Last update 09 Feb 2026

GC-310

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
GC 310, GC310
Target
ATP7B
Action
stimulants
Mechanism
ATP7B gene stimulants(ATPase copper transporting beta gene stimulants)
Therapeutic Areas
Nervous System DiseasesCongenital DisordersDigestive System Disorders+ [2]
Active Indication
Hepatolenticular DegenerationNeuromuscular Diseases
Inactive Indication-
Originator Organization
Beijing GeneCradle Technology Co., Ltd.Beijing GeneCradle Technology Co., Ltd.
Active Organization
Beijing GeneCradle Technology Co., Ltd.Beijing GeneCradle Technology Co., Ltd.
Inactive Organization-
License Organization-
Drug Highest PhasePhase 1/2
First Approval Date-
RegulationOrphan Drug (United States)
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Related

2
Clinical Trials associated with GC-310
ChiCTR2500113213
/ Not yet recruitingPhase 1/2IIT
Multicenter, open, single-dose, dose escalation phase I/II clinical trial to evaluate the safety, tolerance and efficacy of GC310 adeno-associated virus injection in the treatment of Wilson disease patients
NCT07173933
/ Not yet recruitingPhase 1/2
A Multicenter, Open-label, Single-dose, Dose-escalation Phase I/II Clinical Trial Evaluating the Safety, Tolerability, and Efficacy of GC310 Adeno-associated Virus Injection in the Treatment of Patients With Wilson's Disease (WD)
100 Clinical Results associated with GC-310
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100 Translational Medicine associated with GC-310
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100 Patents (Medical) associated with GC-310
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100 Deals associated with GC-310
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Hepatolenticular DegenerationPhase 2
China
Beijing GeneCradle Technology Co., Ltd.Beijing GeneCradle Technology Co., Ltd.
01 Oct 2025
Neuromuscular DiseasesDiscovery
China
Beijing GeneCradle Technology Co., Ltd.Beijing GeneCradle Technology Co., Ltd.
-
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
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Deal

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Core Patent

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Clinical Trial

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Approval

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Regulation

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