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AAV9.NPC.hNPC1
- Development stage
- Preclinical
- Lead developer
- University College London
- Modality
- Gene Therapies
- Administration
- Intracerebroventricular
01
Overview
AAV9.NPC.hNPC1 is an adeno-associated virus serotype 9 (AAV9) gene therapy candidate designed for the treatment of Niemann-Pick type C1 (NP-C) disease. It utilizes a novel truncated endogenous NPC promoter to drive the expression of the human NPC1 (hNPC1) gene, aiming to restore the function of the NPC1 protein, which is deficient in patients with this lethal neurovisceral lysosomal storage disorder. The therapy is intended for intracerebroventricular (ICV) administration to target the progressive neurodegeneration characteristic of the disease. Pre-clinical studies conducted by researchers at University College London (UCL) have demonstrated that the vector significantly extends survival and improves behavioral and neuropathological outcomes in mouse models, even when administered after the onset of disease pathology. The program is currently in pre-clinical development, with efforts focused on manufacturing scale-up and biomarker identification to support future clinical trials.
- Other names
- AAV9-hNPC1AAV-9-hNPC1AAV 9-hNPC1
02
Targets
N-glycan (Cell-surface N-linked galactose-containing glycan)
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