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GC310
- Development stage
- Unknown
- Lead developer
- GeneCradle
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
GC310 is an **AAV-based gene therapy** designed to treat Wilson's disease by delivering a functional, truncated copy of the ATP7B gene using an adeno-associated virus serotype 5 (AAV5) vector. This gene therapy aims to restore ATP7B protein function in hepatocytes, promoting copper homeostasis and potentially correcting the metabolic defect underlying Wilson's disease. The therapy is under clinical investigation to assess its safety, tolerability, and efficacy, including changes in copper metabolism biomarkers and hepatic/neurological function. The drug is in Phase I/II clinical trials and has shown good preclinical safety and efficacy profiles[1][2][3][4][5].
- Brand names
- GC310GC-310GC 310
- Other names
- AAV5-ATP7BAAV-5-ATP7BAAV 5-ATP7BGC-310GC310GC 310
02
Targets
ATP7B (Copper ion transporter ATPase 7B)
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