Alpha-1 Antitrypsin Deficiency Pipeline: 40+ Pioneering Companies Driving Innovation in Targeted AATD Therapies | DelveInsight

The Alpha-1 antitrypsin deficiency market is entering a new era of innovation, driven by over 20 leading biotech and pharma players, including Intellia Therapeutics, Apic Bio, Linton Pharm, Z Factor Limited, Vertex Pharmaceuticals, Grifols, and Kamada. These visionaries are reshaping treatment paradigms with next-gen therapies that promise greater precision and better outcomes.

DelveInsight's "Alpha-1 Antitrypsin Deficiency Pipeline Insight, 2025" comprehensively analyzes the current clinical landscape and growth prospects in the Alpha-1 antitrypsin deficiency market. The report covers disease insights, treatment guidelines, and a detailed pipeline assessment from preclinical to marketed stages. It includes drug mechanisms, clinical studies, regulatory progress, and key developments such as collaborations, mergers, funding, and designations.

For emerging Alpha-1 antitrypsin deficiency drugs, the Alpha-1 antitrypsin deficiency pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.

Key Takeaways from the Alpha-1 Antitrypsin Deficiency Pipeline Report
• DelveInsight's Alpha-1 antitrypsin deficiency pipeline analysis depicts a robust space with 40+ active players working to develop 40+ pipeline drugs for Alpha-1 antitrypsin deficiency treatment.
• The leading Alpha-1 antitrypsin deficiency companies include Intellia Therapeutics, Apic Bio, Linton Pharm, Z Factor Limited, Vertex Pharmaceuticals, Grifols, Kamada, Octapharma, PPL Therapeutics, Precigen Inc., and others are evaluating their lead assets to improve the Alpha-1 antitrypsin deficiency treatment landscape.
• Key Alpha-1 antitrypsin deficiency pipeline therapies in various stages of development include APB 101, Research programme: gene editing therapeutics, LP 503, ZF 874, VX 864, KB408, Alpha-1-antitrypsin inhalation, Alpha-1 antitrypsin, OctaAlpha1, Alpha-1-antitrypsin subcutaneous, WVE-006, BEAM-30, and others.
• In March 2025, Korro Bio, Inc. (Nasdaq: KRRO) announced that the FDA has granted orphan drug designation to its investigational RNA-editing therapy, KRRO-110, for the treatment of Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disorder affecting the lungs and liver.
• In March 2025, Beam Therapeutics Inc. (Nasdaq: BEAM) announced that the FDA has cleared its investigational new drug (IND) application for BEAM-302, a base editing therapy aimed at treating alpha-1 antitrypsin deficiency (AATD). AATD is a genetic disorder that impacts the lungs and liver, often leading to early-onset emphysema and liver disease, with no curative treatments currently available.
• In October 2024, Wave Life Sciences announced that it is expecting to share multidose data from the RestorAATion-2 trial in 2025.
• In September 2024, Krystal Biotech amended the protocol for its KB408 Phase I SERPENTINE-1 study to include mandatory bronchoscopies in this cohort to measure alpha-1 antitrypsin expression.
• In July 2024, Beam Therapeutics entered into a settlement agreement with a research institution pursuant to which, in exchange for a release of claims in its favor, the company agreed, among other things, to pay the research institution an upfront payment of USD 15 million and to make additional payments contingent upon the development and commercialization of BEAM-302.
• In February 2024, Grifols announced that it had introduced 4 and 5 g vials of PROLASTIN used for long-term AAT augmentation therapy in patients with severe AATD who show evidence of progressive lung disease.
• In January 2024, the US FDA approved the supplement Biologics License Application (sBLA) for Alpha-1-Proteinase Inhibitor ZEMAIRA.

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Alpha-1 Antitrypsin Deficiency Overview
Alpha-1 antitrypsin deficiency (AATD) is a genetic condition that can lead to both lung and liver disease, with symptoms typically appearing between the ages of 20 and 50. The condition is caused by mutations in the SERPINA1 gene, which encodes the alpha-1 antitrypsin protein-a protective agent that shields lung tissue from damage by neutrophil elastase, a powerful enzyme. In AATD, insufficient levels or dysfunctional forms of this protein allow neutrophil elastase to damage the alveoli, contributing to lung disease. Additionally, abnormal alpha-1 antitrypsin can accumulate in the liver, potentially causing liver damage. Environmental factors such as smoking, chemical exposure, and airborne irritants can worsen the severity of the disease.

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Alpha-1 Antitrypsin Deficiency Treatment Analysis: Drug Profile
VX-814 and VX-864: Vertex Pharmaceuticals
Vertex Pharmaceuticals is developing small molecule therapies aimed at treating the root cause of Alpha-1 Antitrypsin Deficiency (AATD) by targeting both lung and liver manifestations of the disease. The company is currently evaluating two clinical candidates, VX-814 and VX-864, which are in Phase II development, as part of a broader pipeline focused on AATD.

ZF874: Z Factor Ltd
ZF874 is a first-in-class small molecule designed to act as a molecular "patch" that corrects the misfolding of the faulty Z variant of alpha-1 antitrypsin (Z-A1AT). Developed using a proprietary protein crystal structure, ZF874 promotes proper folding of the A1AT protein, helping reduce liver accumulation of misfolded proteins while restoring functional A1AT in circulation to protect the lungs. In mouse models engineered to express human Z-A1AT, oral administration of ZF874 significantly increased levels of functional protein and cleared misfolded variants from the liver. The drug is currently in Phase I clinical development.

Key Alpha-1 Antitrypsin Deficiency Therapies and Companies
• Inhaled Alpha 1-Antitrypsin (AAT): Kamada Pharmaceuticals
• Fazirsiran (ARO-AAT/TAK-999): Arrowhead Pharmaceuticals and Takeda
• Alvelestat (MPH-966): Mereo BioPharma/AstraZeneca
• SAR447537/INBRX-101: Sanofi/Inhibrx Biosciences
• WVE-006: Wave Life Sciences
• BEAM-302: Beam Therapeutics
• Alpha-1 AT 15% (SC): Grifols
• KB408: Krystal Biotech
• VX-814 and VX-864: Vertex Pharmaceuticals
• ZF874: Z Factor Ltd
• APB-101: Apic Bio

Learn more about the novel and emerging Alpha-1 antitrypsin deficiency pipeline therapies @ https://www.delveinsight.com/report-store/alpha-antitrypsin-deficiency-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Alpha-1 Antitrypsin Deficiency Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

By Stage
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

By Route of Administration
• Infusion
• Intradermal
• Intramuscular
• Intranasal
• Intravaginal
• Oral
• Parenteral
• Subcutaneous
• Topical

By Molecule Type
• Gene therapies
• Small molecule
• Vaccines
• Polymers
• Peptides
• Monoclonal antibodies

Scope of the Alpha-1 Antitrypsin Deficiency Pipeline Report
• Coverage: Global
• Key Alpha-1 Antitrypsin Deficiency Companies: Intellia Therapeutics, Apic Bio, Linton Pharm, Z Factor Limited, Vertex Pharmaceuticals, Grifols, Kamada, Octapharma, PPL Therapeutics, Precigen Inc., and others.
• Key Alpha-1 Antitrypsin Deficiency Pipeline Therapies: APB 101, Research programme: gene editing therapeutics, LP 503, ZF 874, VX 864, KB408, Alpha-1-antitrypsin inhalation, Alpha-1 antitrypsin, OctaAlpha1, Alpha-1-antitrypsin subcutaneous, WVE-006, BEAM-30, and others.

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Table of Contents
1. Introduction
2. Executive Summary
3. Alpha-1 Antitrypsin Deficiency Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Alpha-1 Antitrypsin Deficiency Pipeline Therapeutics
6. Alpha-1 Antitrypsin Deficiency Pipeline: Late-Stage Products (Phase III)
7. Alpha-1 Antitrypsin Deficiency Pipeline: Mid-Stage Products (Phase II)
8. Alpha-1 Antitrypsin Deficiency Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix

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