DelveInsight’s, “Alpha-1 antitrypsin deficiency (AATD) – Pipeline Insight, 2020,” report provides comprehensive insights about 25+ companies and 25+ pipeline drugs in Alpha-1 antitrypsin deficiency 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: Overview
Alpha-1 antitrypsin deficiency is an inherited disorder that may cause lung disease and liver disease. The signs and symptoms of the condition and the age at which they appear vary among individuals. Alpha1-antitrypsin is a protein made by the liver whose function is to protect the lungs. If these proteins are malformed or deficient, the impact is a predisposition for obstructive pulmonary disease and liver disease. This deficiency may predispose an individual to several illnesses and most commonly manifests as chronic obstructive pulmonary disease (including bronchiectasis) and liver disease (especially cirrhosis and hepatoma), or more rarely, as a skin condition called panniculitis. A deficiency of A1AT allows substances that break down proteins (so-called proteolytic enzymes) to attack various tissues of the body. The attack results in destructive changes in the lungs (emphysema) and may also affect the liver and skin.
People with alpha-1 antitrypsin deficiency usually develop the first signs and symptoms of lung disease between ages 20 and 50. The earliest symptoms are shortness of breath following mild activity, reduced ability to exercise, and wheezing. Other signs and symptoms can include 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). Characteristic features of emphysema include difficulty breathing, a hacking cough, and a barrel-shaped chest. Smoking or exposure to tobacco smoke accelerates the appearance of emphysema symptoms and damage to the lungs.
The diagnosis of A1AD is based on a low concentration of A1AT blood plasma in combination with a high-risk phenotype (demonstrated by isoelectric focusing) or genotype (by specific allele analysis [usually for the Z and S alleles and sometimes for the F and I alleles on commercial tests]). In some instances, further testing to sequence the A1AT gene is needed to establish a firm diagnosis (i.e., mapping all the chemical elements [called nucleotides] that make up the A1AT gene).
This segment of the Alpha-1 antitrypsin deficiency 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.
DCR-A1AT is a subcutaneously administered ribonucleic acid interference (RNAi) therapeutic that is being investigated for the treatment of liver disease in patients with alpha-1 antitrypsin (A1AT) deficiency. DCR-A1AT, which incorporates Dicerna’s proprietary GalXC technology, is designed to target SERPINA1, reducing production of abnormal A1AT in the liver. A clinical trial program investigating the safety and efficacy of DCR-A1AT is currently underway. The U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Dicerna’s DCR-A1AT for the treatment of alpha-1 antitrypsin (A1AT) deficiency.
ARO-AAT is being developed to treat the liver disease associated with alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder that severely damages the liver and lungs of affected individuals. ARO-AAT is designed to knock down the hepatic production of the mutant alpha-1 antitrypsin (Z-AAT) protein, the cause of progressive liver disease in AATD patients. Reducing production of the inflammatory Z-AAT protein is expected to halt the progression of liver disease and potentially allow it to regenerate and repair.
Further product details are provided in the report……..
This segment of the report provides insights about the different Alpha-1 antitrypsin deficiency drugs segregated based on following parameters that define the scope of the report, such as:
There are approx. 25+ key companies which are developing the therapies for Alpha-1 antitrypsin deficiency. The companies which have their Alpha-1 antitrypsin deficiency drug candidates in the mid to advanced stage, i.e. phase III and Phase II include, Kamada, Grifols Therapeutics, CSL Behring, Inhibrx, Alnylam Pharmaceuticals etc.
DelveInsight’s report covers around 25+ products under different phases of clinical development like
Alpha-1 antitrypsin deficiency 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.
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Alpha-1 antitrypsin deficiency therapeutic drugs key players involved in developing key drugs.
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Alpha-1 antitrypsin deficiency drugs.
Current Treatment Scenario and Emerging Therapies:
Introduction
Executive Summary
Alpha-1 antitrypsin deficiency: 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 – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
• Alpha-1 antitrypsin deficiency companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Alpha-1 antitrypsin deficiency Collaboration Deals
• Company-Company Collaborations (Licensing / Partnering) Analysis
• Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
• Comparative Analysis
Inhaled AAT: Kamada, Ltd.
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Mid Stage Products (Phase II)
• Comparative Analysis
Alvelestat: Mereo BioPharma
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Mid Stage Products (Phase I/II)
DCR A1AT: Dicerna Pharmaceuticals
• Product Description
• Research and Development
• Product Development Activities
ALN-AAT02: Alnylam Pharmaceuticals
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Pre-clinical and Discovery Stage Products
• Comparative Analysis
OctaAlpha1: Octapharma
• Product Description
• Research and Development
• Product Development Activities
Drug profiles in the detailed report…..
Inactive Products
• Comparative Analysis
Alpha-1 antitrypsin deficiency Key Companies
Alpha-1 antitrypsin deficiency Key Products
Alpha-1 antitrypsin deficiency- Unmet Needs
Alpha-1 antitrypsin deficiency- Market Drivers and Barriers
Alpha-1 antitrypsin deficiency- Future Perspectives and Conclusion
Alpha-1 antitrypsin deficiency Analyst Views
Alpha-1 antitrypsin deficiency Key Companies
Appendix
List of Tables
Table 1 Total Products for Alpha-1 antitrypsin deficiency
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
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
• Octapharma
• Dicerna Pharmaceuticals
• Alnylam Pharmaceuticals
• Arrowhead Pharmaceuticals
• Apollo Therapeutics
• Vertex Pharmaceuticals
• AstraZeneca
• Kamada, Ltd.
