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Alpha-1 Antitrypsin Deficiency Market to Show Remarkable Growth Trends from 2024 to 2034, DelveInsight Reports | Grifols, Kamada Pharmaceuticals, Takeda, Arrowhead Pharmaceuticals, Mereo BioPharma, AstraZeneca, Sanofi, Inhibrx Biosciences
DelveInsight's "Alpha-1 Antitrypsin Deficiency Market Insights, Epidemiology, and Market Forecast-2034′′ report offers an in-depth understanding of the Alpha-1 Antitrypsin Deficiency, historical and forecasted epidemiology as well as the Alpha-1 Antitrypsin Deficiency market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.To Know in detail about the Alpha-1 Antitrypsin Deficiency market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Alpha-1 Antitrypsin Deficiency Market Forecast
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Some of the key facts of the Alpha-1 Antitrypsin Deficiency Market Report:
• Among the 7MM countries, Alpha-1 Antitrypsin Deficiency market size was valued approximately USD 830 million in 2023 and is anticipated to grow with a significant CAGR during the study period (2020-2034)
• In May 2025, Beam Therapeutics (Nasdaq: BEAM) announced that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-302, a liver-targeted base editing treatment developed for alpha-1 antitrypsin deficiency (AATD)-a genetic condition that leads to serious lung and liver complications and represents a significant unmet medical need.
• In March 2025, Beam Therapeutics Inc. (Nasdaq: BEAM) announced that the U.S. Food and Drug Administration (FDA) has approved the investigational new drug (IND) application for BEAM-302, a potential therapy for alpha-1 antitrypsin deficiency.
• In November 2024, Korro Bio has received approval in Australia to initiate the two-part Phase I/IIa REWRITE trial for its investigational therapy, KRRO-110, targeting alpha-1 antitrypsin deficiency (AATD). The approvals were granted by the Australian Bellberry Human Research Ethics Committee (HREC) and the Therapeutic Goods Administration (TGA).
• In October 2024, Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company advancing RNA-based therapies, has announced encouraging proof-of-mechanism data from its ongoing Phase 1b/2a RestorAATion-2 trial evaluating WVE-006 for alpha-1 antitrypsin deficiency (AATD). WVE-006, a GalNAc-conjugated, subcutaneously administered A-to-I RNA editing oligonucleotide (AIMer), was developed using Wave's advanced oligonucleotide chemistry platform. This innovative therapy is specifically designed to target both lung and liver diseases associated with AATD.
• In April 2024, Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on advancing RNA medicines to improve human health, announced the approval of its first clinical trial application (CTA) for the RestorAATion-2 trial of WVE-006. This first-in-class RNA editing oligonucleotide is being developed for treating alpha-1 antitrypsin deficiency (AATD). WVE-006 is a GalNAc-conjugated, subcutaneously administered therapy that does not utilize a lipid nanoparticle (LNP) delivery system.
• In April 2024, Enlivex Therapeutics announced promising topline safety and efficacy results from its Phase II clinical trial of Allocetra, an off-the-shelf cell therapy, for the treatment of sepsis. The placebo-controlled, randomized trial aims to identify the optimal dose of Allocetra when administered in combination with the standard care for sepsis caused by various infections.
• Among the 7 major markets (7MM), the United States held the highest market share for Alpha-1 Antitrypsin Deficiency, reaching approximately USD 700 million in 2023.
• In 2023, the United States represented the largest share of the Alpha-1 Antitrypsin Deficiency market among the 7MM, contributing around 84% of the total market size.
• In 2023, the total number of prevalent Alpha-1 Antitrypsin Deficiency cases across the 7MM was approximately 224,750. The prevalence in Japan was notably lower compared to Europe and the United States.
• By 2034, PROLASTIN/PROLASTIN-C/LYNSPAD is anticipated to generate the highest revenue among all emerging therapies in the 7MM.
• In 2020, the United States represented around 59% of the total prevalent cases of Alpha-1 Antitrypsin Deficiency across the 7MM, while the EU4 and the UK accounted for 40%, and Japan contributed approximately 1%.
• In 2023, the diagnosed prevalent cases of Alpha-1 Antitrypsin Deficiency in the United States are estimated to be around 10,700, with the number projected to rise by 2034.
• According to estimates, in 2020, lung-related comorbidities were the most prevalent among Alpha-1 Antitrypsin Deficiency cases in the US, affecting approximately 77% of patients. Other comorbid conditions were observed in about 15% of cases, while liver diseases were present in around 8%.
• Key Alpha-1 Antitrypsin Deficiency Companies: Grifols, Kamada Pharmaceuticals, Takeda, Arrowhead Pharmaceuticals, Mereo BioPharma, AstraZeneca, Sanofi, Inhibrx Biosciences, Wave Life Sciences, Beam Therapeutics, Intellia Therapeutics, Krystal Biotech, BioMari, AlveoGene, Korro Bio, and others
• Key Alpha-1 Antitrypsin Deficiency Therapies: PROLASTIN-C LIQUID/LYNSPAD, GLASSIA, Inhaled Alpha 1-Antitrypsin (AAT), Fazirsiran (ARO-AAT/TAK-999), Inhaled AAT, Alvelestat (MPH-966), SAR447537 (INBRX-101), WVE-006, BEAM-302, NTLA-3001, KB408, BMN 349, AVG-001, KRRO-110, and others
• The Alpha-1 Antitrypsin Deficiency market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Alpha-1 Antitrypsin Deficiency pipeline products will significantly revolutionize the Alpha-1 Antitrypsin Deficiency market dynamics.
Alpha-1 Antitrypsin Deficiency Overview
Alpha-1 Antitrypsin Deficiency (AATD) is a genetic disorder that affects the liver and lungs. It occurs when there is a deficiency in the production of alpha-1 antitrypsin (AAT) protein, which is mainly produced by the liver and helps protect the lungs from damage caused by inflammation. Without enough AAT, the walls of the lungs can be damaged by enzymes released from white blood cells, leading to conditions such as emphysema and chronic obstructive pulmonary disease (COPD).
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Alpha-1 Antitrypsin Deficiency Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.
Alpha-1 Antitrypsin Deficiency Epidemiology Segmentation:
The Alpha-1 Antitrypsin Deficiency market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:
• Total Prevalence of Alpha-1 Antitrypsin Deficiency
• Prevalent Cases of Alpha-1 Antitrypsin Deficiency by severity
• Gender-specific Prevalence of Alpha-1 Antitrypsin Deficiency
• Diagnosed Cases of Episodic and Chronic Alpha-1 Antitrypsin Deficiency
Download the report to understand which factors are driving Alpha-1 Antitrypsin Deficiency epidemiology trends @ Alpha-1 Antitrypsin Deficiency Epidemiology Forecast
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Alpha-1 Antitrypsin Deficiency Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Alpha-1 Antitrypsin Deficiency market or expected to get launched during the study period. The analysis covers Alpha-1 Antitrypsin Deficiency market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Alpha-1 Antitrypsin Deficiency Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
Alpha-1 Antitrypsin Deficiency Therapies and Key Companies
• PROLASTIN-C LIQUID/LYNSPAD: Grifols
• GLASSIA: Kamada Pharmaceuticals/Takeda
• Inhaled Alpha 1-Antitrypsin (AAT): Kamada Pharmaceuticals
• Fazirsiran (ARO-AAT/TAK-999): Arrowhead Pharmaceuticals and Takeda
• Inhaled AAT: Kamada Pharmaceuticals
• Alvelestat (MPH-966): Mereo BioPharma/AstraZeneca
• SAR447537 (INBRX-101): Sanofi/Inhibrx Biosciences
• WVE-006: Wave Life Sciences
• BEAM-302: Beam Therapeutics
• NTLA-3001: Intellia Therapeutics
• KB408: Krystal Biotech
• BMN 349: BioMari
• AVG-001: AlveoGene
• KRRO-110: Korro Bio
Discover more about therapies set to grab major Alpha-1 Antitrypsin Deficiency market share @ Alpha-1 Antitrypsin Deficiency Treatment Landscape
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Alpha-1 Antitrypsin Deficiency Market Strengths
• Ongoing research and an increase in disease understanding have led to the identification of therapies with effective and convenient routes of administration, including subcutaneous, inhalation, and oral, with the potential to improve patient's QoL.
• Active patient registries and support organizations provide information, advocacy, and support to alpha-1 antitrypsin deficiency individuals
Alpha-1 Antitrypsin Deficiency Market Opportunities
• Several preclinical and early-phase assets utilizing novel approaches like RNA editing and gene therapy are under investigation, and conducting further research and clinical trials may offer curative therapy.
• No therapies are approved for liver disease associated with alpha-1 antitrypsin deficiency, allowing pharma players to capitalize on this untapped market by grabbing first mover advantage.
Scope of the Alpha-1 Antitrypsin Deficiency Market Report
• Study Period: 2020-2034
• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
• Key Alpha-1 Antitrypsin Deficiency Companies: Grifols, Kamada Pharmaceuticals, Takeda, Arrowhead Pharmaceuticals, Mereo BioPharma, AstraZeneca, Sanofi, Inhibrx Biosciences, Wave Life Sciences, Beam Therapeutics, Intellia Therapeutics, Krystal Biotech, BioMari, AlveoGene, Korro Bio, and others
• Key Alpha-1 Antitrypsin Deficiency Therapies: PROLASTIN-C LIQUID/LYNSPAD, GLASSIA, Inhaled Alpha 1-Antitrypsin (AAT), Fazirsiran (ARO-AAT/TAK-999), Inhaled AAT, Alvelestat (MPH-966), SAR447537 (INBRX-101), WVE-006, BEAM-302, NTLA-3001, KB408, BMN 349, AVG-001, KRRO-110, and others
• Alpha-1 Antitrypsin Deficiency Therapeutic Assessment: Alpha-1 Antitrypsin Deficiency current marketed and Alpha-1 Antitrypsin Deficiency emerging therapies
• Alpha-1 Antitrypsin Deficiency Market Dynamics: Alpha-1 Antitrypsin Deficiency market drivers and Alpha-1 Antitrypsin Deficiency market barriers
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
• Alpha-1 Antitrypsin Deficiency Unmet Needs, KOL's views, Analyst's views, Alpha-1 Antitrypsin Deficiency Market Access and Reimbursement
To know more about Alpha-1 Antitrypsin Deficiency companies working in the treatment market, visit @ Alpha-1 Antitrypsin Deficiency Clinical Trials and Therapeutic Assessment
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Table of Contents
1. Alpha-1 Antitrypsin Deficiency Market Report Introduction
2. Executive Summary for Alpha-1 Antitrypsin Deficiency
3. SWOT analysis of Alpha-1 Antitrypsin Deficiency
4. Alpha-1 Antitrypsin Deficiency Patient Share (%) Overview at a Glance
5. Alpha-1 Antitrypsin Deficiency Market Overview at a Glance
6. Alpha-1 Antitrypsin Deficiency Disease Background and Overview
7. Alpha-1 Antitrypsin Deficiency Epidemiology and Patient Population
8. Country-Specific Patient Population of Alpha-1 Antitrypsin Deficiency
9. Alpha-1 Antitrypsin Deficiency Current Treatment and Medical Practices
10. Alpha-1 Antitrypsin Deficiency Unmet Needs
11. Alpha-1 Antitrypsin Deficiency Emerging Therapies
12. Alpha-1 Antitrypsin Deficiency Market Outlook
13. Country-Wise Alpha-1 Antitrypsin Deficiency Market Analysis (2020-2034)
14. Alpha-1 Antitrypsin Deficiency Market Access and Reimbursement of Therapies
15. Alpha-1 Antitrypsin Deficiency Market Drivers
16. Alpha-1 Antitrypsin Deficiency Market Barriers
17. Alpha-1 Antitrypsin Deficiency Appendix
18. Alpha-1 Antitrypsin Deficiency Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
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DelveInsight's 'Alpha-1 Antitrypsin Deficiency Epidemiology Forecast to 2034' report delivers an in-depth understanding of the disease, historical and forecasted Alpha-1 Antitrypsin Deficiency epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
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Overview
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Alpha 1-Antitrypsin Deficiency Treatment Market show exponential growth by 2024
Alpha 1-Antitrypsin Deficiency Treatment Market: Overview
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