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AAV2-GDNF gene therapy
AAV2-GDNF gene therapy delivers a viral vector carrying the GDNF gene to the brain, where it is expressed and promotes the survival and growth of dopaminergic neurons.
AAV2-GDNF gene therapy delivers a viral vector carrying the GDNF gene to the brain, where it is expressed and promotes the survival and growth of dopaminergic neurons. Used for Parkinson's disease.
At a glance
| Generic name | AAV2-GDNF gene therapy |
|---|---|
| Also known as | Biological |
| Sponsor | Brain Neurotherapy Bio, Inc. |
| Drug class | gene therapy |
| Target | GDNF |
| Modality | Biologic |
| Therapeutic area | Neurology |
| Phase | Phase 2 |
Mechanism of action
The delivered GDNF protein acts as a neurotrophic factor, supporting the health and function of dopaminergic neurons in the brain. This can potentially slow or halt the progression of neurodegenerative diseases such as Parkinson's disease.
Approved indications
- Parkinson's disease
Common side effects
- Injection site reactions
- Flu-like symptoms
- Headache
Key clinical trials
- AAV2-GDNF for Advanced Parkinson s Disease (PHASE1)
- GDNF Gene Therapy for Multiple System Atrophy (PHASE1)
- A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD) (PHASE2)
- GDNF Gene Therapy for Parkinson's Disease (PHASE1)
- Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease (PHASE1, PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- AAV2-GDNF gene therapy CI brief — competitive landscape report
- AAV2-GDNF gene therapy updates RSS · CI watch RSS
- Brain Neurotherapy Bio, Inc. portfolio CI