Beijing Jinlan Gene's GC310 AAV Injection Enters Phase I/II Clinical Trial for Wilson’s Disease

Data from the Drug Clinical Trial Registration and Information Disclosure Platform shows that Beijing Genecradle Therapeutics Co., Ltd.'s Phase I/II clinical trial for GC310 Adeno-Associated Virus Injection to evaluate the safety, tolerability, and efficacy in treating patients with Wilson's Disease (WD) has been initiated. This is a multicenter, open-label, single-dose, dose-escalation study. The clinical trial registration number is CTR20253706, with the initial information disclosure date on September 17, 2025.

The drug formulation is an injection, preset with two dose groups at 3.0E + 13 d.vg/kg and 6.0E + 13 d.vg/kg, administered as a single dose. The primary objective of this trial is to evaluate the safety and tolerability of GC310 intravenous infusion in treating WD patients and to determine the recommended dose for Phase IIb clinical trials; the secondary objective is to assess changes in blood GC310 vector genome copy numbers and immunogenicity following GC310 intravenous infusion treatment in WD patients.

GC310 Adeno-Associated Virus Injection is a biologic product indicated for Wilson's Disease (WD). Wilson's Disease is an autosomal recessive inherited disorder of copper metabolism, where excessive copper accumulation in the body damages the liver, nervous system, and more. Symptoms include abnormal liver function, tremors, dysarthria, etc., and can be diagnosed through tests such as serum ceruloplasmin.

The primary endpoint indicators of this trial include the incidence of adverse events within 12 weeks after GC310 administration and the incidence of DLT events within 4 weeks; secondary endpoint indicators include changes from baseline in multiple indicators, such as serum ceruloplasmin concentration within 52 weeks after GC310 administration, and AAV viral shedding.

Currently, the trial status is ongoing (not yet recruiting).

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Editor: Xiaolang Express