
Hereditary Transthyretin Amyloidosis (hATTR) - Pipeline Insight, 2024
Description
Hereditary Transthyretin Amyloidosis (hATTR) - Pipeline Insight, 2024
DelveInsight’s, “Hereditary transthyretin amyloidosis – Pipeline Insight, 2024,” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Hereditary transthyretin 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
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. Symptoms start in adulthood and get worse over time. Signs and symptoms depend on where the amyloid protein is building up. Amyloid build-up in the nerves of the peripheral nervous system causes a loss of sensation in the lower limbs, feet, and hands (peripheral neuropathy). Amyloid build-up can also affect the involuntary body functions, such as blood pressure, heart rate, and digestion. Other areas of the body that may be affected are the heart, kidneys, eyes, and gastrointestinal tract. Disease heterogeneity and its rarity make a diagnosis of hATTR amyloidosis challenging. Diagnosis can be confirmed via biopsy of the affected tissue or organ followed by staining with Congo red to confirm the presence of amyloid. Diagnosis can be established less invasively through biopsy of the salivary gland, an endoscopic biopsy of the gastric mucosa, or subcutaneous fat aspiration. Western blot analysis, immunohistochemical staining, laser microdissection, proteomics, and mass spectrometry are subsequently used to characterize amyloid type. Current treatment options for patients with TTR amyloidosis are limited, with only symptomatic treatment and transplantation. For patients diagnosed with TTR-FAP who have mild or moderate disease and confirmed by genetic testing and biopsy, a liver transplant is the current standard of care.
""Hereditary transthyretin amyloidosis - Pipeline Insight, 2024"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Hereditary transthyretin amyloidosis pipeline landscape is provided which includes the disease overview and Hereditary transthyretin amyloidosis treatment guidelines. The assessment part of the report embraces, in depth Hereditary transthyretin 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, Hereditary transthyretin 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 Hereditary transthyretin amyloidosis R&D. The therapies under development are focused on novel approaches to treat/improve Hereditary transthyretin amyloidosis.
This segment of the Hereditary transthyretin amyloidosis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.
Hereditary transthyretin amyloidosis Emerging Drugs
- Vutrisiran: Alnylam Pharmaceuticals
- AKCEA-TTR-LRx: Ionis Pharmaceuticals
Further product details are provided in the report……..
Hereditary transthyretin amyloidosis: Therapeutic Assessment
This segment of the report provides insights about the different Hereditary transthyretin amyloidosis drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Hereditary transthyretin amyloidosis
- Phases
- 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
- Oral
- Parenteral
- Intravenous
- Subcutaneous
- Topical
- Molecule Type
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
Hereditary transthyretin amyloidosis: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Hereditary transthyretin 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 Hereditary transthyretin amyloidosis drugs.
Hereditary transthyretin amyloidosis Report Insights
- Hereditary transthyretin amyloidosis Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Hereditary transthyretin amyloidosis drugs?
- How many Hereditary transthyretin amyloidosis drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Hereditary transthyretin amyloidosis?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Hereditary transthyretin 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 Hereditary transthyretin amyloidosis and their status?
- What are the key designations that have been granted to the emerging drugs?
Table of Contents
40 Pages
- Introduction
- Executive Summary
- Hereditary transthyretin amyloidosis: Overview
- Causes
- Mechanism of Action
- Signs and Symptoms
- Diagnosis
- Disease Management
- Pipeline Therapeutics
- Comparative Analysis
- Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
- Late Stage Products (Pregistration)
- Comparative Analysis
- Vutrisiran: Alnylam Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Comparative Analysis
- Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early stage products (Phase I)
- Comparative Analysis
- PRX 004: Prothena
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Hereditary transthyretin amyloidosis Key Companies
- Hereditary transthyretin amyloidosis Key Products
- Hereditary transthyretin amyloidosis- Unmet Needs
- Hereditary transthyretin amyloidosis- Market Drivers and Barriers
- Hereditary transthyretin amyloidosis- Future Perspectives and Conclusion
- Hereditary transthyretin amyloidosis Analyst Views
- Hereditary transthyretin amyloidosis Key Companies
- Appendix
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