Alpha-1 Antitrypsin Deficiency Pipeline Insight 2025: 40+ Companies Advancing Targeted Therapies for a Rare Genetic Disorder | DelveInsight

DelveInsight's "Alpha-1 Antitrypsin Deficiency - Pipeline Insight, 2025" provides a detailed analysis of the emerging therapeutic landscape for Alpha-1 Antitrypsin Deficiency (AATD), a rare, inherited condition caused by mutations in the SERPINA1 gene. The disorder leads to low levels or dysfunction of alpha-1 antitrypsin, a critical protease inhibitor that protects lung tissue. AATD is associated with progressive pulmonary complications such as early-onset emphysema, as well as liver diseases, including cirrhosis and hepatocellular carcinoma, due to toxic protein buildup.

While current standard treatment relies heavily on intravenous augmentation therapy using plasma-derived AAT, it remains palliative and does not prevent disease progression or address liver manifestations. However, 2025 marks a turning point in the field, with over 40 companies globally advancing a diverse pipeline of next-generation therapies aimed at correcting the underlying molecular defect. These include gene therapies using adeno-associated virus (AAV) and lentiviral vectors, RNA interference (RNAi), mRNA-based therapies, and CRISPR-Cas9-based gene editing-all targeting the root cause of AATD by restoring or regulating AAT expression.

Among the frontrunners, Arrowhead Pharmaceuticals' RNAi therapy ARO-AAT has shown potential in reducing mutant Z-AAT protein accumulation in the liver, with early clinical data indicating improved biomarkers of liver health. Companies like Takeda, Vertex Pharmaceuticals, Intellia Therapeutics, SpliceBio, and Krystal Biotech are also exploring innovative modalities to either halt liver injury or boost circulating AAT levels. Many of these programs have secured orphan drug designation, Fast Track, or Rare Pediatric Disease status from regulatory authorities, including the FDA and EMA, accelerating development timelines and access.

As interest in rare genetic diseases grows, the AATD pipeline is witnessing increased investment, collaborative R&D, and improved diagnostics, including expanded genetic screening. These advances are setting the stage for a shift from symptomatic management to disease-modifying or potentially curative interventions-bringing new hope to patients long underserved by conventional therapies.

Interested in learning more about the current treatment landscape and the key drivers shaping the Alpha-1 Antitrypsin Deficiency pipeline? Click here: https://www.delveinsight.com/report-store/alpha-antitrypsin-deficiency-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Key Takeaways from the Alpha-1 Antitrypsin Deficiency Pipeline Report
• DelveInsight's Alpha-1 Antitrypsin Deficiency pipeline analysis depicts a strong 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 DUR-928, Canakinumab, IMM 124-E, Research Programme: humanized virus suppressing factor, F652, SZN-043, SRT-015, and others.
• In May 2025, Beam Therapeutics (Nasdaq: BEAM) announced the FDA granted orphan drug designation to BEAM-302, a liver-targeted lipid nanoparticle therapy using base editing to correct mutations in alpha-1 antitrypsin deficiency (AATD) patients.
• In May 2025, Beam Therapeutics (Nasdaq: BEAM) announced that the FDA granted RMAT designation to BEAM-302, a liver-targeted base editing therapy for alpha-1 antitrypsin deficiency (AATD), a genetic disorder causing lung and liver disease with high unmet medical need.
• In March 2025, Beam Therapeutics Inc. (Nasdaq: BEAM) announced that the U.S. Food and Drug Administration (FDA) had cleared the investigational new drug (IND) application for BEAM-302, a treatment for alpha-1 antitrypsin deficiency (AATD).

Request a sample and discover the recent breakthroughs happening in the Alpha-1 Antitrypsin Deficiency pipeline landscape at https://www.delveinsight.com/report-store/alpha-antitrypsin-deficiency-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Alpha-1 Antitrypsin Deficiency Overview
Alpha-1 Antitrypsin Deficiency (AATD) is a rare, inherited genetic disorder caused by mutations in the SERPINA1 gene, leading to low levels or malfunctioning of alpha-1 antitrypsin (AAT) protein. This protein protects the lungs from neutrophil elastase, an enzyme that can damage lung tissue if not properly regulated.

Individuals with AATD are at increased risk of developing chronic obstructive pulmonary disease (COPD), especially emphysema, and liver disease, including cirrhosis and hepatocellular carcinoma. Symptoms often include shortness of breath, wheezing, chronic cough, and in some cases, unexplained liver problems. Early diagnosis and lifestyle modifications, such as avoiding smoking and environmental irritants, are key to managing disease progression.

Treatment options include augmentation therapy, where AAT protein derived from human plasma is administered to slow lung function decline. Other supportive treatments may include bronchodilators, corticosteroids, pulmonary rehabilitation, and, in severe cases, lung or liver transplantation. Genetic counseling and family screening are also recommended due to the hereditary nature of the disease.

Find out more about Alpha-1 Antitrypsin Deficiency medication at https://www.delveinsight.com/report-store/alpha-antitrypsin-deficiency-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

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) and addressing related lung and liver damage. Their two investigational candidates, VX-814 and VX-864, are currently in Phase II clinical development. These therapies are part of Vertex's broader effort to build a portfolio targeting AATD.

ZF874: Z Factor Ltd
ZF874 is an oral investigational drug developed using proprietary structural insights to target the misfolded Z-AAT protein. It works by stabilizing the faulty protein, promoting correct folding, and reducing harmful polymer buildup in the liver while restoring functional AAT levels in the bloodstream. In preclinical studies involving genetically modified mice, ZF874 significantly improved blood levels of functional protein and cleared liver accumulation. It is currently in Phase I clinical trials.

Learn more about the novel and emerging Alpha-1 Antitrypsin Deficiency pipeline therapies at 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: DUR-928, Canakinumab, IMM 124-E, Research Programme: humanized virus suppressing factor, F652, SZN-043, SRT-015, and others.

To dive deep into rich insights for drugs used for Alpha-1 Antitrypsin Deficiency treatment, visit: https://www.delveinsight.com/report-store/alpha-antitrypsin-deficiency-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

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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