United States Alpha-1 Antitrypsin Deficiency Therapy Market 2031 | Growth Drivers, Key Players & Investment Opportunities

Market Size and Growth

The global Alpha-1 Antitrypsin Deficiency Therapy market is growing at a high CAGR during the forecast period (2024-2031).

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Key Development:

United States: Recent AATD Therapy Developments

✅ In November 2025, Korro Bio reported interim data from its Phase 1/2a REWRITE trial of RNA‐editing candidate KRRO-110 in AATD patients: while functional M‐AAT protein was generated, the increase did not reach the pre-defined protective threshold, prompting the company to shift toward a next‐generation, GalNAc‐conjugated construct expected for development in 2026.

✅ In October 2025, CRISPR Therapeutics announced that its gene‐editing candidate CTX460 built on the novel SyNTaseTM platform achieved > 90% mRNA correction and a 5‐fold increase in AAT protein levels in preclinical AATD models, paving the way for first‐in‐human trials expected by mid‐2026.

✅ In October 2025, CRISPR Therapeutics' SyNTase platform which underlies CTX460 was highlighted at the European Society of Gene and Cell Therapy Congress 2025, reinforcing the potential for a one‐time, in vivo gene‐correction therapy for AATD that could surpass conventional augmentation therapies.

✅ In May 2025, AIRNA raised an oversubscribed US$155 million Series B funding round to advance its RNA‐editing candidate AIR-001 for AATD into Phase 1/2 trials a therapy designed to restore functional AAT production via subcutaneous dosing, offering a potential non‐invasive alternative to conventional therapy.

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Japan: Recent AATD Therapy/Context Developments

✅ In November 2025, it was reported that Korro Bio despite being a U.S.-based biotech acknowledged the failure of KRRO-110 to meet expected protein‐level thresholds in its REWRITE trial, prompting withdrawal and staff layoffs. This underscores the challenge of translating RNA‐editing therapies into effective treatments a cautionary note relevant to global efforts including those in Japan.

✅ In July 2025, a clinical case report described siblings in Japan being newly diagnosed with AATD following liver complications, highlighting real‐world unmet needs for therapeutic options in Japan, where AATD is rare but under‐recognized.

✅ As of 2025, AATD remains classified as a designated rare (難病) disease in Japan under the Nanbyō-law framework but no AAT augmentation therapy has yet been approved domestically, reinforcing the urgency and potential impact if global gene‐ or RNA‐editing treatments become available in Japan.

✅ The rarity of AATD in Japan (estimated 2.03‐2.08 per 10 million) often leads to under‐diagnosis, meaning that even incremental advances in global AATD therapy such as those by CRISPR or AIRNA could have outsized importance for the few Japanese patients who are identified.

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Future Pipelines & Projects in Alpha‐1 Antitrypsin Deficiency (AATD) Therapy

The AATD treatment landscape is evolving rapidly, with gene-editing, base-editing, and next-gen biologics set to transform patient care.

✅ Prime Medicine - Prime Editing + Liver-Targeted Delivery

Preclinical models show restoration of normal AAT levels.

IND filing expected by mid‐2026, potentially leading to first-in-human trials shortly after.

✅ CRISPR Therapeutics - SyNTase Editing

Preclinical data show efficient SERPINA1 mutation correction in animals.

Promises one-time therapy potential, addressing both lung and liver disease.

✅ Beam Therapeutics - BEAM-302 (Base Editing Therapy)

IND cleared in early 2025, first clinical base-editing trial for AATD.

Marks a major milestone for gene-editing pipelines in rare diseases.

✅ Next-Gen Biologics & Augmentation Therapies

Improved recombinant AAT proteins and subcutaneous/infrequent infusion formats.

Enhances patient convenience, adherence, and overall treatment efficacy.

✅ Why This Pipeline Matters

Curative potential: Shifts focus from symptom management to genetic correction.

Dual organ benefit: Protects lungs and liver simultaneously.

Market opportunity: Rising patient diagnosis and regulatory interest make this an exciting space for biotech partnerships and investments.

Key Players:

=> Kamada Pharmaceuticals, Takeda Pharmaceutical Company Limited, CSL, Vertex Pharmaceuticals Incorporated, Grifols, Arrowhead Pharmaceuticals, Inc., Dicerna Pharmaceuticals, Inc., Inhibrx, Inc., Wave Life Sciences and Mereo Biopharma Group PLC among others.

Key Segments:

By Therapy

Augmentation Therapy leads with Prolastin-C, Aralast, Zemaira, Glassia, capturing 40% market share, primarily used to replace deficient alpha-1 antitrypsin in patients. Medication accounts for 35%, including Bronchodilators (Ipratrop!um Bromide, Theophylline) and Antibiotics (Doxycycline, Dapsone), addressing symptom management and infection control. Oxygen Therapy and Smoking Cessation Therapy hold 15%, while Others contribute 10%, covering supportive and adjunct therapies.

By Route of Administration

Inhaled therapies dominate with 55% share, driven by bronchodilator use and patient convenience. Intravenous therapies account for 35%, mainly for augmentation therapy infusions. Others make up 10%, including combination or experimental delivery methods.

By End-User

Hospitals lead with 50% share, offering comprehensive treatment and infusion services. Ambulatory Surgical Centers account for 30%, supporting outpatient management of AATD. Others represent 20%, including specialty clinics, homecare programs, and research centers.

By Region

North America - 35% Share
Dominated by well-established healthcare infrastructure, high awareness, and insurance coverage for augmentation therapy.

Europe - 30% Share
Growth driven by government-funded healthcare, specialized AATD centers, and rising adoption of augmentation therapies.

Asia Pacific - 20% Share
Emerging awareness, increasing diagnostic capabilities, and growing healthcare access drive market growth.

Latin America - 10% Share
Opportunities in specialized urban centers with expanding hospital and outpatient services.

Middle East & Africa - 5% Share
Growth is limited but rising awareness and specialized treatment adoption provide niche opportunities.

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