This comprehensive market research report provides a strategic analysis of the Global Alpha-1 Antitrypsin Deficiency (AATD) Treatment Market. It covers historical data (2020–2024), the base year (2025), and a long-term forecast period (2026–2036).
1. Market Overview
The global Alpha-1 Antitrypsin Deficiency (AATD) treatment market is characterized by a transition from traditional plasma-derived augmentation therapies to innovative gene-editing and RNA-based solutions. As of 2025, the market is valued at approximately USD 1.95 Billion and is projected to reach USD 4.85 Billion by 2036, expanding at a CAGR of 8.6% during the forecast period.
2. Expanded Key Players
The competitive landscape includes established plasma-protein leaders and high-growth biotech firms focusing on curative therapies.
Established Leaders:
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CSL Behring (Zemaira)
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Grifols, S.A. (Prolastin-C)
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Takeda Pharmaceutical Company Ltd. (Glassia and Aralast NP)
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Kamada Ltd. (Inhaled AAT development)
Biotech & Gene Therapy Innovators:
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Vertex Pharmaceuticals: Small molecule correctors.
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Beam Therapeutics: Base editing technologies.
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Alnylam Pharmaceuticals: RNAi therapeutics.
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Arrowhead Pharmaceuticals: Silencing the production of mutant Z-protein.
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Intellia Therapeutics: CRISPR/Cas9-based treatments.
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Inhibrx, Inc. (Recently acquired interest/partnerships for recombinant proteins).
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Dicerna Pharmaceuticals (Novo Nordisk): RNAi-based silencing.
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Mereo BioPharma: Focusing on neutrophil elastase inhibitors.
3. Market Segmentation Analysis
A. By Treatment Type
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Augmentation Therapy: The current gold standard; involves intravenous infusion of purified human AAT protein.
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Bronchodilators & Corticosteroids: Used for symptomatic management of COPD-like symptoms.
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Gene Therapy & RNAi: Emerging segment aimed at "silencing" the liver's production of toxic proteins or "editing" the DNA to produce functional AAT.
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Synthetic Protein Inhibitors: Small molecules designed to prevent lung tissue degradation.
B. By Route of Administration
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Intravenous (IV): Most common; high bio-availability but requires hospital visits.
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Inhalation: High growth potential; allows for direct delivery to lung tissue and home administration.
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Oral: Primarily for new small-molecule pipeline drugs.
C. By Application (Indication)
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Pulmonary Manifestations: Treatment of emphysema and COPD.
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Hepatic Manifestations: Addressing liver cirrhosis caused by protein misfolding (unmet medical need).
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Panniculitis: Treatment of skin-related complications.
4. Regional Analysis
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North America (Market Leader): Dominates with over 45% market share. Driven by high diagnosis rates, favorable reimbursement policies (Medicare/Private), and the presence of major R&D hubs.
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Europe: Second-largest market. High prevalence in Northern/Western Europe (Scandinavia, UK, Germany). Strict regulatory frameworks but strong patient advocacy groups.
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Asia-Pacific: Anticipated to be the fastest-growing region. Growth is fueled by improving diagnostic infrastructure in China and India and increasing awareness among pulmonologists.
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Rest of World: Growth is limited by high treatment costs and lack of specialized screening programs in MEA and Latin America.
5. Porter’s Five Forces Analysis
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Bargaining Power of Suppliers (High): Specialized plasma collection and fractionation require massive infrastructure, giving existing suppliers significant leverage.
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Bargaining Power of Buyers (Moderate): Governments and insurance providers exert pressure on pricing, though the "Orphan Drug" status of treatments provides some protection to manufacturers.
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Threat of New Entrants (Low): High R&D costs, stringent clinical trial requirements, and the complexity of biologics manufacturing act as significant barriers.
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Threat of Substitutes (Low): There are currently no direct substitutes for augmentation therapy that address the underlying deficiency, though gene therapy poses a future threat to the plasma model.
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Competitive Rivalry (High): Intense competition between Takeda, Grifols, and CSL Behring for market share in augmentation, while biotechs race to be the first to market with a functional cure.
6. SWOT Analysis
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Strengths: High patient loyalty to brands; Orphan Drug designations providing market exclusivity; established safety profiles for augmentation.
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Weaknesses: Extremely high cost of treatment (USD 100k+ annually per patient); limited efficacy in reversing existing lung/liver damage.
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Opportunities: Massive undiagnosed population (estimated 90% of sufferers remain undiagnosed); expansion into liver-targeted therapies.
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Threats: Potential for gene therapy to "cure" the patient, eliminating the need for lifelong weekly infusions; volatility in plasma supply chains.
7. Trend Analysis, Drivers & Challenges
Key Drivers:
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Precision Medicine: Increased genetic testing and newborn screening are identifying more patients earlier.
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Biotech Innovation: Advances in CRISPR and RNAi are shifting the focus from "management" to "cures."
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Aging Population: Rising global geriatric population increases the burden of chronic respiratory diseases.
Key Challenges:
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Pricing Pressures: Increased scrutiny on orphan drug pricing by health technology assessment (HTA) bodies.
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Diagnostic Gap: Many patients are misdiagnosed with standard COPD or Asthma for years before being tested for AATD.
8. Value Chain Analysis
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R&D & Clinical Trials: Focus on identifying new protein variants and delivery mechanisms.
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Sourcing: Plasma collection (for augmentation) or synthetic genetic material (for gene therapy).
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Manufacturing: Complex bio-processing and purification.
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Distribution: Cold-chain logistics are essential for biologics.
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End-User: Specialized clinics and infusion centers play a critical role in administration and monitoring.
9. Strategic Recommendations
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For Manufacturers: Invest in inhaled formulations of AAT. This improves patient compliance and reduces the logistical burden of IV infusions.
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For Stakeholders: Collaborate with Diagnostic Tech companies to integrate AATD screening into standard COPD diagnostic kits.
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For Investors: Target companies focused on Liver-specific AATD treatment, as this remains a significantly underserved segment compared to lung treatments.
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For Healthcare Providers: Implement mandatory "one-time" AATD testing for all patients diagnosed with COPD, as recommended by the World Health Organization (WHO).
Table of Contents
Global Alpha- Antitrypsin Deficiency Treatment Market Research Report 2026
1 Industry Overview of Alpha- Antitrypsin Deficiency Treatment
1.1 Definition and Specifications of Alpha- Antitrypsin Deficiency Treatment
1.1.1 Definition of Alpha- Antitrypsin Deficiency Treatment
1.1.2 Specifications of Alpha- Antitrypsin Deficiency Treatment
1.2 Classification of Alpha- Antitrypsin Deficiency Treatment
1.2.1 CT-2009
1.2.2 POL-6014
1.2.3 ARO-AAT
1.2.4 ALNAAT-02
1.2.5 Others
1.3 Applications of Alpha- Antitrypsin Deficiency Treatment
1.3.1 Clinic
1.3.2 Hospital
1.3.3 Others
1.4 Market Segment by Regions
1.4.1 North America
1.4.2 Europe
1.4.3 China
1.4.4 Japan
1.4.5 Southeast Asia
1.4.6 India
2 Manufacturing Cost Structure Analysis of Alpha- Antitrypsin Deficiency Treatment
2.1 Raw Material and Suppliers
2.2 Manufacturing Cost Structure Analysis of Alpha- Antitrypsin Deficiency Treatment
2.3 Manufacturing Process Analysis of Alpha- Antitrypsin Deficiency Treatment
2.4 Industry Chain Structure of Alpha- Antitrypsin Deficiency Treatment
3 Technical Data and Manufacturing Plants Analysis of Alpha- Antitrypsin Deficiency Treatment
3.1 Capacity and Commercial Production Date of Global Alpha- Antitrypsin Deficiency Treatment Major Manufacturers
3.2 Manufacturing Plants Distribution of Global Alpha- Antitrypsin Deficiency Treatment Major Manufacturers
3.3 R&D Status and Technology Source of Global Alpha- Antitrypsin Deficiency Treatment Major Manufacturers
3.4 Raw Materials Sources Analysis of Global Alpha- Antitrypsin Deficiency Treatment Major Manufacturers
4 Global Alpha- Antitrypsin Deficiency Treatment Overall Market Overview
4.1 E Overall Market Analysis
4.2 Capacity Analysis
4.2.1 E Global Alpha- Antitrypsin Deficiency Treatment Capacity and Growth Rate Analysis
4.2.2 Alpha- Antitrypsin Deficiency Treatment Capacity Analysis (Company Segment)
4.3 Sales Analysis
4.3.1 E Global Alpha- Antitrypsin Deficiency Treatment Sales and Growth Rate Analysis
4.3.2 Alpha- Antitrypsin Deficiency Treatment Sales Analysis (Company Segment)
4.4 Sales Price Analysis
4.4.1 E Global Alpha- Antitrypsin Deficiency Treatment Sales Price
4.4.2 Alpha- Antitrypsin Deficiency Treatment Sales Price Analysis (Company Segment)
5 Alpha- Antitrypsin Deficiency Treatment Regional Market Analysis
5.1 North America Alpha- Antitrypsin Deficiency Treatment Market Analysis
5.1.1 North America Alpha- Antitrypsin Deficiency Treatment Market Overview
5.1.2 North America E Alpha- Antitrypsin Deficiency Treatment Local Supply, Import, Export, Local Consumption Analysis
5.1.3 North America E Alpha- Antitrypsin Deficiency Treatment Sales Price Analysis
5.1.4 North America Alpha- Antitrypsin Deficiency Treatment Market Share Analysis
5.2 Europe Alpha- Antitrypsin Deficiency Treatment Market Analysis
5.2.1 Europe Alpha- Antitrypsin Deficiency Treatment Market Overview
5.2.2 Europe E Alpha- Antitrypsin Deficiency Treatment Local Supply, Import, Export, Local Consumption Analysis
5.2.3 Europe E Alpha- Antitrypsin Deficiency Treatment Sales Price Analysis
5.2.4 Europe Alpha- Antitrypsin Deficiency Treatment Market Share Analysis
5.3 China Alpha- Antitrypsin Deficiency Treatment Market Analysis
5.3.1 China Alpha- Antitrypsin Deficiency Treatment Market Overview
5.3.2 China E Alpha- Antitrypsin Deficiency Treatment Local Supply, Import, Export, Local Consumption Analysis
5.3.3 China E Alpha- Antitrypsin Deficiency Treatment Sales Price Analysis
5.3.4 China Alpha- Antitrypsin Deficiency Treatment Market Share Analysis
5.4 Japan Alpha- Antitrypsin Deficiency Treatment Market Analysis
5.4.1 Japan Alpha- Antitrypsin Deficiency Treatment Market Overview
5.4.2 Japan E Alpha- Antitrypsin Deficiency Treatment Local Supply, Import, Export, Local Consumption Analysis
5.4.3 Japan E Alpha- Antitrypsin Deficiency Treatment Sales Price Analysis
5.4.4 Japan Alpha- Antitrypsin Deficiency Treatment Market Share Analysis
5.5 Southeast Asia Alpha- Antitrypsin Deficiency Treatment Market Analysis
5.5.1 Southeast Asia Alpha- Antitrypsin Deficiency Treatment Market Overview
5.5.2 Southeast Asia E Alpha- Antitrypsin Deficiency Treatment Local Supply, Import, Export, Local Consumption Analysis
5.5.3 Southeast Asia E Alpha- Antitrypsin Deficiency Treatment Sales Price Analysis
5.5.4 Southeast Asia Alpha- Antitrypsin Deficiency Treatment Market Share Analysis
5.6 India Alpha- Antitrypsin Deficiency Treatment Market Analysis
5.6.1 India Alpha- Antitrypsin Deficiency Treatment Market Overview
5.6.2 India E Alpha- Antitrypsin Deficiency Treatment Local Supply, Import, Export, Local Consumption Analysis
5.6.3 India E Alpha- Antitrypsin Deficiency Treatment Sales Price Analysis
5.6.4 India Alpha- Antitrypsin Deficiency Treatment Market Share Analysis
6 Global E Alpha- Antitrypsin Deficiency Treatment Segment Market Analysis (by Type)
6.1 Global E Alpha- Antitrypsin Deficiency Treatment Sales by Type
6.2 Different Types of Alpha- Antitrypsin Deficiency Treatment Product Interview Price Analysis
6.3 Different Types of Alpha- Antitrypsin Deficiency Treatment Product Driving Factors Analysis
6.3.1 CT-2009 Growth Driving Factor Analysis
6.3.2 POL-6014 Growth Driving Factor Analysis
6.3.3 ARO-AAT Growth Driving Factor Analysis
6.3.4 ALNAAT-02 Growth Driving Factor Analysis
6.3.5 Others Growth Driving Factor Analysis
7 Global E Alpha- Antitrypsin Deficiency Treatment Segment Market Analysis (by Application)
7.1 Global E Alpha- Antitrypsin Deficiency Treatment Consumption by Application
7.2 Different Application of Alpha- Antitrypsin Deficiency Treatment Product Interview Price Analysis
7.3 Different Application of Alpha- Antitrypsin Deficiency Treatment Product Driving Factors Analysis
7.3.1 Clinic of Alpha- Antitrypsin Deficiency Treatment Growth Driving Factor Analysis
7.3.2 Hospital of Alpha- Antitrypsin Deficiency Treatment Growth Driving Factor Analysis
7.3.3 Others of Alpha- Antitrypsin Deficiency Treatment Growth Driving Factor Analysis
8 Major Manufacturers Analysis of Alpha- Antitrypsin Deficiency Treatment
8.1 Adverum Biotechnologies, Inc.
8.1.1 Company Profile
8.1.2 Product Picture and Specifications
8.1.2.1 Product A
8.1.2.2 Product B
8.1.3 Adverum Biotechnologies, Inc. Alpha- Antitrypsin Deficiency Treatment Sales, Ex-factory Price, Revenue, Gross Margin Analysis
8.1.4 Adverum Biotechnologies, Inc. Alpha- Antitrypsin Deficiency Treatment Business Region Distribution Analysis
8.2 Alnylam Pharmaceuticals, Inc.
8.2.1 Company Profile
8.2.2 Product Picture and Specifications
8.2.2.1 Product A
8.2.2.2 Product B
8.2.3 Alnylam Pharmaceuticals, Inc. Alpha- Antitrypsin Deficiency Treatment Sales, Ex-factory Price, Revenue, Gross Margin Analysis
8.2.4 Alnylam Pharmaceuticals, Inc. Alpha- Antitrypsin Deficiency Treatment Business Region Distribution Analysis
8.3 Applied Genetic Technologies Corporation
8.3.1 Company Profile
8.3.2 Product Picture and Specifications
8.3.2.1 Product A
8.3.2.2 Product B
8.3.3 Applied Genetic Technologies Corporation Alpha- Antitrypsin Deficiency Treatment Sales, Ex-factory Price, Revenue, Gross Margin Analysis
8.3.4 Applied Genetic Technologies Corporation Alpha- Antitrypsin Deficiency Treatment Business Region Distribution Analysis
8.4 Arrowhead Pharmaceuticals, Inc.
8.4.1 Company Profile
8.4.2 Product Picture and Specifications
8.4.2.1 Product A
8.4.2.2 Product B
8.4.3 Arrowhead Pharmaceuticals, Inc. Alpha- Antitrypsin Deficiency Treatment Sales, Ex-factory Price, Revenue, Gross Margin Analysis
8.4.4 Arrowhead Pharmaceuticals, Inc. Alpha- Antitrypsin Deficiency Treatment Business Region Distribution Analysis
8.5 Carolus Therapeutics, Inc.
8.5.1 Company Profile
8.5.2 Product Picture and Specifications
8.5.2.1 Product A
8.5.2.2 Product B
8.5.3 Carolus Therapeutics, Inc. Alpha- Antitrypsin Deficiency Treatment Sales, Ex-factory Price, Revenue, Gross Margin Analysis
8.5.4 Carolus Therapeutics, Inc. Alpha- Antitrypsin Deficiency Treatment Business Region Distribution Analysis
8.6 Cevec Pharmaceuticals GmbH
8.6.1 Company Profile
8.6.2 Product Picture and Specifications
8.6.2.1 Product A
8.6.2.2 Product B
8.6.3 Cevec Pharmaceuticals GmbH Alpha- Antitrypsin Deficiency Treatment Sales, Ex-factory Price, Revenue, Gross Margin Analysis
8.6.4 Cevec Pharmaceuticals GmbH Alpha- Antitrypsin Deficiency Treatment Business Region Distribution Analysis
8.7 Dicerna Pharmaceuticals, Inc.
8.7.1 Company Profile
8.7.2 Product Picture and Specifications
8.7.2.1 Product A
8.7.2.2 Product B
8.7.3 Dicerna Pharmaceuticals, Inc. Alpha- Antitrypsin Deficiency Treatment Sales, Ex-factory Price, Revenue, Gross Margin Analysis
8.7.4 Dicerna Pharmaceuticals, Inc. Alpha- Antitrypsin Deficiency Treatment Business Region Distribution Analysis
8.8 Digna Biotech, S.L.
8.8.1 Company Profile
8.8.2 Product Picture and Specifications
8.8.2.1 Product A
8.8.2.2 Product B
8.8.3 Digna Biotech, S.L. Alpha- Antitrypsin Deficiency Treatment Sales, Ex-factory Price, Revenue, Gross Margin Analysis
8.8.4 Digna Biotech, S.L. Alpha- Antitrypsin Deficiency Treatment Business Region Distribution Analysis
8.9 Editas Medicine, Inc.
8.9.1 Company Profile
8.9.2 Product Picture and Specifications
8.9.2.1 Product A
8.9.2.2 Product B
8.9.3 Editas Medicine, Inc. Alpha- Antitrypsin Deficiency Treatment Sales, Ex-factory Price, Revenue, Gross Margin Analysis
8.9.4 Editas Medicine, Inc. Alpha- Antitrypsin Deficiency Treatment Business Region Distribution Analysis
8.10 Grifols, S.A.
8.10.1 Company Profile
8.10.2 Product Picture and Specifications
8.10.2.1 Product A
8.10.2.2 Product B
8.10.3 Grifols, S.A. Alpha- Antitrypsin Deficiency Treatment Sales, Ex-factory Price, Revenue, Gross Margin Analysis
8.10.4 Grifols, S.A. Alpha- Antitrypsin Deficiency Treatment Business Region Distribution Analysis
8.11 Inhibrx
8.12 Intellia Therapeutics, Inc.
8.13 International Stem Cell Corporation
8.14 Ionis Pharmaceuticals, Inc.
8.15 Kamada Ltd.
8.16 Polyphor Ltd.
8.17 ProMetic Life Sciences Inc.
8.18 rEVO Biologics, Inc.
8.19 Sangamo BioSciences, Inc.
9 Development Trend of Analysis of Alpha- Antitrypsin Deficiency Treatment Market
9.1 Global Alpha- Antitrypsin Deficiency Treatment Market Trend Analysis
9.1.1 Global 2018-2025 Alpha- Antitrypsin Deficiency Treatment Market Size (Volume and Value) Forecast
9.1.2 Global 2018-2025 Alpha- Antitrypsin Deficiency Treatment Sales Price Forecast
9.2 Alpha- Antitrypsin Deficiency Treatment Regional Market Trend
9.2.1 North America 2018-2025 Alpha- Antitrypsin Deficiency Treatment Consumption Forecast
9.2.2 Europe 2018-2025 Alpha- Antitrypsin Deficiency Treatment Consumption Forecast
9.2.3 China 2018-2025 Alpha- Antitrypsin Deficiency Treatment Consumption Forecast
9.2.4 Japan 2018-2025 Alpha- Antitrypsin Deficiency Treatment Consumption Forecast
9.2.5 Southeast Asia 2018-2025 Alpha- Antitrypsin Deficiency Treatment Consumption Forecast
9.2.6 India 2018-2025 Alpha- Antitrypsin Deficiency Treatment Consumption Forecast
9.3 Alpha- Antitrypsin Deficiency Treatment Market Trend (Product Type)
9.4 Alpha- Antitrypsin Deficiency Treatment Market Trend (Application)
10 Alpha- Antitrypsin Deficiency Treatment Marketing Type Analysis
10.1 Alpha- Antitrypsin Deficiency Treatment Regional Marketing Type Analysis
10.2 Alpha- Antitrypsin Deficiency Treatment International Trade Type Analysis
10.3 Traders or Distributors with Contact Information of Alpha- Antitrypsin Deficiency Treatment by Region
10.4 Alpha- Antitrypsin Deficiency Treatment Supply Chain Analysis
11 Consumers Analysis of Alpha- Antitrypsin Deficiency Treatment
11.1 Consumer 1 Analysis
11.2 Consumer 2 Analysis
11.3 Consumer 3 Analysis
11.4 Consumer 4 Analysis
12 Conclusion of the Global Alpha- Antitrypsin Deficiency Treatment Market Professional Survey Report
Methodology
Analyst Introduction
Data Source
List of Tables and Figures
Figure Picture of Alpha- Antitrypsin Deficiency Treatment
Table Product Specifications of Alpha- Antitrypsin Deficiency Treatment
Table Classification of Alpha- Antitrypsin Deficiency Treatment
Figure Global Production Market Share of Alpha- Antitrypsin Deficiency Treatment by Type in
Figure CT-2009 Picture
Table Major Manufacturers of CT-2009
Figure POL-6014 Picture
Table Major Manufacturers of POL-6014
Figure ARO-AAT Picture
Table Major Manufacturers of ARO-AAT
Figure ALNAAT-02 Picture
Table Major Manufacturers of ALNAAT-02
Figure Others Picture
Table Major Manufacturers of Others
Table Applications of Alpha- Antitrypsin Deficiency Treatment
Figure Global Consumption Volume Market Share of Alpha- Antitrypsin Deficiency Treatment by Application in
Figure Clinic Examples
Table Major Consumers in Clinic
Figure Hospital Examples
Table Major Consumers in Hospital
Figure Others Examples
Table Major Consumers in Others
Figure Market Share of Alpha- Antitrypsin Deficiency Treatment by Regions
Figure North America Alpha- Antitrypsin Deficiency Treatment Market Size (Million USD) (2013-2025)
Figure Europe Alpha- Antitrypsin Deficiency Treatment Market Size (Million USD) (2013-2025)
Figure China Alpha- Antitrypsin Deficiency Treatment Market Size (Million USD) (2013-2025)
Figure Japan Alpha- Antitrypsin Deficiency Treatment Market Size (Million USD) (2013-2025)
Figure Southeast Asia Alpha- Antitrypsin Deficiency Treatment Market Size (Million USD) (2013-2025)
Figure India Alpha- Antitrypsin Deficiency Treatment Market Size (Million USD) (2013-2025)
Table Alpha- Antitrypsin Deficiency Treatment Raw Material and Suppliers
Table Manufacturing Cost Structure Analysis of Alpha- Antitrypsin Deficiency Treatment in
Figure Manufacturing Process Analysis of Alpha- Antitrypsin Deficiency Treatment
Figure Industry Chain Structure of Alpha- Antitrypsin Deficiency Treatment
Table Capacity and Commercial Production Date of Global Alpha- Antitrypsin Deficiency Treatment Major Manufacturers
Table Manufacturing Plants Distribution of Global Alpha- Antitrypsin Deficiency Treatment Major Manufacturers
Table R&D Status and Technology Source of Global Alpha- Antitrypsin Deficiency Treatment Major Manufacturers
Table Raw Materials Sources Analysis of Global Alpha- Antitrypsin Deficiency Treatment Major Manufacturers
Table Global Capacity, Sales , Price, Cost, Sales Revenue (M USD) and Gross Margin of Alpha- Antitrypsin Deficiency Treatment E
Figure Global E Alpha- Antitrypsin Deficiency Treatment Market Size (Volume) and Growth Rate
Figure Global E Alpha- Antitrypsin Deficiency Treatment Market Size (Value) and Growth Rate
Table E Global Alpha- Antitrypsin Deficiency Treatment Capacity and Growth Rate
Table Global Alpha- Antitrypsin Deficiency Treatment Capacity (K Pcs) List (Company Segment)
Table E Global Alpha- Antitrypsin Deficiency Treatment Sales (K Pcs) and Growth Rate
Table Global Alpha- Antitrypsin Deficiency Treatment Sales (K Pcs) List (Company Segment)
Table E Global Alpha- Antitrypsin Deficiency Treatment Sales Price (USD/Pcs)
Table Global Alpha- Antitrypsin Deficiency Treatment Sales Price (USD/Pcs) List (Company Segment)
Figure North America Capacity Overview
Table North America Supply, Import, Export and Consumption (K Pcs) of Alpha- Antitrypsin Deficiency Treatment E
Figure North America E Alpha- Antitrypsin Deficiency Treatment Sales Price (USD/Pcs)
Figure North America Alpha- Antitrypsin Deficiency Treatment Sales Market Share
Figure Europe Capacity Overview
Table Europe Supply, Import, Export and Consumption (K Pcs) of Alpha- Antitrypsin Deficiency Treatment E
Figure Europe E Alpha- Antitrypsin Deficiency Treatment Sales Price (USD/Pcs)
Figure Europe Alpha- Antitrypsin Deficiency Treatment Sales Market Share
Figure China Capacity Overview
Table China Supply, Import, Export and Consumption (K Pcs) of Alpha- Antitrypsin Deficiency Treatment E
Figure China E Alpha- Antitrypsin Deficiency Treatment Sales Price (USD/Pcs)
Figure China Alpha- Antitrypsin Deficiency Treatment Sales Market Share
Figure Japan Capacity Overview
Table Japan Supply, Import, Export and Consumption (K Pcs) of Alpha- Antitrypsin Deficiency Treatment E
Figure Japan E Alpha- Antitrypsin Deficiency Treatment Sales Price (USD/Pcs)
Figure Japan Alpha- Antitrypsin Deficiency Treatment Sales Market Share
Figure Southeast Asia Capacity Overview
Table Southeast Asia Supply, Import, Export and Consumption (K Pcs) of Alpha- Antitrypsin Deficiency Treatment E
Figure Southeast Asia E Alpha- Antitrypsin Deficiency Treatment Sales Price (USD/Pcs)
Figure Southeast Asia Alpha- Antitrypsin Deficiency Treatment Sales Market Share
Figure India Capacity Overview
Table India Supply, Import, Export and Consumption (K Pcs) of Alpha- Antitrypsin Deficiency Treatment E
Figure India E Alpha- Antitrypsin Deficiency Treatment Sales Price (USD/Pcs)
Figure India Alpha- Antitrypsin Deficiency Treatment Sales Market Share
Table Global E Alpha- Antitrypsin Deficiency Treatment Sales (K Pcs) by Type
Table Different Types Alpha- Antitrypsin Deficiency Treatment Product Interview Price
Table Global E Alpha- Antitrypsin Deficiency Treatment Sales (K Pcs) by Application
Table Different Application Alpha- Antitrypsin Deficiency Treatment Product Interview Price
Table Adverum Biotechnologies, Inc. Information List
Table Product Overview
Table Adverum Biotechnologies, Inc. Alpha- Antitrypsin Deficiency Treatment Revenue (Million USD), Sales (K Pcs), Ex-factory Price (USD/Pcs)
Figure Adverum Biotechnologies, Inc. Alpha- Antitrypsin Deficiency Treatment Business Region Distribution
Table Alnylam Pharmaceuticals, Inc. Information List
Table Product Overview
Table Alnylam Pharmaceuticals, Inc. Alpha- Antitrypsin Deficiency Treatment Revenue (Million USD), Sales (K Pcs), Ex-factory Price (USD/Pcs)
Figure Alnylam Pharmaceuticals, Inc. Alpha- Antitrypsin Deficiency Treatment Business Region Distribution
Table Applied Genetic Technologies Corporation Information List
Table Product Overview
Table Applied Genetic Technologies Corporation Alpha- Antitrypsin Deficiency Treatment Revenue (Million USD), Sales (K Pcs), Ex-factory Price (USD/Pcs)
Figure Applied Genetic Technologies Corporation Alpha- Antitrypsin Deficiency Treatment Business Region Distribution
Table Arrowhead Pharmaceuticals, Inc. Information List
Table Product Overview
Table Arrowhead Pharmaceuticals, Inc. Alpha- Antitrypsin Deficiency Treatment Revenue (Million USD), Sales (K Pcs), Ex-factory Price (USD/Pcs)
Figure Arrowhead Pharmaceuticals, Inc. Alpha- Antitrypsin Deficiency Treatment Business Region Distribution
Table Carolus Therapeutics, Inc. Information List
Table Product Overview
Table Carolus Therapeutics, Inc. Alpha- Antitrypsin Deficiency Treatment Revenue (Million USD), Sales (K Pcs), Ex-factory Price (USD/Pcs)
Figure Carolus Therapeutics, Inc. Alpha- Antitrypsin Deficiency Treatment Business Region Distribution
Table Cevec Pharmaceuticals GmbH Information List
Table Product Overview
Table Cevec Pharmaceuticals GmbH Alpha- Antitrypsin Deficiency Treatment Revenue (Million USD), Sales (K Pcs), Ex-factory Price (USD/Pcs)
Figure Cevec Pharmaceuticals GmbH Alpha- Antitrypsin Deficiency Treatment Business Region Distribution
Table Dicerna Pharmaceuticals, Inc. Information List
Table Product Overview
Table Dicerna Pharmaceuticals, Inc. Alpha- Antitrypsin Deficiency Treatment Revenue (Million USD), Sales (K Pcs), Ex-factory Price (USD/Pcs)
Figure Dicerna Pharmaceuticals, Inc. Alpha- Antitrypsin Deficiency Treatment Business Region Distribution
Table Digna Biotech, S.L. Information List
Table Product Overview
Table Digna Biotech, S.L. Alpha- Antitrypsin Deficiency Treatment Revenue (Million USD), Sales (K Pcs), Ex-factory Price (USD/Pcs)
Figure Digna Biotech, S.L. Alpha- Antitrypsin Deficiency Treatment Business Region Distribution
Table Editas Medicine, Inc. Information List
Table Product Overview
Table Editas Medicine, Inc. Alpha- Antitrypsin Deficiency Treatment Revenue (Million USD), Sales (K Pcs), Ex-factory Price (USD/Pcs)
Figure Editas Medicine, Inc. Alpha- Antitrypsin Deficiency Treatment Business Region Distribution
Table Grifols, S.A. Information List
Table Product Overview
Table Grifols, S.A. Alpha- Antitrypsin Deficiency Treatment Revenue (Million USD), Sales (K Pcs), Ex-factory Price (USD/Pcs)
Figure Grifols, S.A. Alpha- Antitrypsin Deficiency Treatment Business Region Distribution
Table Inhibrx Information List
Table Intellia Therapeutics, Inc. Information List
Table International Stem Cell Corporation Information List
Table Ionis Pharmaceuticals, Inc. Information List
Table Kamada Ltd. Information List
Table Polyphor Ltd. Information List
Table ProMetic Life Sciences Inc. Information List
Table rEVO Biologics, Inc. Information List
Table Sangamo BioSciences, Inc. Information List
Figure Global 2018-2025 Alpha- Antitrypsin Deficiency Treatment Market Size (K Pcs) and Growth Rate Forecast
Figure Global 2018-2025 Alpha- Antitrypsin Deficiency Treatment Market Size (Million USD) and Growth Rate Forecast
Figure Global 2018-2025 Alpha- Antitrypsin Deficiency Treatment Sales Price (USD/Pcs) Forecast
Figure North America 2018-2025 Alpha- Antitrypsin Deficiency Treatment Consumption Volume (K Pcs) and Growth Rate Forecast
Figure China 2018-2025 Alpha- Antitrypsin Deficiency Treatment Consumption Volume (K Pcs) and Growth Rate Forecast
Figure Europe 2018-2025 Alpha- Antitrypsin Deficiency Treatment Consumption Volume (K Pcs) and Growth Rate Forecast
Figure Southeast Asia 2018-2025 Alpha- Antitrypsin Deficiency Treatment Consumption Volume (K Pcs) and Growth Rate Forecast
Figure Japan 2018-2025 Alpha- Antitrypsin Deficiency Treatment Consumption Volume (K Pcs) and Growth Rate Forecast
Figure India 2018-2025 Alpha- Antitrypsin Deficiency Treatment Consumption Volume (K Pcs) and Growth Rate Forecast
Table Global Sales Volume (K Pcs) of Alpha- Antitrypsin Deficiency Treatment by Type 2018-2025
Table Global Consumption Volume (K Pcs) of Alpha- Antitrypsin Deficiency Treatment by Application 2018-2025
Table Traders or Distributors with Contact Information of Alpha- Antitrypsin Deficiency Treatment by Region
Expanded Key Players
The competitive landscape includes established plasma-protein leaders and high-growth biotech firms focusing on curative therapies.
Established Leaders:
-
CSL Behring (Zemaira)
-
Grifols, S.A. (Prolastin-C)
-
Takeda Pharmaceutical Company Ltd. (Glassia and Aralast NP)
-
Kamada Ltd. (Inhaled AAT development)
Biotech & Gene Therapy Innovators:
-
Vertex Pharmaceuticals: Small molecule correctors.
-
Beam Therapeutics: Base editing technologies.
-
Alnylam Pharmaceuticals: RNAi therapeutics.
-
Arrowhead Pharmaceuticals: Silencing the production of mutant Z-protein.
-
Intellia Therapeutics: CRISPR/Cas9-based treatments.
-
Inhibrx, Inc. (Recently acquired interest/partnerships for recombinant proteins).
-
Dicerna Pharmaceuticals (Novo Nordisk): RNAi-based silencing.
-
Mereo BioPharma: Focusing on neutrophil elastase inhibitors.
3. Market Segmentation Analysis
A. By Treatment Type
-
Augmentation Therapy: The current gold standard; involves intravenous infusion of purified human AAT protein.
-
Bronchodilators & Corticosteroids: Used for symptomatic management of COPD-like symptoms.
-
Gene Therapy & RNAi: Emerging segment aimed at "silencing" the liver's production of toxic proteins or "editing" the DNA to produce functional AAT.
-
Synthetic Protein Inhibitors: Small molecules designed to prevent lung tissue degradation.
B. By Route of Administration
-
Intravenous (IV): Most common; high bio-availability but requires hospital visits.
-
Inhalation: High growth potential; allows for direct delivery to lung tissue and home administration.
-
Oral: Primarily for new small-molecule pipeline drugs.
C. By Application (Indication)
-
Pulmonary Manifestations: Treatment of emphysema and COPD.
-
Hepatic Manifestations: Addressing liver cirrhosis caused by protein misfolding (unmet medical need).
-
Panniculitis: Treatment of skin-related complications.
4. Regional Analysis
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North America (Market Leader): Dominates with over 45% market share. Driven by high diagnosis rates, favorable reimbursement policies (Medicare/Private), and the presence of major R&D hubs.
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Europe: Second-largest market. High prevalence in Northern/Western Europe (Scandinavia, UK, Germany). Strict regulatory frameworks but strong patient advocacy groups.
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Asia-Pacific: Anticipated to be the fastest-growing region. Growth is fueled by improving diagnostic infrastructure in China and India and increasing awareness among pulmonologists.
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Rest of World: Growth is limited by high treatment costs and lack of specialized screening programs in MEA and Latin America.
