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NV-101
- Development stage
- Preclinical
- Lead developer
- nVector
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Injection
01
Overview
NV-101 is an adeno-associated virus (AAV) gene therapy candidate developed by nVector for the treatment of Amyotrophic Lateral Sclerosis (ALS). It utilizes a proprietary AAV capsid, designated AV-01, which is engineered for superior efficacy and diffusion within the central nervous system compared to standard vectors. The therapy is designed to target cells in the brain and spinal cord, inducing the localized expression of Glial Cell Line-Derived Neurotrophic Factor (GDNF). GDNF is a potent neurotrophic factor that provides critical survival signals to motor neurons, potentially slowing or halting the progressive neurodegeneration characteristic of ALS. NV-101 is based on nVector's GreenGlia Technology platform.
- Other names
- NV-101-nVector-GDNFNV101-nVector-GDNFNV 101-nVector-GDNFAV-01-GDNFAV01-GDNFAV 01-GDNF
02
Targets
GFRA1 (Glial cell line-derived neurotrophic factor family receptor alpha 1)
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