Drug intelligence / Profile preview

CGT-102

Development stage
Preclinical
Lead developer
CavGene Therapeutics
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Parenteral
01

Overview

CGT-102 is a preclinical-stage RNA interference (RNAi) gene therapy developed by CavGene Inc. in collaboration with N-Vector and Iron Horse Diagnostics. It is designed to treat levodopa-induced dyskinesia (LID), a common motor complication in patients with Parkinson's disease. The therapy specifically targets and knocks down the expression of the Cav1.3 voltage-gated L-type calcium channel (encoded by the CACNA1D gene) in the striatum. Research indicates that the upregulation of Cav1.3 channels contributes to the maladaptive synaptic plasticity responsible for dyskinesia. By utilizing a viral vector to deliver RNAi triggers, CGT-102 aims to restore normal calcium signaling and alleviate dyskinetic movements without compromising the motor benefits of levodopa therapy.

02

Targets

CACNA1D (Voltage-dependent L-type calcium channel subunit alpha-1D)

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