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Alpha-1 Antitrypsin Deficiency Pipeline Drugs Insights Report 2025: Emerging Therapies and Innovation Trends by DelveInsight

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Alpha-1 Antitrypsin Deficiency Pipeline Drugs Insights Report 2025: Emerging Therapies and Innovation Trends by DelveInsight

October 30
18:30 2025
Alpha-1 Antitrypsin Deficiency Pipeline Drugs Insights Report 2025: Emerging Therapies and Innovation Trends by DelveInsight

DelveInsight’s “Alpha-1 Antitrypsin Deficiency Vasculitis Pipeline Insight 2025” report provides comprehensive insights about 12+ companies and 12+ pipeline drugs in the Alpha-1 Antitrypsin Deficiency Vasculitis pipeline landscape. It covers the Alpha-1 Antitrypsin Deficiency Vasculitis Pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Alpha-1 Antitrypsin Deficiency Vasculitis Pipeline Therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Curious about the latest updates in the Alpha-1 Antitrypsin Deficiency Vasculitis Pipeline? Click here to explore the therapies and trials making headlines @ Alpha-1 Antitrypsin Deficiency Vasculitis Pipeline Outlook Report

Key Takeaways from the Alpha-1 Antitrypsin Deficiency Vasculitis Pipeline Report

  • On 24 October 2025, Sanofi announced a Phase 2, Single Arm, Open Label Extension Study, Evaluating the Long-Term Safety and Clinical Efficacy of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency (AATD) Emphysema.
  • On 15 October 2025, Arrowhead Pharmaceuticals conducted a study is to evaluate the the safety and efficacy of the investigational product, fazirsiran (TAK-999, ARO-AAT), administered subcutaneously to patients with alpha-1 antitrypsin deficiency associated liver disease (AATD).
  • DelveInsight’s Alpha-1 Antitrypsin Deficiency Vasculitis Pipeline report depicts a robust space with 12+ active players working to develop 12+ pipeline therapies for Alpha-1 Antitrypsin Deficiency Vasculitis treatment.
  • The leading Alpha-1 Antitrypsin Deficiency Vasculitis Companies such as Octapharma, Dicerna Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Vertex Pharmaceuticals, AstraZeneca, Kamada, Ltd and others.
  • Promising Alpha-1 Antitrypsin Deficiency Vasculitis Therapies such as Alpha-1 15%, Fazirsiran Injection, Aerosolized, Recombinant Alpha 1-Antitrypsin, WVE-006, VX-864, KRRO-110, ADVM-043 and others.

Want to know which companies are leading innovation in Alpha-1 Antitrypsin Deficiency Vasculitis? Dive into the full pipeline insights @ Alpha-1 Antitrypsin Deficiency Vasculitis Clinical Trials Assessment

The Alpha-1 Antitrypsin Deficiency Vasculitis Pipeline Report provides disease overview, pipeline scenario and therapeutic assessment of the key pipeline therapies in this domain. The Alpha-1 Antitrypsin Deficiency Vasculitis Pipeline Report also highlights the unmet needs with respect to the Alpha-1 Antitrypsin Deficiency Vasculitis.

Alpha-1 Antitrypsin Deficiency Vasculitis Overview

Alpha-1-antitrypsin (AAT) is a protein produced in the liver that protects the body’s tissues from being damaged by infection-fighting agents released by its immune system. In alpha-1 antitrypsin deficiency, the body’s normal production of AAT is reduced, resulting in the destruction of sensitive lung tissue. AAT deficiency is inherited. The severity of disease depends in part on the abnormalities present in the genes inherited from each parent. It is estimated that there are between 80,000 to 100,000 individuals affected by AAT deficiency in the United States.

Alpha-1 Antitrypsin Deficiency Vasculitis Emerging Drugs Profile

  • ARO AAT: Arrowhead Pharmaceuticals

ARO AAT is a second generation, N-Acetylgalactosamine (GalNAc) based, subcutaneously administered RNA interference based therapeutic. 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. In June 2019, the US FDA has granted Fast Track designation to ARO-AAT. ARO-AAT is in Phase III clinical studies for the treatment of liver disease associated with alpha-1 antitrypsin deficiency (AATD).

  • ALN-AAT02: Alnylam Pharmaceuticals

ALN AAT02 is a subcutaneously administered RNAi therapeutic that targets alpha-1 antitrypsin (AAT). ALN-AAT02 is based on the Alnylam’s enhanced stabilization chemistry plus (ESC+)-GalNAc-conjugate technology, which enables subsequent subcutaneous dosing with increased selectivity and a wide therapeutic index. The drug is currently in Phase I/II clinical development for the treatment of ZZ Type AAT Deficiency Liver Disease.

If you’re tracking ongoing Alpha-1 Antitrypsin Deficiency Vasculitis Clinical trials, this press release is a must-read. Tap to see the breakthroughs @ Alpha-1 Antitrypsin Deficiency Vasculitis Treatment Drugs

The Alpha-1 Antitrypsin Deficiency Vasculitis Pipeline report provides insights into:-

  • The report provides detailed insights about companies that are developing therapies for the treatment of Alpha-1 Antitrypsin Deficiency Vasculitis with aggregate therapies developed by each company for the same.
  • It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Alpha-1 Antitrypsin Deficiency Vasculitis Treatment.
  • Alpha-1 Antitrypsin Deficiency Vasculitis Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
  • Alpha-1 Antitrypsin Deficiency Vasculitis Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
  • Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Alpha-1 Antitrypsin Deficiency Vasculitis market.

Alpha-1 Antitrypsin Deficiency Vasculitis Companies

Octapharma, Dicerna Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Vertex Pharmaceuticals, AstraZeneca, Kamada, Ltd and others

Alpha-1 Antitrypsin Deficiency Vasculitis Pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as,

  • Intra-articular
  • Intraocular
  • Intrathecal
  • Intravenous
  • Ophthalmic
  • Oral
  • Parenteral
  • Subcutaneous
  • Topical
  • Transdermal

Alpha-1 Antitrypsin Deficiency Vasculitis Products have been categorized under various Molecule types such as,

  • Oligonucleotide
  • Peptide
  • Small molecule

From emerging drug candidates to competitive intelligence, the Alpha-1 Antitrypsin Deficiency Vasculitis Pipeline Report covers it all – check it out now @ Alpha-1 Antitrypsin Deficiency Vasculitis Market Drivers and Barriers, and Future Perspectives

Scope of the Alpha-1 Antitrypsin Deficiency Vasculitis Pipeline Report

  • Coverage- Global
  • Alpha-1 Antitrypsin Deficiency Vasculitis Companies- Octapharma, Dicerna Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Vertex Pharmaceuticals, AstraZeneca, Kamada, Ltd and others.
  • Alpha-1 Antitrypsin Deficiency Vasculitis Therapies- Alpha-1 15%, Fazirsiran Injection, Aerosolized, Recombinant Alpha 1-Antitrypsin, WVE-006, VX-864, KRRO-110, ADVM-043 and others.
  • Alpha-1 Antitrypsin Deficiency Vasculitis Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
  • Alpha-1 Antitrypsin Deficiency Vasculitis Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

Stay ahead in Healthcare Research – discover what’s next for the Alpha-1 Antitrypsin Deficiency Vasculitis Treatment landscape in this detailed analysis @ Alpha-1 Antitrypsin Deficiency Vasculitis Emerging Drugs and Major Players

Table of Contents

  1. Introduction
  2. Executive Summary
  3. Alpha-1 Antitrypsin Deficiency: Overview
  4. Pipeline Therapeutics
  5. Therapeutic Assessment
  6. Alpha-1 Antitrypsin Deficiency– DelveInsight’s Analytical Perspective
  7. Late Stage Products (Phase III)
  8. ARO AAT: Arrowhead Pharmaceuticals
  9. Drug profiles in the detailed report…..
  10. Mid Stage Products (Phase II)
  11. Drug Name: Company Name
  12. Drug profiles in the detailed report…..
  13. Early Stage Products (Phase I/II)
  14. ALN-AAT02: Alnylam Pharmaceuticals
  15. Drug profiles in the detailed report…..
  16. Preclinical and Discovery Stage Products
  17. Drug Name: Company Name
  18. Drug profiles in the detailed report…..
  19. Inactive Products
  20. Alpha-1 Antitrypsin Deficiency Key Companies
  21. Alpha-1 Antitrypsin Deficiency Key Products
  22. Alpha-1 Antitrypsin Deficiency- Unmet Needs
  23. Alpha-1 Antitrypsin Deficiency- Market Drivers and Barriers
  24. Alpha-1 Antitrypsin Deficiency- Future Perspectives and Conclusion
  25. Alpha-1 Antitrypsin Deficiency Analyst Views
  26. Alpha-1 Antitrypsin Deficiency Key Companies
  27. Appendix

About Us

DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.

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Website: https://www.delveinsight.com/report-store/alpha-1-antitrypsin-deficiency-aatd-pipeline-insight