ReGenX

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
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

1Source:  Gene Therapy Clinical Trials Worldwide, provided by the Journal of Gene Medicine