DelveInsight’s, “Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease – Pipeline Insights, 2021,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease 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.
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease: Overview
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease is a common genetic cause of liver disease, with the most severe mutation found in 1:3500 live births. A1AT is a large, 52‐kDa serum glycoprotein abundantly produced by the liver. The deficiency is caused by mutation in the A1AT protein encoding gene SERPINA1 and is inherited in an autosomal recessive manner. Mutations in SERPINA1 leads to the formation of harmful aggregates of mutant A1AT protein in the ER of hepatocytes leading to inflammation, fibrosis, cirrhosis, and increased risk of hepatocellular carcinoma in adults. Risk factors for progression of A1ATD liver disease included male sex, age older than 50 years, viral hepatitis, and diabetes.
The symptoms of Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease depends on the condition and the age at which they appear vary among individuals. The earliest symptoms are shortness of breath following mild activity, reduced ability to exercise, and wheezing, unintentional weight loss, recurring respiratory infections, fatigue, and rapid heartbeat upon standing. Affected individuals often develop emphysema, which is a lung disease caused by damage to the small air sacs in the lungs (alveoli). Symptoms of A1ATD include jaundice, dark urine, itchy skin and bleeding. The liver is gradually scarred and liver function can decline over time. About 10 percent of infants with alpha-1 antitrypsin deficiency develop liver disease, which often causes yellowing of the skin and whites of the eyes (jaundice). In rare cases, people with alpha-1 antitrypsin deficiency develop a skin condition called panniculitis, which is characterized by hardened skin with painful lumps or patches.
Diagnosis of Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease is underreported and underdiagnosed, often with a diagnostic delay of 5 to 10 years.
The diagnosis of A1ATD may require biopsies, blood tests, CAT scans and ultrasound. Following diagnosis, patients found to have A1ATD are largely managed supportively with close monitoring for progression of liver disease like fibrosis or HCC. Phenotyping by isoelectric focusing is the gold standard of diagnosis. Anti-human A1AT antibody can also confirm the presence of ATZ globules. Electron microscopy studies can identify multiple globular inclusions within dilated, congested rough ER cisterns, adjacent to secondary lysosomes.
Treatment options for the A1ATD lung disease are based on the severity of the liver affected and the stage of diagnosis. The treatment regimen may include Chaperones Autophagy-Enhancing agents, Bile acids as small molecule therapy, agents inhibiting ATZ polymerization, Gene therapy and editing, and Cell therapy. Sometimes a liver transplant may be needed if significant liver damage has been caused. It is important to avoid all contact with smoking because lung disease (emphysema) develops over the second decade of life.
This segment of the Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease 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.
DCR-A1AT is a ribonucleic acid interference (RNAi) therapeutic, investigated for the treatment of liver disease in patients with alpha-1 antitrypsin (A1AT) deficiency. DCR-A1AT utilizes Dicerna’s GalXCTM technology, which enables subcutaneous delivery and optimizes the activity of the RNAi pathway so that it operates in the most specific and potent fashion.
APB-101 is a “liver-sparing” gene therapy designed as a one-time treatment for Alpha-1 patients. APB-101, targets the liver via an AAV delivered Dual Function Vector (df-AAV) whereby the Z-AAT protein is silenced and M-AAT protein is augmented. In pre-clinical studies the molecule has demonstrated the ability to reduce levels of the mutant Alpha-1 protein (Z-AAT) and at the same time program liver cells to produce the correct Alpha-1 protein (M-AAT).
Further product details are provided in the report……..
This segment of the report provides insights about the different Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease drugs segregated based on following parameters that define the scope of the report, such as:
There are approx. 5+ key companies which are developing the therapies for Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease. The companies which have their Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease drug candidates in the most advanced stage, i.e. phase II/III include, Arrowhead Pharmaceuticals.
DelveInsight’s report covers around 5+ products under different phases of clinical development like
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
Products have been categorized under various Molecule types such as
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease 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 Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease drugs.
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Report Insights
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Report Assessment
Current Treatment Scenario and Emerging Therapies:
Introduction
Executive Summary
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease: 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
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
• Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Collaboration Deals
• Company-Company Collaborations (Licensing / Partnering) Analysis
• Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase II/II)
• Comparative Analysis
TAK-999: Arrowhead Pharmaceuticals
• Product Description
• Research and Development
• Product Development Activities
Mid Stage Products (Phase II)
• Comparative Analysis
VX-864: Vertex Pharmaceuticals
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Early Stage Products (Phase I/II)
• Comparative Analysis
DCR-A1AT: Dicerna Pharmaceuticals
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Pre-clinical and Discovery Stage Products
• Comparative Analysis
APB-101: Apic Bio
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Inactive Products
• Comparative Analysis
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Key Companies
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Key Products
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease- Unmet Needs
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease- Market Drivers and Barriers
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease- Future Perspectives and Conclusion
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Analyst Views
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Key Companies
Appendix
List of Table
Table 1 Total Products for Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
List of Figures
Figure 1 Total Products for Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
• Alnylam Pharmaceuticals
• Dicerna Pharmaceuticals
• Novartis
• Apic Bio
• Vertex Pharmaceuticals
• Arrowhead Pharmaceuticals
