ALPHA 1-ANTITRYPSIN DEFICIENCY
Clinical trials for ALPHA 1-ANTITRYPSIN DEFICIENCY explained in plain language.
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Gene therapy trial aims to fix protein deficiency behind lung and liver disease
Disease control Recruiting nowThis early-stage study is testing a new gene therapy called BEAM-302 in adults with Alpha-1 antitrypsin deficiency, a genetic condition that can damage the lungs and liver. The main goals are to find a safe dose and see if the treatment can increase levels of a missing protective…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1, PHASE2 • Sponsor: Beam Therapeutics Inc. • Aim: Disease control
Last updated Apr 02, 2026 03:41 UTC
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Extended trial tests new therapy for genetic lung disease
Disease control Recruiting nowThis study aims to evaluate the long-term safety and effectiveness of an investigational drug called SAR447537 (INBRX-101) for adults with emphysema caused by Alpha-1 antitrypsin deficiency. The trial will follow 185 participants who continue receiving the treatment to monitor si…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE2 • Sponsor: Sanofi • Aim: Disease control
Last updated Apr 02, 2026 03:41 UTC
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Daily inhaler tested to halt genetic lung disease progression
Disease control Recruiting nowThis study is testing whether inhaling a protein called Alpha-1 Antitrypsin (AAT) daily can slow down worsening lung function in people with a genetic condition called Alpha-1 Antitrypsin Deficiency. For the first two years, 220 participants will either receive the active inhaled…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE3 • Sponsor: Kamada, Ltd. • Aim: Disease control
Last updated Mar 31, 2026 12:10 UTC
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First pill for genetic lung & liver disease enters human testing
Disease control Recruiting nowThis is a very early study to check if a new oral drug called BMN 349 is safe for people with Alpha-1 Antitrypsin Deficiency, a genetic condition that can damage the lungs and liver. A small group of 12 adults with specific genetic types of the disease will take a single dose of …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated Mar 30, 2026 14:34 UTC
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First-in-Human trial tests new shot for genetic lung and liver disease
Disease control Recruiting nowThis is a first-step study to see if a new injected medicine called AIR-001 is safe and how it behaves in the body of people with Alpha-1 Antitrypsin Deficiency (AATD). It will enroll 54 adults with a specific genetic form (PiZZ) of the disease. The main goal is to check for side…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • Sponsor: AIRNA Corporation • Aim: Disease control
Last updated Mar 30, 2026 14:30 UTC
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One-Shot gene therapy tested for rare lung disease
Disease control Recruiting nowThis is an early safety study for a new gene therapy designed to treat Alpha-1 Antitrypsin Deficiency, a genetic condition that can cause serious lung disease. Researchers are testing a single injection that aims to help the body produce a protective protein long-term. The study …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • Sponsor: Weill Medical College of Cornell University • Aim: Disease control
Last updated Mar 30, 2026 14:29 UTC
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First human tests begin for new genetic disease therapy
Disease control Recruiting nowThis early-stage study is testing a new drug called WVE-006 for people with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can damage the lungs and liver. Researchers will give the drug to 24 participants to first check if it's safe and how the body handles it. T…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1, PHASE2 • Sponsor: Wave Life Sciences Ltd. • Aim: Disease control
Last updated Mar 23, 2026 15:18 UTC
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Hunt for early warning signs in genetic lung disease
Knowledge-focused Recruiting nowThis study aims to find early signs that predict which people with a specific genetic lung disease (Alpha-1 PiMZ) are most likely to get worse. Researchers will follow 80 participants for three years, using CT scans and blood tests to track changes in their lungs. The goal is to …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: Columbia University • Aim: Knowledge-focused
Last updated Apr 01, 2026 14:42 UTC
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Scientists probe hidden link between genetic disorders and lung inflammation
Knowledge-focused Recruiting nowThis study aims to understand how two genetic conditions—Alpha-1 Antitrypsin Deficiency and Cystic Fibrosis—affect the function of special immune cells in the lungs called macrophages. Researchers will recruit 220 adults with these conditions to collect blood samples and measure …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: University of Florida • Aim: Knowledge-focused
Last updated Mar 24, 2026 12:02 UTC
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Massive 50,000-Person study unlocks secrets of rare genetic disorder
Knowledge-focused Recruiting nowThis study aims to understand the real-world use and impact of genetic testing for Alpha-1 Antitrypsin Deficiency, a rare inherited condition. It will enroll 50,000 people at risk for the disease to learn about who gets tested, why, and what happens afterward. The research focuse…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: University of Florida • Aim: Knowledge-focused
Last updated Mar 19, 2026 14:55 UTC