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

 

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Key Takeaways from the Alpha 1 Antitrypsin Deficiency Pipeline Report

• In February 2025:- Grifols Therapeutics Inc.:- The purpose of this study is to evaluate the safety and tolerability of 72 milligrams per kilogram (mg/kg) and 180 mg/kg Alpha-1 15%, administered as a single-dose subcutaneous (SC) infusion and subsequently as weekly SC infusions over 8 weeks in participants with Alpha1-Antitrypsin Deficiency (AATD).
• In January 2025:- Takeda:- The liver produces a protein called alpha-1 antitrypsin (AAT). AAT is normally released into the bloodstream. In some people, the liver makes an abnormal version of the AAT protein, called Z-AAT. Making an abnormal version of the AAT protein can result in liver disease as Z-AAT builds up in liver cells, which leads to liver problems such as liver scarring (fibrosis), continuing liver damage (cirrhosis), and eventually endstage liver disease.
• In January 2025:- Korro Bio Inc.:- The purpose of this first-in human (FIH) study is to assess the safety, tolerability, and pharmacokinetics (PK) of single and multiple doses of KRRO-110 in both healthy adult participants and in clinically stable patients with Alpha-1 antitrypsin deficiency (AATD).
• In January 2025:- Arrowhead Pharmaceuticals:– The purpose of AROAAT2001 (SEQUOIA) is to evaluate the safety, efficacy and tolerability of multiple doses of the investigational product, Fazirsiran Injection, administered subcutaneously to participants with alpha-1 antitrypsin deficiency (AATD).
• In January 2025:- Kamada Ltd:- The goal of this clinical trial is to learn if AAT for inhalation, at a dose of 80 mg/day can slow the progression of lung disease in people who have lung disease caused by severe genetic deficiency in Alpha 1 Antitrypsin (AATD).
• DelveInsight’s Alpha 1 Antitrypsin Deficiency pipeline report depicts a robust space with 12+ active players working to develop 12+ pipeline therapies for Alpha 1 Antitrypsin Deficiency treatment.
• The leading Alpha 1 Antitrypsin Deficiency Companies such as Octapharma, Dicerna Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Vertex Pharmaceuticals, AstraZeneca, Kamada Ltd and others.
• Promising Alpha-1 Antitrypsin Deficiency Pipeline Therapies such as Fazirsiran Injection, Aerosolized, Recombinant Alpha 1-Antitrypsin, INBRX-101/rhAAT-Fc, ADVM-043, and others.

 

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Alpha 1 Antitrypsin Deficiency 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.

 

The Alpha 1 Antitrypsin Deficiency pipeline report provides insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Alpha 1 Antitrypsin Deficiency 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 Treatment.
• Alpha 1 Antitrypsin Deficiency Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Alpha 1 Antitrypsin Deficiency 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 market.

 

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Alpha 1 Antitrypsin Deficiency Companies

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

 

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

• Oligonucleotide
• Peptide
• Small molecule

 

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Scope of the Alpha 1 Antitrypsin Deficiency Pipeline Report

• Coverage- Global
• Alpha 1 Antitrypsin Deficiency Companies- Octapharma, Dicerna Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Vertex Pharmaceuticals, AstraZeneca, Kamada Ltd and others.
• Alpha-1 Antitrypsin Deficiency Pipeline Therapies– Fazirsiran Injection, Aerosolized, Recombinant Alpha 1-Antitrypsin, INBRX-101/rhAAT-Fc, ADVM-043, and others.
• Alpha 1 Antitrypsin Deficiency Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Alpha 1 Antitrypsin Deficiency Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

 

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Table of Content

1. Introduction
2. Alpha-1 Antitrypsin Deficiency Executive Summary
3. Alpha-1 Antitrypsin Deficiency: Overview
4. Alpha-1 Antitrypsin Deficiency Pipeline Therapeutics
5. Alpha-1 Antitrypsin Deficiency Pipeline 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

 

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