Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease- Pipeline Insight, 2025
Description
DelveInsight’s, “Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease- Pipeline Insight, 2025” report provides comprehensive insights about 10+ companies and 15+ 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.
Geography Covered
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease: Overview
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease, is an inherited metabolic disorder in which this protein is absent or defective. The deficiency is caused by mutation in the A1AT protein encoding gene SERPINA1 and is inherited in an autosomal recessive manner. Alpha-1 antitrypsin is a large, 52‐kDa serum glycoprotein and a serine protease inhibitor. AAT is primarily produced in the liver and plays a crucial role in protecting the lungs from inflammation caused by infection and irritants such as tobacco smoke.
Alpha-1 antitrypsin deficiency occurs worldwide, but its prevalence varies by population. This disorder affects about 1 in 1,500 to 3,500 individuals with European ancestry. It is uncommon in people of Asian descent. Many individuals with alpha-1 antitrypsin deficiency are likely undiagnosed, particularly people with a lung condition called chronic obstructive pulmonary disease (COPD). COPD can be caused by alpha-1 antitrypsin deficiency; however, the alpha-1 antitrypsin deficiency is often never diagnosed. Some people with alpha-1 antitrypsin deficiency are misdiagnosed with asthma.
Some people with A1ATD experience no symptoms at all. But in some people it can lead to the development of lung conditions and liver disease. Symptoms of A1ATD Liver disease includes breathlessness during exercise or physical activity, cough that lasts a long time, production of phlegm, wheezing, repeated chest infections, prolonged jaundice, hepatomegaly, cholestasis, muscle wasting and abdominal pain.
In children, A1ATD Liver Disease is the most frequent genetic etiology for pediatric liver disease and transplantation. It is caused by a mutation in chromosome 14, the disease is inherited in an autosomal recessive manner with codominant expression. There are over 100 variants of the alpha-1 antitrypsin (A1AT) gene (SERPINA1), which can be influenced by other genetic and environmental factors, leading to marked variation in the onset and severity of disease. The liver disease associated with A1ATD is a gain-of-toxic function mechanism.
The diagnosis of Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease is confirmed with laboratory analysis. Phenotyping by isoelectric focusing is the gold standard of diagnosis. Although liver biopsy is not required for diagnosis, it may be helpful in difficult cases and/or for prognostication. 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 involves smoking cessation, preventive vaccinations, bronchodilators, supplemental oxygen when indicated, and physical rehabilitation in a program similar to that designed for patients with smoking-related COPD. Augmentation therapy may help slow lung damage. Evolving therapies under research studies for the treatment of A1ATD Liver Disease management includes: Chaperones, Autophagy-Enhancing Agents, Bile Acids as Small Molecule Therapy, Inhibiting Polymerization of ATZ, Gene Therapy and Editing, and Cell Therapy.
""Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease pipeline landscape is provided which includes the disease overview and Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease treatment guidelines. The assessment part of the report embraces, in depth Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease 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, Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
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, II/III 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.
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Emerging Drugs
Further product details are provided in the report……..
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease: Therapeutic Assessment
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:
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
Current Treatment Scenario and Emerging Therapies:
Geography Covered
- Global coverage
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease: Overview
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease, is an inherited metabolic disorder in which this protein is absent or defective. The deficiency is caused by mutation in the A1AT protein encoding gene SERPINA1 and is inherited in an autosomal recessive manner. Alpha-1 antitrypsin is a large, 52‐kDa serum glycoprotein and a serine protease inhibitor. AAT is primarily produced in the liver and plays a crucial role in protecting the lungs from inflammation caused by infection and irritants such as tobacco smoke.
Alpha-1 antitrypsin deficiency occurs worldwide, but its prevalence varies by population. This disorder affects about 1 in 1,500 to 3,500 individuals with European ancestry. It is uncommon in people of Asian descent. Many individuals with alpha-1 antitrypsin deficiency are likely undiagnosed, particularly people with a lung condition called chronic obstructive pulmonary disease (COPD). COPD can be caused by alpha-1 antitrypsin deficiency; however, the alpha-1 antitrypsin deficiency is often never diagnosed. Some people with alpha-1 antitrypsin deficiency are misdiagnosed with asthma.
Some people with A1ATD experience no symptoms at all. But in some people it can lead to the development of lung conditions and liver disease. Symptoms of A1ATD Liver disease includes breathlessness during exercise or physical activity, cough that lasts a long time, production of phlegm, wheezing, repeated chest infections, prolonged jaundice, hepatomegaly, cholestasis, muscle wasting and abdominal pain.
In children, A1ATD Liver Disease is the most frequent genetic etiology for pediatric liver disease and transplantation. It is caused by a mutation in chromosome 14, the disease is inherited in an autosomal recessive manner with codominant expression. There are over 100 variants of the alpha-1 antitrypsin (A1AT) gene (SERPINA1), which can be influenced by other genetic and environmental factors, leading to marked variation in the onset and severity of disease. The liver disease associated with A1ATD is a gain-of-toxic function mechanism.
The diagnosis of Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease is confirmed with laboratory analysis. Phenotyping by isoelectric focusing is the gold standard of diagnosis. Although liver biopsy is not required for diagnosis, it may be helpful in difficult cases and/or for prognostication. 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 involves smoking cessation, preventive vaccinations, bronchodilators, supplemental oxygen when indicated, and physical rehabilitation in a program similar to that designed for patients with smoking-related COPD. Augmentation therapy may help slow lung damage. Evolving therapies under research studies for the treatment of A1ATD Liver Disease management includes: Chaperones, Autophagy-Enhancing Agents, Bile Acids as Small Molecule Therapy, Inhibiting Polymerization of ATZ, Gene Therapy and Editing, and Cell Therapy.
""Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease pipeline landscape is provided which includes the disease overview and Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease treatment guidelines. The assessment part of the report embraces, in depth Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease 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, Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease 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 Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease R&D. The therapies under development are focused on novel approaches to treat/improve Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease.
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, II/III 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.
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease Emerging Drugs
- Fazirsiran: Arrowhead Pharmaceuticals
- Alvelestat: Mereo BioPharma
Further product details are provided in the report……..
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease: Therapeutic Assessment
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:
- Major Players in Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease
- 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
- Intravenous
- Subcutaneous
- Oral
- Intramuscular
- Molecule Type
- Monoclonal antibody
- Small molecule
- Peptide
- Product Type
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 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 Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease drugs?
- How many Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease 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 Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease and their status?
- What are the key designations that have been granted to the emerging drugs?
- Arrowhead Pharmaceuticals
- Kamada Pharmaceuticals
- Novo Nordisk
- Peak Bio Inc
- Wave Life Sciences
- Fazirsiran
- Inhaled AAT
- Belcesiran (DCR-A1AT)
- PHP 303
- WVE-006
Table of Contents
60 Pages
- 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
- Late Stage Products (Phase III)
- Comparative Analysis
- Fazirsiran: Arrowhead Pharmaceuticals
- 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…..
- Early Stage Products (Phase I/II)
- Comparative Analysis
- Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Comparative Analysis
- Drug Name: Company Name
- 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
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