Beijing Genecradle Therapeutics Co., Ltd. (hereinafter referred to as "Genecradle Therapeutics") has initiated patient recruitment for the Phase I/II clinical trial of its independently developed AAV gene therapy drug, GC310 Adeno-Associated Virus Injection, for the treatment of Wilson disease (WD). The trial plans to enroll 10-15 adult patients with Wilson disease.

This study is a multicenter, open-label, single-dose, dose-escalation clinical trial. It has been approved by the Ethics Committee of Peking Union Medical College Hospital, the leading institution, and will be launched first, with other research centers to follow.

 Project Introduction

GC310 Adeno-Associated Virus Injection is an AAV gene therapy drug independently developed by Genecradle Therapeutics for the treatment of Wilson disease. This therapy precisely delivers a functional miniATP7B gene, enabling the target tissue to express the biologically active miniATP7B copper ion transporter protein through a single treatment, restoring copper ion metabolism and increasing ceruloplasmin levels, potentially improving the condition fundamentally. In preclinical studies, GC310 Injection has demonstrated good drug safety and significant efficacy. Experimental results show that the drug can effectively reduce liver and urinary copper levels in Wilson disease mouse models, increase ceruloplasmin activity, and significantly improve symptoms.

The gene therapy has received tacit approval for registration of clinical trials from the National Medical Products Administration and Orphan Drug Designation from the FDA.

 Recruitment Information from Peking Union Medical College Hospital

Peking Union Medical College Hospital will soon conduct a "Multicenter, Open-Label, Single-Dose, Dose-Escalation Phase I/II Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of GC310 Adeno-Associated Virus Injection in Patients with Wilson Disease (WD)." The hospital is now publicly recruiting participants. If you meet the following main criteria and are interested in participating, please consult the clinical physician.

【Project Introduction】

This trial is applicable to adult patients diagnosed with Wilson disease (WD). The trial has obtained tacit approval from the Center for Drug Evaluation (CDE), with the clinical trial application number CXSL2400774, and has been approved by the Ethics Committee of Peking Union Medical College Hospital. The trial drug is not yet marketed.

【Eligibility Criteria for Participants】

The study plans to recruit a total of 10-15 subjects, with the main inclusion criteria as follows:

1. At the time of signing the informed consent form, the subject is ≥18 years old, regardless of gender;

2. Diagnosed with Wilson disease (WD);

3. Serum ceruloplasmin concentration <100 mg/L;

4. Willing and able to comply with all study protocol requirements and procedures, and voluntarily participate and sign the informed consent form.

【Contact Person and Contact Information】

Contact Person: Dr. Sun, Phone: +86-13691577626

【Institution Name and Address】

Institution Name: Peking Union Medical College Hospital

Institution Address: No. 1 Shuai Fu Yuan, Dongcheng District, Beijing, Peking Union Medical College Hospital

 Recruitment Poster