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.

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Conditions

The condition(s) this trial relates to.

alpha 1-antitrypsin deficiency Congenital Abnormalities hereditary disease Infant, Newborn, Diseases liver disorder lung disorder Pathologic Processes Pathological Conditions, Signs and Symptoms pulmonary emphysema respiratory system disorder Subcutaneous Emphysema

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Clinical Study Center

    RECRUITING

    Melbourne, Australia

  • Clinical Study Center

    RECRUITING

    Cambridge, United Kingdom

  • Clinical Study Center

    RECRUITING

    London, United Kingdom

  • Clinical Study Center

    RECRUITING

    Southampton, United Kingdom

  • Clinical Study Center 1

    RECRUITING

    Tbilisi, Georgia

  • Clinical Study Center 2

    RECRUITING

    Tbilisi, Georgia

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