Also known as: AATD
Alpha-1 antitrypsin deficiency is an inherited disorder caused by SERPINA1 variants that reduce functional alpha-1 antitrypsin and increase risk of early emphysema/COPD and liver disease. Clinical burden varies by genotype and environmental exposures such as smoking.
Canaccord Genuity initiated Beam Therapeutics with a buy rating and $74 price target, highlighting near-term catalysts. Beam reached an agreement with the FDA on an accelerated approval pathway for its BEAM-302 AATD therapy, which showed early clinical proof-of-concept for in vivo base editing. The company plans a BLA for risto-cel and a pivotal plan for BEAM-302 in early 2026.
Krystal Biotech posted Q4 2025 net revenue of $107.1 million from Vyjuvek sales, with full-year revenue reaching $389.1 million. The company expanded to over 90 patients across Germany, France, and Japan while advancing its pipeline with new FDA designations.
YolTech Therapeutics received FDA approval for its IND application for YOLT-202, an in vivo gene-editing therapy for Alpha-1 Antitrypsin Deficiency. Interim data from an ongoing trial showed rapid increases in AAT levels to normal range with favorable safety.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT06996756 |
Gene Therapy for Alpha 1- Antitrypsin Deficiency |
RECRUITING | PHASE1 |
| NCT06738017 |
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants |
ACTIVE_NOT_RECRUITING | PHASE1 |
| NCT06505603 |
PiMZ Longitudinal Cohort (PiMZ Logic) |
RECRUITING | |
| NCT06389877 |
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) |
RECRUITING | PHASE1/PHASE2 |
| NCT06049082 |
A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency |
RECRUITING | PHASE1 |
| NCT05897424 |
Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT05856331 |
Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema |
COMPLETED | PHASE2 |
| NCT05687474 |
Baby Detect : Genomic Newborn Screening |
COMPLETED | |
| NCT05579431 |
A Phase 1, First-in-human Study of VX-634 |
COMPLETED | PHASE1 |
| NCT05297812 |
Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease |
ACTIVE_NOT_RECRUITING |
