New drug AIR-001 enters first human tests for rare lung condition
NCT ID NCT07431112
First seen Jun 26, 2026 · Last updated Jul 24, 2026 · Updated 2 times
Summary
This early-stage trial tests a new drug called AIR-001 in 54 adults with alpha-1 antitrypsin deficiency (AATD) who have a specific genetic type (PiZZ). The study aims to check safety and how the body processes the drug. Participants receive increasing doses of AIR-001 under the skin to find the best and safest levels.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- AIR-001
- What this could lead to
- If successful, this could lead to a new treatment that helps control lung damage in people with alpha-1 antitrypsin deficiency.
- What could go wrong
- This is a very early Phase 1 trial with only 54 people, focused on safety. It is too soon to know if AIR-001 will work or be safe long-term.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Email: •••••@•••••
Locations
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Clinical Study Center
RECRUITINGMelbourne, Australia
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Clinical Study Center
RECRUITINGCambridge, United Kingdom
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Clinical Study Center
RECRUITINGLondon, United Kingdom
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Clinical Study Center
RECRUITINGSouthampton, United Kingdom
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Clinical Study Center 1
RECRUITINGTbilisi, Georgia
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Clinical Study Center 2
RECRUITINGTbilisi, Georgia
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Other studies related to the condition(s) this trial covers.
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- Gene-Editing shot aims to fix lung and liver damage in rare disease
- Alpha-1 patients donate samples to power future discoveries