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Coverage: 2020-01-01 onwards, with up to 300 results per query from PubMed and Europe PMC and separate limits on other sources. This is a selected index, not a complete literature search.
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Alpha-1 Antitrypsin Deficiency: Research Summary
Printed from RareWays (rareways.org)
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Advances in Precision Editing Therapies for Alpha-1 Antitrypsin Deficiency.
Gao Jenny et al., Human gene therapy (1 October 2026)
https://pubmed.ncbi.nlm.nih.gov/41588875/
- 2.
Lung Function Variability in Alpha-1 Antitrypsin Deficiency: Implications for Clinical Trials.
Strange Charlie et al., Chronic obstructive pulmonary diseases (Miami, Fla.) (25 September 2026)
https://pubmed.ncbi.nlm.nih.gov/42610689/
- 3.
Exploring clinical heterogeneity in alpha-1 antitrypsin deficiency: a clustering analysis of patients with Pi*ZZ and Pi*SZ genotypes from the Spanish data in EARCO registry
Laura Villar-Aguilar et al., Respiratory Research (21 September 2026)
https://doi.org/10.1186/s12931-026-03919-5
- 4.
The Alpha-1 Antitrypsin Z-allele associates with sarcopenia in the UK Biobank.
Attaway Amy et al., medRxiv : the preprint server for health sciences (14 September 2026)
https://pubmed.ncbi.nlm.nih.gov/42779858/
- 5.
Obstructive Sleep Apnea in Individuals with Alpha-1 Antitrypsin Deficiency: A Single Center Experience.
Boppana Leela Krishna Teja et al., Lung (11 September 2026)
https://pubmed.ncbi.nlm.nih.gov/42726312/
Clinical Trials : Australian Sites
Ask your doctor whether you or your child may be eligible for any of these trials. Each has its ID and full eligibility criteria at the link shown.
- 1.
A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)
NCT07227207 · Recruiting · Phase 1 · Tessera Therapeutics, Inc.
Ages 18 to 70
https://clinicaltrials.gov/study/NCT07227207
- 2.
A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)
NCT07431112 · Recruiting · Phase 1 · AIRNA Corporation
Ages 18 to 74
https://clinicaltrials.gov/study/NCT07431112
- 3.
A Phase 1/2a, Single- and Multiple-dose Escalation Study of KRRO-110
NCT06677307 · Active (not recruiting) · Phase 1 · Korro Bio, Inc.
https://clinicaltrials.gov/study/NCT06677307
- 4.
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD
NCT06405633 · Active (not recruiting) · Phase 1 · Wave Life Sciences USA, Inc.
Ages 18 to 70
https://clinicaltrials.gov/study/NCT06405633
- 5.
Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema
NCT05897424 · Active (not recruiting) · Phase 2 · Sanofi
Ages 18 to 80
https://clinicaltrials.gov/study/NCT05897424
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Alpha-1 Antitrypsin Deficiency
Alpha-1 antitrypsin deficiency is an inherited condition where the body does not produce enough of a protective protein for the lungs, leading to early-onset emphysema and liver disease. It is significantly underdiagnosed, often mistaken for COPD or asthma. Gene and RNA editing therapies in clinical trials offer hope for a future cure.
This overview is general information, not an individual medical assessment. Research is selected automatically and may include mismatches. How we select and explain research.
Most Recent Research
Genome and RNA editing modalities have revolutionized precision gene therapy, offering a safer alternative to traditional gene replacement approaches. Alpha-1 antitrypsin deficiency (AATD) is a compelling model for precision medicine because the disease mechanism is well defined-mutations in a single gene are responsible for both liver and lung pathology. In this review, we summarize the current preclinical and clinical efforts for AATD, with an emphasis on genome and RNA editing strategies.
Common Questions
What is Alpha-1 Antitrypsin Deficiency?
Alpha-1 antitrypsin deficiency is an inherited condition where the body does not produce enough of a protective protein for the lungs, leading to early-onset emphysema and liver disease. It is significantly underdiagnosed, often mistaken for COPD or asthma. Gene and RNA editing therapies in clinical trials offer hope for a future cure.
How many clinical trials are available for Alpha-1 Antitrypsin Deficiency?
RareWays currently indexes 49 clinical trials for Alpha-1 Antitrypsin Deficiency, of which 7 are actively recruiting. These counts cover indexed trials across countries, not just your country. An Australian site does not necessarily mean that site is recruiting. Check locations, eligibility and current availability with the study team.
Where does the research data for Alpha-1 Antitrypsin Deficiency come from?
RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is refreshed automatically every day. All articles and trials link directly to their original sources.
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