Alpha 1-Antitrypsin Deficiency - Pipeline Insight, 2026

DelveInsight's, "Alpha 1-Antitrypsin Deficiency - Pipeline Insight, 2026" report provides comprehensive insights about 18+ companies and 20+ pipeline drugs in Alpha 1-Antitrypsin Deficiency pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered:

• Global coverage

Alpha 1-Antitrypsin Deficiency: Understanding

Alpha 1-Antitrypsin Deficiency: Overview

Alpha-1 antitrypsin deficiency (AATD) is an inherited disorder caused by reduced levels or dysfunctional forms of alpha-1 antitrypsin (AAT), a protein that protects lung tissue from neutrophil elastase. It primarily affects the lungs and liver, with disease severity varying substantially according to genotype and environmental factors. AATD can cause early-onset emphysema, chronic obstructive pulmonary disease (COPD) and bronchiectasis, while abnormal AAT accumulation in hepatocytes can lead to liver disease. Smoking markedly accelerates pulmonary damage and disease progression.

Pulmonary manifestations commonly include progressive shortness of breath, chronic cough, wheezing, sputum production and reduced exercise tolerance, often resembling asthma or conventional COPD. Liver involvement can present with neonatal cholestasis in children and fibrosis, cirrhosis or hepatocellular carcinoma in adults. Some individuals may remain asymptomatic for years, contributing to substantial underdiagnosis and diagnostic delays. Rare extra-pulmonary manifestations include necrotizing panniculitis and C-ANCA-positive vasculitis.

AATD is primarily caused by pathogenic variants in the SERPINA1 gene, which encodes alpha-1 antitrypsin. The most severe deficiency commonly occurs with the PI*ZZ genotype, resulting in markedly reduced circulating AAT levels. Insufficient AAT allows uncontrolled neutrophil elastase activity in the lungs, promoting destruction of alveolar tissue and emphysema, while certain abnormal AAT proteins polymerize and accumulate within hepatocytes, causing liver injury. Smoking, occupational pollutants and other environmental exposures can further increase pulmonary disease risk

Diagnosis generally begins with measurement of serum AAT concentration, followed by phenotyping and/or genetic testing to identify pathogenic SERPINA1 variants and characterize disease severity. Pulmonary function testing, chest CT and liver assessments help determine the extent of organ involvement. Current management emphasizes smoking avoidance, vaccination, pulmonary rehabilitation and standard COPD therapies, while intravenous AAT augmentation therapy is the established disease-modifying treatment for selected patients with AATD-associated emphysema. Advanced lung or liver disease may require transplantation, while inhaled AAT, recombinant proteins, RNA-based approaches and gene-editing therapies remain under investigation.

"Alpha 1-Antitrypsin Deficiency- Pipeline Insight, 2026" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Alpha 1-Antitrypsin Deficiency pipeline landscape is provided which includes the disease overview and Alpha 1-Antitrypsin Deficiency treatment guidelines. The assessment part of the report embraces, in depth Alpha 1-Antitrypsin Deficiency commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Alpha 1-Antitrypsin Deficiency collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights:

• The companies and academics are working to assess challenges and seek opportunities that could influence Alpha 1-Antitrypsin Deficiency R&D. The therapies under development are focused on novel approaches to treat/improve Alpha 1-Antitrypsin Deficiency.

Alpha 1-Antitrypsin Deficiency Emerging Drugs Chapters

This segment of the Alpha 1-Antitrypsin Deficiency report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Alpha 1-Antitrypsin Deficiency Emerging Drugs

Fazirsiran: Takeda and Arrowhead Pharmaceuticals

Fazirsiran (TAK-999) is an investigational GalNAc-conjugated RNA interference (RNAi) therapy being developed by Takeda and Arrowhead Pharmaceuticals for alpha-1 antitrypsin deficiency (AATD)-associated liver disease. It is designed to selectively silence SERPINA1 mRNA in hepatocytes, reducing production of abnormal Z-AAT protein and its accumulation in the liver. The resulting reduction in toxic protein accumulation is intended to slow or prevent progression of liver fibrosis and associated complications. Fazirsiran is currently in Phase III development for AATD-associated liver disease in the US and EU.

SAR447537: Sanofi

SAR447537 (efdoralprin alfa; formerly INBRX-101) is an investigational recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein being developed by Sanofi for AATD-associated emphysema. It is designed to restore functional AAT levels and inhibit neutrophil elastase, thereby protecting lung tissue from protease-mediated damage. Its Fc fusion is intended to extend the drug's half-life and potentially enable once-every-3- to 4-week dosing versus weekly plasma-derived AAT augmentation. SAR447537 is being evaluated in a Phase II open-label extension study for long-term safety and efficacy.

KB408: Krystal Biotech

KB408 is an investigational gene therapy being developed by Krystal Biotech for alpha-1 antitrypsin deficiency (AATD). It utilizes Krystal's proprietary STAR-D platform to deliver a functional copy of the SERPINA1 gene, with the aim of enabling sustained production of functional alpha-1 antitrypsin (AAT). The approach is designed to address the underlying genetic deficiency rather than provide repeated protein replacement therapy. KB408 is being evaluated in Phase I/II clinical development in patients with AATD-associated lung disease.

Alpha 1-Antitrypsin Deficiency: Therapeutic Assessment

This segment of the report provides insights about the different Alpha 1-Antitrypsin Deficiency drugs segregated based on following parameters that define the scope of the report, such as:

• Major Players in Alpha 1-Antitrypsin Deficiency
• There are approx. 18+ key companies which are developing the therapies for Alpha 1-Antitrypsin Deficiency. The companies which have their Alpha 1-Antitrypsin Deficiency drug candidates in the most advanced stage, i.e. Phase III include, Takeda and Arrowhead Pharmaceuticals.
• Phases

DelveInsight's report covers around 20+ products under different phases of clinical development like

• 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
• Route of Administration

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

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Alpha 1-Antitrypsin Deficiency: Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Alpha 1-Antitrypsin Deficiency therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Alpha 1-Antitrypsin Deficiency drugs.

Alpha 1-Antitrypsin Deficiency Report Insights

• Alpha 1-Antitrypsin Deficiency Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Alpha 1-Antitrypsin Deficiency Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions:

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Alpha 1-Antitrypsin Deficiency drugs?
• How many Alpha 1-Antitrypsin Deficiency drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Alpha 1-Antitrypsin Deficiency?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Alpha 1-Antitrypsin Deficiency therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Alpha 1-Antitrypsin Deficiency and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Takeda
• Arrowhead Pharmaceuticals
• Sanofi
• Krystal Biotech
• Beam Therapeutics
• AIRNA Corporation
• Tessera Therapeutics
• CRISPR Therapeutics
• Wave Life Sciences
• Congruence Therapeutics
• Prime Medicine

Key Products

• Fazirsiran
• SAR447537
• KB-408
• BEAM-302
• AIR-001
• TSRA-196
• CTX460
• WVE-006
• CGX-195
• PM647