attr amyloidosis pipeline insight

DelveInsight’s, “ATTR Amyloidosis - Pipeline Insight, 2025” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in ATTR Amyloidosis pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

Geography Covered

• Global coverage

 

ATTR Amyloidosis: Understanding

ATTR Amyloidosis: Overview

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a form of systemic amyloidosis characterized by the deposition of misfolded transthyretin (TTR) protein in the heart muscle, leading to cardiac dysfunction. While cardiac amyloidosis can also result from the deposition of immunoglobulin light-chain (AL) aggregates, transthyretin amyloidosis is the primary cause of cardiac involvement. ATTR is a systemic disease, often affecting other organs, though isolated cardiac involvement can occur. The diagnosis of ATTR-CM has historically been delayed due to limited diagnostic tools, but advances in bone scintigraphy and new therapeutic options have improved both diagnosis and treatment. As detection methods improve, ATTR-CM is being recognized more frequently, particularly in patients with heart failure with preserved ejection fraction, a population previously thought to have a lower prevalence of the condition.

 

Transthyretin (TTR), previously known as prealbumin, is a protein primarily produced by the liver that functions as a carrier for retinol (vitamin A) and thyroxine. It normally circulates as a stable tetramer, but structural changes can cause it to misfold and aggregate, leading to deposits in tissues such as the myocardium and peripheral nerves. Transthyretin amyloidosis (ATTR) can be hereditary (hATTR) due to mutations in the TTR gene on chromosome 18, or age-related (wild-type ATTR, wATTR), where the normal TTR becomes prone to misfolding with aging. Both forms can lead to ATTR cardiomyopathy (ATTR-CM), with wATTR-CM now recognized as more common than hATTR-CM.

 

Misfolded transthyretin (TTR) protein forms insoluble fibers that deposit in the heart, leading to myocardial stiffness, fibrosis, and hypertrophy. This deposition compromises ventricular compliance, causing diastolic dysfunction and eventually contributing to systolic dysfunction in advanced stages. Increased left ventricular end-diastolic and left atrial pressures can lead to atrial arrhythmias. Although ventricular arrhythmias can occur in ATTR cardiomyopathy (ATTR-CM), they are less common than in AL cardiomyopathy, likely due to the different cardiotoxic effects. In addition to the heart, TTR deposition also affects the autonomic and peripheral nervous systems, with hereditary ATTR (hATTR) more frequently involving the nerves, while wild-type ATTR (wATTR) typically presents with more prominent cardiac involvement. 

 

The management of cardiac involvement in ATTR-CM focuses on maintaining euvolemia with dietary sodium restriction and diuretics, particularly loop diuretics, as patients often struggle with volume management due to small LV cavity and autonomic dysfunction. Beta-blockers and ACE inhibitors are generally poorly tolerated. In advanced cases, midodrine and compression stockings may be used, and verapamil should be avoided. Arrhythmias are managed with rhythm control, and patients with atrial fibrillation are typically on lifelong anticoagulation. Permanent pacemaker support may be required for conduction system disease, and bi-ventricular pacing is considered for significant dys-synchrony. Specific therapies targeting transthyretin, such as tafamidis and patisiran, help slow disease progression by stabilizing the TTR protein. Liver transplantation is an option for hereditary forms but is less common due to the availability of targeted therapies.

 

"ATTR Amyloidosis- Pipeline Insight, 2025" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the ATTR Amyloidosis pipeline landscape is provided which includes the disease overview and ATTR Amyloidosis treatment guidelines. The assessment part of the report embraces, in depth ATTR Amyloidosis commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, ATTR Amyloidosis collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

 

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence ATTR Amyloidosis R&D. The therapies under development are focused on novel approaches to treat/improve ATTR Amyloidosis.

 

ATTR Amyloidosis Emerging Drugs Chapters

This segment of the ATTR Amyloidosis report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

 

ATTR Amyloidosis Emerging Drugs

• NTLA-2001: Intellia Therapeutics

NTLA-2001 is based on Nobel Prize-winning CRISPR/Cas9 technology, and has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. NTLA-2001 is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein. NTLA-2001 is the first investigational CRISPR therapy to be administered systemically to edit genes inside the human body. Interim Clinical studies showed the administration of NTLA-2001 led to consistent, deep and long-lasting TTR reduction. Intellia leads development and commercialization of NTLA-2001 as part of a multi-target discovery, development and commercialization collaboration with Regeneron. The US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM). Currently, the drug is in Phase III stage of its development for the treatment of ATTR Amyloidosis.

 

• YOLT-201: YolTech Therapeutics Co., Ltd

YOLT-201 Injection utilizes several lipid components including ionizable lipids as primary excipients to encapsulate mRNA and sgRNA raw materials, forming lipid nanoparticles (LNP). Upon intravenous injection into the body, plasma ApoE protein binds to the surface of LNP particles. Liver cells expressing the LDLR receptor recognize ApoE protein and engulf the LNP through endocytosis, forming endosomes. The decrease in pH within endosomes promotes electrostatic interactions between ionizable lipids and endosomal membranes, leading to membrane disruption and the release of mRNA and sgRNA. mRNA, in the cytoplasm, binds to ribosomes, translating the editor protein. The editor protein, in combination with sgRNA, enters the cell nucleus. sgRNA specifically locates the editor to the TTR gene sequence, and the editor protein modifies the target TTR gene, preventing its normal transcription into mRNA. This process stops the production of the TTR protein, achieving the goal of a one-time administration for a comprehensive cure of ATTR diseases. Currently, the drug is in Phase I/II stage of its development for the treatment of ATTR Amyloidosis.

Further product details are provided in the report……..

 

ATTR Amyloidosis: Therapeutic Assessment

This segment of the report provides insights about the different ATTR Amyloidosis drugs segregated based on following parameters that define the scope of the report, such as:

 

Major  Players in ATTR Amyloidosis

There are approx. 3+ key companies which are developing the therapies for ATTR Amyloidosis. The companies which have their ATTR Amyloidosis drug candidates in the most advanced stage, i.e. Phase III include, Intellia Therapeutics.

 

Phases

DelveInsight’s report covers around 3+ products under different phases of clinical development like

• 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

 

Route of Administration

ATTR 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
• Intravenous
• Subcutaneous
• Parenteral 
• Topical

 

Molecule Type

Products have been categorized under various Molecule types such as

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

 

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

 

ATTR Amyloidosis: Pipeline Development Activities 

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses ATTR Amyloidosis therapeutic drugs key players involved in developing key drugs. 

 

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging ATTR Amyloidosis drugs.

 

ATTR Amyloidosis Report Insights

• ATTR Amyloidosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

 

ATTR Amyloidosis Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
•  Unmet Needs

 

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing ATTR Amyloidosis drugs?
• How many ATTR Amyloidosis drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of ATTR Amyloidosis?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the ATTR Amyloidosis therapeutics? 
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies? 
• What are the clinical studies going on for ATTR Amyloidosis and their status?
• What are the key designations that have been granted to the emerging drugs?