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CGT-103
- Development stage
- Preclinical
- Lead developer
- CavGene Therapeutics
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies
01
Overview
CGT-103 is a preclinical RNA interference (RNAi) gene therapy developed by CavGene Inc. in partnership with Iron Horse Diagnostics (nVector). The drug is designed to target and knockdown the expression of the Cav1.3 voltage-gated L-type calcium channel (encoded by the CACNA1D gene) in the striatum. This approach aims to address levodopa-induced dyskinesias (LID) in patients with Parkinson's disease by normalizing aberrant calcium signaling that contributes to motor complications. By utilizing a viral vector to deliver RNAi, CGT-103 represents a targeted genetic approach to managing the side effects of long-term Parkinson's therapy.
02
Targets
CACNA1D (Voltage-dependent L-type calcium channel subunit alpha-1D)
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