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AAV-hAADC-2 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| AAV-hAADC-2 (9x10^10 vector genomes) | EXPERIMENTAL | - |
| AAV-hAADC-2 (3x10^11 vector genomes) | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| AAV-hAADC-2 | GENETIC | 9 x 10\^10 vector genomes (vg) of AAV-hAADC-2 in a single dose of 200 µL bilaterally infused over 4 striatal targets |
Inclusion Criteria: * Documented informed consent. * Diagnosis of idiopathic Parkinson's disease (two of four cardinal features - tremor, bradykinesia, rigidity and postural instability - plus early dopaminergic responsiveness of symptoms and absence of any cardinal signs of more widespread neurolo...
AAV-hAADC-2 is an investigational gene therapy being studied for the treatment of Parkinson's Disease. It is designed to deliver a gene that may help produce an enzyme involved in dopamine synthesis, potentially addressing the motor symptoms of the condition. It is currently in clinical development and is not yet approved.
AAV-hAADC-2 is being developed by Sanofi, a multinational pharmaceutical company listed on the stock exchange under the ticker SNY. The company is conducting clinical research to evaluate the safety and efficacy of this gene therapy for Parkinson's Disease.
AAV-hAADC-2 is in Phase 1 clinical development. This early-stage trial is designed to assess the safety, tolerability, and preliminary efficacy of the gene therapy in patients with Parkinson's Disease. The study has been completed, but the drug remains investigational and has not received regulatory approval.
AAV-hAADC-2 has been studied in one clinical trial, identified as NCT00229736, titled 'A Study of AAV-hAADC-2 in Subjects With Parkinson's Disease.' This Phase 1 trial enrolled 10 participants in the United States and has been completed. The study included adults aged 40 years and older.
AAV-hAADC-2 is a gene therapy that uses an adeno-associated virus (AAV) vector to deliver the human aromatic L-amino acid decarboxylase (AADC) gene. This gene encodes an enzyme that converts levodopa to dopamine, potentially enhancing dopamine production in the brain and improving motor function in Parkinson's Disease patients.