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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderRecruitingPhase 12026-10-06NCT07431112
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Subcutaneous Emphysema, alpha 1-antitrypsin deficiency, hereditary disease, liver disorder, lung disorder, pulmonary emphysema, respiratory system disorderRecruitingPhase 1 / Phase 22026-10-05NCT06389877
Long-term Follow-up of Participants Dosed With an Investigational Gene Editing Therapy for Cardiovascular DiseaseArterial Occlusive Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dyslipidemias, Hypercholesterolemia, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Nutritional and Metabolic Diseases, arteriosclerosis disorder, atherosclerosis, atherosclerotic cardiovascular disease, cardiovascular disorder, familial hypercholesterolemia, hereditary disease, hyperlipidemia, hyperlipoproteinemia, inborn errors of metabolism, metabolic disease, vascular disorderEnrolling by invitationNot stated2026-04-08NCT06112327