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AAV2CUhCLN2
- Development stage
- Phase 1
- Lead developer
- Weill Cornell Medicine
- Modality
- Gene Therapies
- Administration
- Intracerebral
01
Overview
AAV2CUhCLN2 is a gene therapy vector designed for the treatment of Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL), a fatal childhood neurodegenerative lysosomal storage disease. The vector consists of an adeno-associated virus serotype 2 (AAV2) capsid containing a modified genome with the human CLN2 cDNA, which encodes tripeptidyl peptidase I (TPP-I), the enzyme deficient in LINCL patients. It is administered directly to the central nervous system to provide persistent expression of TPP-I in neurons, aiming to prevent further neurodegeneration and slow disease progression.
- Other names
- AAV2CUhCLN2AAV-2CUhCLN2AAV 2CUhCLN2
02
Targets
TPP1 (Tripeptidyl peptidase 1)
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