-6019: Novo Nordisk
NN-6019 is an investigational humanized monoclonal antibody designed to target and deplete amyloid deposits in hereditary and wild-type ATTR amyloidosis (hATTR and wtATTR) without affecting the normal tetrameric form of TTR. Preclinical studies have shown that NN-6019 promotes the clearance of insoluble amyloid fibrils through antibody-mediated phagocytosis and inhibits amyloid formation. This approach has the potential to benefit ATTR patients at high risk for early mortality due to amyloid accumulation in vital organs. NN-6019 is currently in Phase II clinical trials for the treatment of ATTR amyloidosis.

Learn more about the novel and emerging ATTR amyloidosis pipeline therapies @ https://www.delveinsight.com/report-store/attr-amyloidosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

ATTR 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
• Parenteral
• Topical

By Molecule Type
• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Scope of the ATTR Amyloidosis Pipeline Report
• Coverage: Global
• Key ATTR Amyloidosis Companies: Alnylam Pharmaceuticals, Novo Nordisk, Sirnaomics, Arbor Bio, and others.
• Key ATTR Amyloidosis Pipeline Therapies: Vutrisiran, NN-6019, ALN-TTRsc04, STP152G, ABO-102, and others.

Dive deep into rich insights for drugs used for ATTR amyloidosis treatment; visit @ https://www.delveinsight.com/report-store/attr-amyloidosis-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

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

Contact Us:
Jatin Vimal
jvimal@delveinsight.com
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Healthcare Consulting
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This release was published on openPR.