Cardiac Amyloidosis - Pipeline Insight, 2024

This Cardiac Amyloidosis- Pipeline Insight, 2024,” report provides comprehensive insights about 20+ companies and 20+ pipeline drugs in Cardiac 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

Cardiac Amyloidosis Understanding

Cardiac Amyloidosis: Overview

Cardiac amyloidosis is one of the leading causes of restrictive cardiomyopathy. It typically presents with rapidly progressive diastolic dysfunction in a non-dilated ventricle. It is one of the under-diagnosed disease entities. The diagnosis of cardiac amyloidosis requires a high degree of suspicion, with cardiovascular imaging being pivotal in reaching the diagnosis.

The deposition of amyloid can lead to heart diseases in multiple ways. Direct interstitial infiltration leads to increased ventricular wall thickness, ventricular stiffness, and consequent ventricular diastolic dysfunction. In AL amyloidosis, amyloid can get deposited in arterioles leading to angina or, rarely, myocardial infarction. Amyloid infiltration in atria can lead to atrial interstitial changes, establishing a structural substrate for atrial fibrillation. Even in the absence of atrial fibrillation, atrial amyloidosis increases the risk of atrial thrombosis and thromboembolism. Light chains can also cause direct injury to the myocardial cells via reactive oxygen species.

Cardiac Amyloidosis - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Cardiac Amyloidosis pipeline landscape is provided which includes the disease overview and Cardiac Amyloidosis treatment guidelines. The assessment part of the report embraces, in depth Cardiac 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, Cardiac 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 R&D Cardiac Amyloidosis. The therapies under development are focused on novel approaches to treat/improve Cardiac Amyloidosis.

Cardiac Amyloidosis Emerging Drugs

Acoramidis: Eidos Therapeutics
Eidos Therapeutics is developing a small molecule named Acoramidis (AG10), which was designed by researchers at Stanford University to potently stabilize TTR by mimicking the structure of the T119M TTR variant. T119M is known to protect carriers from developing ATTR due to the formation of unique bonds at the center of the molecule that stabilize the protein approximately 40-fold beyond the native structure. In pre-clinical studies, acoramidis (AG10) was shown to bind TTR tetramers at the same central locations at which the T119M variant forms stabilizing bonds. This unique binding mode is thought to underlie the potent stabilization of TTR by acoramidis (AG10).

NTLA-2001: Intellia Therapeutics
NTLA-2001 is the first CRISPR/Cas9-based therapy candidate to be administered systemically for precision editing of a gene in humans. It is designed to inactivate the TTR gene in liver cells to reduce the production of misfolded TTR protein, which accumulates in tissues throughout the body and causes the debilitating and often fatal complications of ATTR amyloidosis

Cardiac Amyloidosis: Therapeutic Assessment

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

Major Players in Cardiac Amyloidosis

There are approx. 20+ key companies which are developing the therapies Cardiac Amyloidosis. The companies which have their Cardiac Amyloidosis drug candidates in the most advanced stage, i.e preregistration include Alnylam Pharmaceuticals

Phases

This report covers around 20+ 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

Cardiac 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

Molecule Type

Products have been categorized under various Molecule types such as

• Small molecule
• Cell Therapy
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

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

Cardiac Amyloidosis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Cardiac 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 Cardiac Amyloidosis drugs.

Cardiac Amyloidosis Report Insights

• Cardiac Amyloidosis Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Cardiac 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 Cardiac Amyloidosis drugs?
• How many Cardiac Amyloidosis drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Cardiac Amyloidosis?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Cardiac 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 Cardiac Amyloidosis and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Eidos Therapeutics
• Intellia Therapeutics
• Novo Nordisk
• Alnylam Pharmaceuticals
• Precision Biosciences
• Prothena
• Caelum Biosciences
• Zentalis Pharmaceuticals
• Attralus

Key Products

• Acoramidis
• NTLA 2001
• PRX 004
• Vutrisiran
• ALN TTRsc04
• Birtamimab
• Anselamimab
• ZN d5
• Iodine (124I) evuzamitide

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