A Phase 1 Study of AIR-001 in Adults with AATD: RepAIR1
Research type
Research Study
Full title
Phase 1, Open-Label, Single Ascending Dose and Multiple Dose Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered AIR-001 in Adults with Alpha-1 Antitrypsin Deficiency (AATD) Due to PiZZ Genotype
IRAS ID
1013220
Contact name
Kristina Storey
Contact email
Sponsor organisation
AIRNA Corporation
Research summary
Alpha-1 antitrypsin deficiency (AATD) is an inherited condition which affects the liver and lungs and may cause them to not function properly over time. AATD results from several possible genetic mutations to the SERPINIA1 gene, leading to a reduction in the amount of functioning Alpa-1 antitrypsin (AAT) protein in the blood. A working AAT protein is important to the proper function of the liver and lungs and the most common mutation to the SERPINA1 gene is known as PiZZ.
This research study will investigate a new research drug (called AIR-001) to see how safe it is and how well the body tolerates it. This is the first time AIR-001 has been given to humans (known as a Phase I study). AIR-001 may help people with AATD by restoring the working AAT protein.
In the first part of the study, approximately 12 participants will be given increasing doses of AIR-001 and will be monitored for any side effects. AIR-001 will be given as an injection under the skin. Blood samples will be taken to look at how AIR-001 is distributed and metabolised within the body and to look at what effects it has. Participants will also have other assessments to look at the effects of AIR-001.
In the next part of the study, additional participants will be given a dose of AIR-001 over an extended period of time to further assess the safety of AIR-001, and the effects it has on the body.
Up to 54 participants in total are expected to be enrolled into this study. Those in the first part will be in the study for approximately 13 months; those in the second part will be in the study for approximately 17 months.
This study is running at a number of hospitals within the UK, as well as other countries. The results of this study will support the development of AIR-001 as a possible treatment for those with AATD.
REC name
London - Chelsea Research Ethics Committee
REC reference
26/LO/0017
Date of REC Opinion
26 Feb 2026
REC opinion
Further Information Favourable Opinion