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MechanismHBB gene modulators |
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Inactive Indication- |
Drug Highest PhasePhase 1/2 |
First Approval Ctry. / Loc.- |
First Approval Date- |
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Inactive Indication- |
Drug Highest PhasePhase 1/2 |
First Approval Ctry. / Loc.- |
First Approval Date- |
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Inactive Indication- |
Drug Highest PhaseEarly Phase 1 |
First Approval Ctry. / Loc.- |
First Approval Date- |
Study of AAV-hSMN1 (Vesemnogene Lantuparvovec) Gene Therapy in Subjects With Progressive Spinal Muscular Atrophy
This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.
An Open-label Clinical Trial of Ex Vivo Beta-globin Lentiviral Vector Transduction of Autologous CD34+HSPCs (Vebeglogene Autotemcel) for the Treatment of Transfusion Dependent Beta-thalassemia Patients
This is an interventional study to evaluate the safety and efficacy of autologous Hematopoietic Stem and Progenitor Cells (HSPCs) transduced with lentiviral vector encoding functional hemoglobin subunit beta (HBB) gene in patients with transfusion-dependent beta-thalassemia.
100 Clinical Results associated with
0 Patents (Medical) associated with
100 Deals associated with
100 Translational Medicine associated with