Hereditary Transthyretin Amyloidosis Pipeline Drugs Report 2025: MOA, Emerging Therapies, Clinical Trials, Treatment | DelveInsight

DelveInsight's "Hereditary Transthyretin Amyloidosis Pipeline Insight 2025" report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in the Hereditary Transthyretin Amyloidosis pipeline landscape. It covers the Hereditary Transthyretin Amyloidosis Pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Hereditary Transthyretin Amyloidosis Pipeline Therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Hereditary Transthyretin Amyloidosis Pipeline Report

* On 15 October 2025, Intellia Therapeutics announced a study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.
* On 14 October 2025, Eidos Therapeutics, a BridgeBio company conducted a study is to evaluate the long-term safety and tolerability of acoramidis in patients with an established diagnosis of ATTR-CM and heart failure who are concomitantly treated with currently recommended heart failure therapies. Secondary efficacy and PD objectives will be assessed. Exploratory objectives may also be assessed.
* DelveInsight's Hereditary Transthyretin Amyloidosis Pipeline report depicts a robust space with 8+ active players working to develop 8+ pipeline therapies for Hereditary Transthyretin Amyloidosis treatment.
* The leading Hereditary Transthyretin Amyloidosis Companies such as Alnylam Pharmaceuticals, Ionis Pharmaceuticals, Eidos Therapeutics, Intellia Therapeutics, Corino Therapeutics, Novo Nordisk and others.
* Promising Hereditary Transthyretin Amyloidosis Therapies such as Patisiran, Vutrisiran, Inotersen, Eplontersen, Tafamidis Meglumine, NTLA-2001, Revusiran (ALN-TTRSC) and others.

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The Hereditary Transthyretin Amyloidosis Pipeline Report provides disease overview, pipeline scenario and therapeutic assessment of the key pipeline therapies in this domain. The Hereditary Transthyretin Amyloidosis Pipeline Report also highlights the unmet needs with respect to the Hereditary Transthyretin Amyloidosis.

Hereditary Transthyretin Amyloidosis Overview

Hereditary transthyretin (ATTR) amyloidosis is characterized by a slowly progressive peripheral sensorimotor and/or autonomic neuropathy as well as non-neuropathic changes of cardiomyopathy, nephropathy, vitreous opacities, and CNS amyloidosis. It is a rare inherited condition characterized by abnormal build-up of a protein called amyloid in the body's organs and tissues.

Hereditary Transthyretin Amyloidosis Emerging Drugs Profile

* Vutrisiran: Alnylam Pharmaceuticals

Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hereditary (hATTR) and wild-type (wtATTR) amyloidosis. It is designed to target and silence specific messenger RNA, blocking the production of wild-type and variant transthyretin (TTR) protein before it is made. Quarterly administration of vutrisiran may help to reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, designed for increased potency and high metabolic stability that allows for infrequent subcutaneous injections. HELIOS-B is a randomized, double-blind, placebo-controlled Phase 3 study of ~600 adult patients with ATTR amyloidosis with cardiomyopathy (including both hATTR and wtATTR amyloidosis). The U.S. Food and Drug Administration (FDA) has accepted the Company's New Drug Application (NDA) for vutrisiran, an investigational RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The FDA has set an action date of April 14, 2022 under the Prescription Drug User Fee Act (PDUFA), and the Agency has indicated that they are not currently planning an advisory committee meeting as part of the NDA review.

* AKCEA-TTR-LRx: Ionis Pharmaceuticals

AKCEA-TTR-LRx is an antisense drug developed using Ionis' proprietary LIgand Conjugated Antisense (LICA) technology platform and is designed to inhibit production of TTR. It was discovered by Ionis and is being co-developed by Ionis and Akcea. In a Phase 1 clinical trial, patients treated with AKCEA-TTR-LRx experienced reductions in TTR of up to 94 percent at the highest dose. CARDIO-TTRansform is a global, double-blind, randomized, placebo-controlled Phase III cardiovascular outcome study. It is designed to compare AKCEA-TTR-LRx to placebo in patients with both wild type and hereditary ATTR cardiomyopathy who are on the current available standard of care.

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The Hereditary Transthyretin Amyloidosis Pipeline report provides insights into:-

* The report provides detailed insights about companies that are developing therapies for the treatment of Hereditary Transthyretin Amyloidosis with aggregate therapies developed by each company for the same.
* It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Hereditary Transthyretin Amyloidosis Treatment.
* Hereditary Transthyretin Amyloidosis Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
* Hereditary Transthyretin Amyloidosis Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
* Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Hereditary Transthyretin Amyloidosis market.

Hereditary Transthyretin Amyloidosis Companies

Alnylam Pharmaceuticals, Ionis Pharmaceuticals, Eidos Therapeutics, Intellia Therapeutics, Corino Therapeutics, Novo Nordisk and others.

Hereditary Transthyretin Amyloidosis Pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as,

* Oral
* Parenteral
* Intravenous
* Subcutaneous
* Topical
* Molecule Type

Hereditary Transthyretin Amyloidosis Products have been categorized under various Molecule types such as,

* Monoclonal Antibody
* Peptides
* Polymer
* Small molecule
* Gene therapy
* Product Type

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Scope of the Hereditary Transthyretin Amyloidosis Pipeline Report

* Coverage- Global
* Hereditary Transthyretin Amyloidosis Companies- Alnylam Pharmaceuticals, Ionis Pharmaceuticals, Eidos Therapeutics, Intellia Therapeutics, Corino Therapeutics, Novo Nordisk and others.
* Hereditary Transthyretin Amyloidosis Therapies- Patisiran, Vutrisiran, Inotersen, Eplontersen, Tafamidis Meglumine, NTLA-2001, Revusiran (ALN-TTRSC) and others.
* Hereditary Transthyretin Amyloidosis Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
* Hereditary Transthyretin Amyloidosis Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Contents

* Introduction
* Executive Summary
* Hereditary Transthyretin Amyloidosis (hATTR): Overview
* Pipeline Therapeutics
* Late Stage Products (Pregistration)
* Vutrisiran: Alnylam Pharmaceuticals
* Drug profiles in the detailed report.....
* Mid Stage Products (Phase II)
* Drug Name: Company Name
* Drug profiles in the detailed report.....
* Early stage products (Phase I)
* PRX 004: Prothena
* Drug profiles in the detailed report.....
* Inactive Products
* Hereditary Transthyretin Amyloidosis (hATTR) Key Companies
* Hereditary Transthyretin Amyloidosis (hATTR) Key Products
* Hereditary Transthyretin Amyloidosis (hATTR)- Unmet Needs
* Hereditary Transthyretin Amyloidosis (hATTR)- Market Drivers and Barriers
* Hereditary Transthyretin Amyloidosis (hATTR)- Future Perspectives and Conclusion
* Hereditary Transthyretin Amyloidosis (hATTR) Analyst Views
* Hereditary Transthyretin Amyloidosis (hATTR) Key Companies
* Appendix

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