AAV Therapeutic Development
Therapeutic development with ReGenX AAV Vector Technology is underway for a variety of diseases and unmet medical needs. Click on a therapeutic area below for more information on these programs.
| Research | Preclinical | Clinical | Marketed | Vectors | |
| OCULAR DISEASES | |||||
| Age‐Related Macular Degeneration | AAV7 | ||||
| Leber Congenital Amaurosis | AAV7 | ||||
| Retinitis Pigmentosa | AAV8 | ||||
| NEURODEGENERATIVE DISORDERS | |||||
| Amyotrophic Lateral Sclerosis | AAV9 | ||||
| Parkinson’s Disease | AAV9 | ||||
| Alzheimer’s Disease | AAV9 | ||||
| METABOLIC DISORDERS | |||||
| Familial Hypercholesteremia | AAV8 | ||||
| Hurler Syndrome (MPS I) | AAV8 | ||||
| Maroteaux‐Lamy Syndrome (MPS VI) | AAV8 | ||||
| Gaucher Disease | AAV8 | ||||