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AAV2-PEDF
- Development stage
- Preclinical
- Lead developer
- Universitat Autònoma de Barcelona
- Modality
- Gene Therapies
- Administration
- Intravitreal, Intratumoral
01
Overview
AAV2-PEDF is an investigational recombinant adeno-associated virus serotype 2 (AAV2) gene therapy vector encoding human pigment epithelium-derived factor (PEDF). PEDF is a secreted glycoprotein belonging to the serpin family that exhibits potent anti-angiogenic, neuroprotective, and anti-inflammatory activities. Following local administration (such as intravitreal injection), the vector transduces retinal cells to provide sustained, long-term expression of PEDF. This local production of PEDF inhibits pathological neovascularization, normalizes capillary density, and protects retinal ganglion cells from degeneration. Preclinical development has been primarily conducted by academic institutions, including the Universitat Autònoma de Barcelona, for the treatment of diabetic retinopathy, wet age-related macular degeneration, and glaucoma, as well as intratumoral delivery in solid tumor models.
- Other names
- AAV2-CBA-PEDFAAV-2-CBA-PEDFAAV 2-CBA-PEDFAAV2-hPEDFAAV-2-hPEDFAAV 2-hPEDFAAV2.PEDFAAV-2.PEDFAAV 2.PEDF
02
Targets
VEGFR-1 (Vascular endothelial growth factor receptor 1)
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