
Alpha- Antitrypsin Deficiency - Pipeline Insight, 2025
Description
DelveInsight’s, “Alpha-1 antitrypsin deficiency -Pipeline Insight, 2025,” report provides comprehensive insights about 40+ companies and 40+ 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.
Geography Covered
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. People with alpha-1 antitrypsin deficiency usually develop the first signs and symptoms of lung disease between ages 20 and 50. Mutations in the SERPINA1 gene cause alpha-1 antitrypsin deficiency. This gene provides instructions for making a protein called alpha-1 antitrypsin, which protects the body from a powerful enzyme called neutrophil elastase. Without enough functional alpha-1 antitrypsin, neutrophil elastase destroys alveoli and causes lung disease. Abnormal alpha-1 antitrypsin can also accumulate in the liver and damage this organ. Environmental factors, such as exposure to tobacco smoke, chemicals, and dust, likely impact the severity of alpha-1 antitrypsin deficiency.
Symptoms
The earliest symptoms of Alpha-1 antitrypsin deficiency 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.
Diagnosis
Alpha-1 antitrypsin deficiency only can be detected through blood tests. One type of blood test measures the body’s level of Alpha-1 antitrypsin. If the Alpha-1 antitrypsin level is lower than normal, the healthcare provider may order a genotype or a phenotype blood test. These genetic analysis tests look at the amount and type of Alpha-1 antitrypsin being produced and compare it with normal patterns. One test in a lifetime is sufficient for diagnosis.
Treatment
At this time, there is no cure for Alpha-1 antitrypsin deficiency, but there are treatments that can improve symptoms. The healthcare provider may prescribe medications such as bronchodilators or inhaled steroids to help open the airways. The healthcare provider also may recommend pulmonary rehabilitation to improve breathing. People who have severe emphysema from Alpha-1 antitrypsin deficiency may be candidates for a lung transplant. A treatment called Alpha-1 antitrypsin augmentation therapy, which may slow down or stop the destruction of lung tissue, may also be prescribed.
Alpha-1 antitrypsin deficiency Emerging Drugs Chapters
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.
Alpha-1 antitrypsin deficiency Emerging Drugs
Further product details are provided in the report……..
Alpha-1 antitrypsin deficiency: Therapeutic Assessment
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:
Alpha-1 antitrypsin deficiency: 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 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 drugs.
Report Highlights
Current Treatment Scenario and Emerging Therapies:
Geography Covered
- Global coverage
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. People with alpha-1 antitrypsin deficiency usually develop the first signs and symptoms of lung disease between ages 20 and 50. Mutations in the SERPINA1 gene cause alpha-1 antitrypsin deficiency. This gene provides instructions for making a protein called alpha-1 antitrypsin, which protects the body from a powerful enzyme called neutrophil elastase. Without enough functional alpha-1 antitrypsin, neutrophil elastase destroys alveoli and causes lung disease. Abnormal alpha-1 antitrypsin can also accumulate in the liver and damage this organ. Environmental factors, such as exposure to tobacco smoke, chemicals, and dust, likely impact the severity of alpha-1 antitrypsin deficiency.
Symptoms
The earliest symptoms of Alpha-1 antitrypsin deficiency 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.
Diagnosis
Alpha-1 antitrypsin deficiency only can be detected through blood tests. One type of blood test measures the body’s level of Alpha-1 antitrypsin. If the Alpha-1 antitrypsin level is lower than normal, the healthcare provider may order a genotype or a phenotype blood test. These genetic analysis tests look at the amount and type of Alpha-1 antitrypsin being produced and compare it with normal patterns. One test in a lifetime is sufficient for diagnosis.
Treatment
At this time, there is no cure for Alpha-1 antitrypsin deficiency, but there are treatments that can improve symptoms. The healthcare provider may prescribe medications such as bronchodilators or inhaled steroids to help open the airways. The healthcare provider also may recommend pulmonary rehabilitation to improve breathing. People who have severe emphysema from Alpha-1 antitrypsin deficiency may be candidates for a lung transplant. A treatment called Alpha-1 antitrypsin augmentation therapy, which may slow down or stop the destruction of lung tissue, may also be prescribed.
Alpha-1 antitrypsin deficiency Emerging Drugs Chapters
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.
Alpha-1 antitrypsin deficiency Emerging Drugs
- VX-814 and VX-864: Vertex Pharmaceuticals
- ZF874: Z Factor Ltd
- APB-101: Apic Bio
Further product details are provided in the report……..
Alpha-1 antitrypsin deficiency: Therapeutic Assessment
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:
- Major Players in Alpha-1 antitrypsin deficiency
- Phases
- Late-stage products (Phase II and Phase II/III)
- Mid-stage products (Phase II and Phase II/III)
- Early-stage products (Phase I/II and Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
- Infusion
- Intradermal
- Intramuscular
- Intranasal
- Intravaginal
- Oral
- Parenteral
- Subcutaneous
- Topical.
- Molecule Type
- Gene therapies
- Small molecule
- Vaccines
- Polymers
- Peptides
- Monoclonal antibodies
- Product Type
Alpha-1 antitrypsin deficiency: 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 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 drugs.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Alpha-1 antitrypsin deficiency R&D. The therapies under development are focused on novel approaches to treat/improve Alpha-1 antitrypsin deficiency.
- In July 2019, Vertex Pharmaceuticals reported that VX 864 received fast track designation from the US FDA for the treatment of alpha-1 antitrypsin (AAT) deficiency.
- Apic Bio’s Thrive platform both silences a disease-causing gene product and replaces a normal gene product in a single “dual-function” vector.
- Apic was founded as a spinout of University of Massachusetts Medical School by scientific co-founders Robert Brown Jr. and Christian Mueller, who were looking to combine miRNA silencing with gene therapy. UMass was recently awarded a U.S. patent for ""rAAV-Based Compositions and Methods,"" which it licensed to Apic Bio.
- Alpha-1 antitrypsin deficiency 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 drugs?
- How many Alpha-1 antitrypsin deficiency 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?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Alpha-1 antitrypsin deficiency 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 and their status?
- What are the key designations that have been granted to the emerging drugs?
- Intellia Therapeutics
- Apic Bio
- Linton Pharm
- Z Factor Limited
- Vertex Pharmaceuticals
- Grifols
- Kamada
- Octapharma
- PPL Therapeutics
- Precigen Inc
- APB 101
- Research programme: gene editing therapeutics
- LP 503
- ZF 874
- VX 864
- Alpha-1-antitrypsin inhalation
- Alpha-1 antitrypsin
- OctaAlpha1
- Alpha-1-antitrypsin subcutaneous
Table of Contents
60 Pages
- 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
- Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
- Mid Stage Products (Phase II)
- Comparative Analysis
- VX-814: Vertex Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
- VX-864: Vertex Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- Comparative Analysis
- ZF874: Z Factor Ltd
- 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
- 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
Pricing
Currency Rates
Questions or Comments?
Our team has the ability to search within reports to verify it suits your needs. We can also help maximize your budget by finding sections of reports you can purchase.