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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)Parkinson disease, basal ganglia disorder, brain disorder, central nervous system disorder, movement disorder, nervous system disorder, neurodegenerative disease, parkinsonian disorder, synucleinopathyActive, not recruitingPhase 22026-10-01NCT06285643
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Disorders, Atrophic, autosomal recessive limb-girdle muscular dystrophy type 2A, autosomal recessive limb-girdle muscular dystrophy type 2I, hereditary disease, limb-girdle muscular dystrophy, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseActive, not recruitingPhase 1 / Phase 22026-08-26NCT05230459
A Study of AB-1005 Gene Therapy in Japanese Adults With Moderate Parkinson's Disease (J-REGENERATE-PD)Parkinson disease, basal ganglia disorder, brain disorder, central nervous system disorder, movement disorder, nervous system disorder, neurodegenerative disease, parkinsonian disorder, synucleinopathyNot yet recruitingPhase 22026-07-29NCT07566429
Prescriptive Infusion Algorithm (PIA)Parkinson disease, basal ganglia disorder, brain disorder, central nervous system disorder, movement disorder, nervous system disorder, neurodegenerative disease, parkinsonian disorder, synucleinopathyNot yet recruitingPhase 22026-07-27NCT07215403
A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, disorder of glycogen metabolism, glycogen storage disease II, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, nervous system disorder, neuromuscular diseaseRecruitingPhase 1 / Phase 22026-07-20NCT07282847
Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects With Non-Ischemic NYHA Class III Heart Failurecardiovascular disorder, congestive heart failure, heart disorder, heart failureActive, not recruitingPhase 22025-12-03NCT05598333
AB-1005 Long-Term Follow-up StudyParkinson disease, autonomic nervous system disorder, basal ganglia disorder, brain disorder, central nervous system disorder, movement disorder, multiple system atrophy, nervous system disorder, neurodegenerative disease, parkinsonian disorder, primary dysautonomia, synucleinopathyEnrolling by invitationNot stated2025-10-02NCT07081841
GDNF Gene Therapy for Parkinson's DiseaseParkinson disease, basal ganglia disorder, brain disorder, central nervous system disorder, movement disorder, nervous system disorder, neurodegenerative disease, parkinsonian disorder, synucleinopathyActive, not recruitingPhase 12025-09-19NCT04167540