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GT0009X

Development stage: Preclinical
Lead developer: ONODERA GT Pharma
Modality: Gene Therapies

Overview

GT0009X is an adeno-associated virus (AAV) gene therapy under development by ONODERA GT Pharma for the treatment of Niemann-Pick disease type C (NPC). The therapeutic approach involves the delivery of a functional NPC1 gene to compensate for the mutations in the endogenous NPC1 gene, which typically result in defective cholesterol transport and lysosomal storage issues. By restoring the expression of the NPC1 protein, GT0009X aims to halt or reverse the progression of neurological and systemic manifestations of the disease. The program is currently in the preclinical stage.

Targets

NPC1 (Niemann-Pick C1 protein binding inhibition)

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