Clinical Trials
Worldwide, there have been over 90 clinical trials using AAV vectors1 which have spanned a wide range of therapeutic areas, including inherited diseases, CNS disorders, heart failure, and vaccines. The trials which have demonstrated at least some efficacy include therapies for lipoprotein lipase deficiency, Parkinson’s disease, heart failure, Leber congenital amaurosis, and hemophilia B.
Clinical Trials Using NAV™ Technology
| Title | Serotype | Disease |
|---|---|---|
| Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis | rAAVrh.10 | Batten Disease/Late Infantile Neuronal Lipofuscinosis (LINCL) |
| Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B | rAAV8 | Hemophilia B |
| Non-Controlled, Non-Randomized Open Label Pilot Trial of Sterotactically Administered Gene Transfer Vector (Rh.10) Containing Heparan Sulphamidase (SGSH) For Treatment of Sanfilippo Type A In Children | rAAVrh.10 | Sanfilippo Type A Syndrome (MPSIIIA) |
| Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy (TG-MLD) | rAAVrh.10 | Metachromatic Leukodystrophy |
Selected Clinical Trials Using Other AAV Vectors
| Rare Diseases: | Serotype | Disease |
|---|---|---|
| Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency | rAAV1 | Alpha-1-antitrypsin deficiency |
| Gene Transfer for Subjects With Hemophilia B Factor IX Deficiency (Transient Immunomodulation) | rAAV2 | Hemophilia B |
| Efficacy and Safety of Human Lipoprotein Lipase (LPL)[S447X] Expressed by an Adeno-Associated Viral Vector in LPL-deficient Subjects | rAAV1 | Lipoprotein Lipase Deficiency |
| Safety Study of Mini-Dystrophin Gene to Treat Duchenne Muscular Dystrophy | rAAV2.5 | Muscular Dystrophy (Duchenne) |
| Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D) | rAAV1 | Muscular Dystrophy (Limb Girdle type 2D; LGMD2D) |
| Phase I clinical trial with an AAV vector and gamma sarcoglycans for treating limb-girdle muscular dystrophy type 2C | rAAV1 | Muscular Dystrophy (Limb Girdle type 2C; LGMD2C) |
| Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease | rAAV1 |
Pompe |
| Retinal Disease | Serotype | Disease |
|---|---|---|
| Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis | rAAV2 | Leber congenital amaurosis (LCA) |
| Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis | rAAV2 | Leber congenital amaurosis (LCA) |
| Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations | rAAV2 | Leber congenital amaurosis (LCA) |
| Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 | rAAV2 | Leber congenital amaurosis (LCA) |
| Safety and Tolerability Study of AAV2-sFLT01 in Patients With Neovascular Age-Related Macular Degeneration (AMD) | rAAV2 | Neovascular Age-Related Macular Degeneration (wet AMD) |
| A Phase I/II controlled, dose-escalating trial to establish the baseline safety and efficacy of a gene therapy product into the eyes of patients with exudative age-related macular degeneration (AMD) | rAAV2 | Neovascular Age-Related Macular Degeneration (wet AMD) |
|
Neurological Diseases: |
Serotype |
Disease |
|---|---|---|
| Randomized, Controlled Study Evaluating CERE-110 in Subjects With Mild to Moderate Alzheimer's Disease | rAAV2 | Alzheimer’s Disease |
| Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease | rAAV2 | Parkinson’s Disease |
| Study of AAV-GAD Gene Transfer Into the Subthalamic Nucleus for Parkinson's Disease | rAAV2 | Parkinson’s Disease |
| Cardiac Disease: | Serotype | Disease |
|---|---|---|
| Efficacy and Safety Study of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure | rAAV1 | Heart Failure |
