Hereditary Transthyretin Amyloidosis (hATTR) Pipeline Insight 2025: Gene Silencers, CRISPR Approaches, and TTR Stabilizers Drive a Paradigm Shift in Rare Systemic Amyloidosis Care | DelveInsight

DelveInsight's "Hereditary Transthyretin Amyloidosis (hATTR) - Pipeline Insight, 2025" provides an in-depth evaluation of the emerging treatment landscape for this progressive, multisystem, and life-threatening condition caused by pathogenic mutations in the TTR gene. Manifesting as peripheral neuropathy, cardiomyopathy, or a mixed phenotype, hATTR remains underdiagnosed and undertreated globally despite advances in genetic testing and disease awareness.

The 2025 pipeline underscores transformative momentum in TTR gene-silencing therapies, CRISPR-based gene editing, and next-generation stabilizers. Alnylam Pharmaceuticals' vutrisiran, an RNAi therapeutic, continues expanding its global footprint following FDA approval, with ongoing studies assessing long-term safety and efficacy across diverse patient phenotypes. Ionis Pharmaceuticals' eplontersen, a ligand-conjugated antisense oligonucleotide co-developed with AstraZeneca, has shown promise in reducing serum TTR levels and improving neuropathy scores in Phase III trials.

In parallel, NTLA-2001 from Intellia Therapeutics and Regeneron represents the first in vivo CRISPR-based candidate for hATTR, aiming for a one-time intravenous administration that permanently knocks out mutant TTR gene expression. Preliminary human data have demonstrated durable TTR reduction and favorable safety, potentially marking a new era of curative potential.

TTR stabilizers like acoramidis (BridgeBio Pharma) are being evaluated in late-stage trials to improve cardiac outcomes, particularly in hATTR-CM patients. Meanwhile, novel oral agents with improved bioavailability and blood-brain barrier penetration are under development to address CNS involvement, a growing concern in long-term hATTR survivors.

Regulatory agencies such as the FDA and EMA are supporting fast-track, breakthrough therapy, and orphan designations, recognizing the urgent unmet needs of hATTR patients. Companies are also adopting decentralized trial designs, wearable-based monitoring, and real-world data integration to optimize evidence generation.

As precision medicine reshapes rare disease drug development, hATTR stands out as a blueprint for genetically informed, multi-modality treatment approaches that not only prolong survival but also enhance functional independence and quality of life.

Interested in learning more about the current treatment landscape and the key drivers shaping the hereditary transthyretin amyloidosis pipeline? Click here: https://www.delveinsight.com/report-store/hereditary-transthyretin-amyloidosis-hattr-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Key Takeaways from the Hereditary Transthyretin Amyloidosis Pipeline Report
• DelveInsight's hereditary transthyretin amyloidosis pipeline analysis depicts a strong space with 8+ active players working to develop 8+ pipeline drugs for hereditary transthyretin amyloidosis treatment.
• The leading hereditary transthyretin amyloidosis companies include Alnylam Pharmaceuticals, Ionis Pharmaceuticals, Eidos Therapeutics, Intellia Therapeutics, Corino Therapeutics, Novo Nordisk, and others are evaluating their lead assets to improve the hereditary transthyretin amyloidosis treatment landscape.
• Key hereditary transthyretin amyloidosis pipeline therapies in various stages of development include Vutrisiran, Acoramidis, AKCEA-TTR-LRx, NTLA-2001, Tolcapone, PRX 004, and others.
• 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 FDA approval of the supplemental New Drug Application (sNDA) for its RNAi therapeutic, AMVUTTRA® (vutrisiran), for treating the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, hospitalizations, and urgent heart failure visits.

Request a sample and discover the recent breakthroughs happening in the hereditary transthyretin amyloidosis pipeline landscape at https://www.delveinsight.com/report-store/hereditary-transthyretin-amyloidosis-hattr-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Hereditary Transthyretin Amyloidosis Overview
Hereditary transthyretin amyloidosis (hATTR) is a rare, progressive genetic disorder caused by mutations in the transthyretin (TTR) gene. These mutations lead to the misfolding of the transthyretin protein, which then forms amyloid fibrils that deposit in various tissues and organs, including peripheral nerves, the heart, and gastrointestinal tract. This accumulation disrupts normal function and causes symptoms such as peripheral neuropathy (numbness, tingling, pain), autonomic dysfunction (digestive issues, blood pressure abnormalities), and cardiomyopathy (heart failure and arrhythmias).

Diagnosis is based on genetic testing, tissue biopsy showing amyloid deposits, and clinical evaluation. Treatment options have advanced significantly and include TTR stabilizers (like tafamidis and acoramidis) that prevent protein misfolding, gene-silencing therapies (such as vutrisiran and AKCEA-TTR-LRx) that reduce TTR production, and emerging gene-editing approaches like NTLA-2001. Early diagnosis and management are crucial to slow disease progression and improve quality of life.

Find out more about hereditary transthyretin amyloidosis medication at https://www.delveinsight.com/report-store/hereditary-transthyretin-amyloidosis-hattr-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Hereditary Transthyretin Amyloidosis Treatment Analysis: Drug Profile
Vutrisiran: Alnylam Pharmaceuticals
Vutrisiran is an investigational RNA interference (RNAi) therapy administered subcutaneously, developed to treat both hereditary (hATTR) and wild-type (wtATTR) transthyretin amyloidosis. It works by targeting and silencing the messenger RNA responsible for producing both wild-type and mutant transthyretin (TTR) proteins, thereby preventing their formation. With quarterly dosing, vutrisiran aims to reduce TTR amyloid deposits in tissues, potentially promoting their clearance and restoring tissue function. The drug leverages Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc conjugate delivery platform, which enhances potency and metabolic stability, allowing for infrequent injections. The Phase 3 HELIOS-B trial is a randomized, double-blind, placebo-controlled study involving around 600 adults with ATTR amyloidosis cardiomyopathy (both hATTR and wtATTR). The FDA has accepted Alnylam's New Drug Application (NDA) for vutrisiran to treat the polyneuropathy of hATTR amyloidosis in adults, with a PDUFA action date set for April 14, 2022, and no advisory committee meeting planned at this time.

AKCEA-TTR-LRx: Ionis Pharmaceuticals
AKCEA-TTR-LRx is an antisense oligonucleotide therapy developed using Ionis Pharmaceuticals' proprietary Ligand Conjugated Antisense (LICA) platform, designed to inhibit transthyretin (TTR) production. Discovered by Ionis and co-developed with Akcea Therapeutics, this drug showed up to a 94% reduction in TTR levels at the highest dose during a Phase 1 clinical trial. The ongoing CARDIO-TTRansform study is a global, double-blind, randomized, placebo-controlled Phase III trial evaluating the cardiovascular outcomes of AKCEA-TTR-LRx in patients with both wild-type and hereditary ATTR cardiomyopathy who are receiving standard of care treatments.

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

Hereditary Transthyretin 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
• Parenteral
• Intravenous
• Subcutaneous
• Topical

By Molecule Type
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Scope of the Hereditary Transthyretin Amyloidosis Pipeline Report
• Coverage: Global
• Key Hereditary Transthyretin Amyloidosis Companies: Alnylam Pharmaceuticals, Ionis Pharmaceuticals, Eidos Therapeutics, Intellia Therapeutics, Corino Therapeutics, Novo Nordisk, and others.
• Key Hereditary Transthyretin Amyloidosis Pipeline Therapies: Vutrisiran, Acoramidis, AKCEA-TTR-LRx, NTLA-2001, Tolcapone, PRX 004, and others.

To dive deep into rich insights for drugs used for hereditary transthyretin amyloidosis treatment, visit: https://www.delveinsight.com/report-store/hereditary-transthyretin-amyloidosis-hattr-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Table of Contents
1. Introduction
2. Executive Summary
3. Hereditary Transthyretin Amyloidosis Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Hereditary Transthyretin Amyloidosis Pipeline Therapeutics
6. Hereditary Transthyretin Amyloidosis Pipeline: Late-Stage Products (Phase III)
7. Hereditary Transthyretin Amyloidosis Pipeline: Mid-Stage Products (Phase II)
8. Hereditary Transthyretin 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
+14699457679
Healthcare Consulting
https://www.delveinsight.com/consulting-services

About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform, PharmDelve.

This release was published on openPR.