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ametefgene parvec
- Development stage
- Phase 2
- Lead developer
- Bayer
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies
- Administration
- Intraparenchymal
01
Overview
Ametefgene parvec (AB-1005) is an investigational adeno-associated virus serotype 2 (AAV2) gene therapy developed by AskBio, a subsidiary of Bayer, for the treatment of Parkinson's disease. It is designed to deliver the human glial cell line-derived neurotrophic factor (GDNF) gene directly into the putamen of the brain via MRI-guided convection-enhanced delivery. GDNF is a potent neurotrophic factor that promotes the survival and phenotypic maintenance of dopaminergic neurons, which are the primary cells lost in Parkinson's disease. By providing sustained expression of GDNF, the therapy aims to slow disease progression and improve motor function in patients with moderate-stage disease. It is currently being evaluated in Phase II clinical trials.
- Other names
- AAV2-GDNFAAV-2-GDNFAAV 2-GDNF
02
Targets
GFRA1 (Glial cell line-derived neurotrophic factor family receptor alpha 1)
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