Early-phase Study of ART002g1 Injection in HeFH: Safety, Tolerability and Preliminary Efficacy

Early-phase Clinical Study on Safety, Tolerability and Preliminary Efficacy of ART002g1 Injection in the Treatment of Heterozygous Familial Hypercholesterolemia

This study is an open-label, single ascending dose (SAD) study designed to evaluate the safety and tolerability of ART002g1 in patients with heterozygous familial hypercholesterolemia (HeFH) who require further reduction in low-density lipoprotein cholesterol (LDL-C). ART002g1 uses base editing technology, which is designed to interfere with the expression of the PCSK9 gene in the liver, thereby reducing the circulating levels of PCSK9 and LDL-C. The primary objectives of this study are to determine the safety and pharmacodynamic (PD) profiles of ART002g1 in this patient population.

Study Overview

• Status: Not yet recruiting
• Conditions: Heterozygous Familial Hypercholesterolemia
• Intervention / Treatment: Drug: ART002g1 Injection

• Study Type: Interventional
• Enrollment (Estimated): 24
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Xueying Ding, MD; Phone Number: +86-02136126102; Email: dingxueying@126.com
• Study Contact Backup: Name: Rong Jiang, MD; Phone Number: +86 13795493921; Email: listening39@163.com

Study Locations

• China
  • Shanghai, China
    • Shanghai General Hospital
    • Contact: Xueying Ding, MD; Phone Number: +86-021-; Email: dingxueying@126.com
    • Contact: Rong Jiang, MD; Email: listening39@163.com
    • Principal Investigator: Xueying Ding, MD
    • Principal Investigator: Rong Jiang, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Subjects must meet all the following criteria to be eligible for enrollment:

1. Male or female, aged 18 to 70 years (inclusive) at the time of signing the Informed Consent Form (ICF);
2. Body weight between 45 and 90 kg (inclusive) at screening;
3. Definite diagnosis of heterozygous familial hypercholesterolemia (HeFH), meeting either of the following two criteria (1) or (2):

（1) HeFH diagnosed to be caused by mutations in the LDLR, APOB, or PCSK9 gene;

(2) Meeting 2 out of the 3 following criteria for adults per the Dutch Lipid Clinical Network (DLCN) criteria:

1. Serum LDL-C ≥ 4.7 mmol/L without prior lipid-lowering treatment;
2. Cutaneous or tendinous xanthomas, or arcus cornealis (in subjects < 45 years old);
3. Presence of FH or early-onset atherosclerotic cardiovascular disease (ASCVD) in first-degree relatives.

Subjects must not be enrolled if they meet any one or more of the following exclusion criteria:

1. Diagnosis of compound heterozygous FH, double heterozygous FH, or homozygous FH (HoFH);
2. Positive for hepatitis B surface antigen (HBsAg), or positive for hepatitis B core antibody (HBcAb) with peripheral blood HBV DNA titer ≥ 1 × 10² copies/L; positive for hepatitis C virus (HCV) antibody with positive peripheral blood HCV RNA; positive for human immunodeficiency virus (HIV) antibody;
3. Any unstable systemic disease, including but not limited to: unstable angina; cerebrovascular accident or transient ischemic attack (within 6 months prior to screening); myocardial infarction (within 6 months prior to screening); history of heart failure (NYHA Class II-IV); severe arrhythmia requiring pharmacotherapy; liver, kidney, or metabolic diseases; or other unstable systemic diseases as determined by the investigator;
4. History of percutaneous transluminal coronary angioplasty (PTCA), percutaneous coronary intervention (PCI), or coronary artery bypass grafting (CABG) within 6 months prior to the first dose; or documented severe coronary artery stenosis as confirmed by coronary CT or coronary angiography within 90 days prior to randomization.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Experimental: ART002g1 Injection - Dose Group 1 ( Single IV Infusion); Participants will receive a single dose of ART002g1.	Drug: ART002g1 Injection; Intravenous (IV) infusion
Experimental: Experimental: ART002g1 Injection - Dose Group 2 (Single IV Infusion); Participants will receive a single dose of ART002g1.	Drug: ART002g1 Injection; Intravenous (IV) infusion
Experimental: Experimental: ART002g1 Injection - Extended Dose Group ( Single IV Infusion); Participants will receive a single Optimal Biological Dose (OBD) of ART002g1, which is based on the results of the ascending dose escalation.	Drug: ART002g1 Injection; Intravenous (IV) infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)	As of Week 48 (W48) post-administration of ART002g1 for Injection

Secondary Outcome Measures

Outcome Measure	Time Frame
Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: Tmax	As of Week 2 (W2) post-administration of ART002g1 for Injection
Pharmacodynamic (PD) Assessments: Serum PCSK9 protein	As of Week 48 (W48) post-administration of ART002g1 for Injection
Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: Cmax	As of Week 2 (W2) post-administration of ART002g1 for Injection
Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: AUC	As of Week 2 (W2) post-administration of ART002g1 for Injection
Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: t½	As of Week 2 (W2) post-administration of ART002g1 for Injection
Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: CL	As of Week 2 (W2) post-administration of ART002g1 for Injection
Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1:Vss	As of Week 2 (W2) post-administration of ART002g1 for Injection
Pharmacodynamic (PD) Assessments: Serum LDL-C	As of Week 48 (W48) post-administration of ART002g1 for Injection

Collaborators and Investigators

• Sponsor: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
• Collaborators: Accuredit Therapeutics US Limited
• Investigators: Principal Investigator: Xueying Ding, MD, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine; Principal Investigator: Rong Jiang, MD, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Study record dates

Study Major Dates

• Study Start (Estimated): March 11, 2026
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): July 1, 2027

Study Registration Dates

• First Submitted: January 12, 2026
• First Submitted That Met QC Criteria: January 19, 2026
• First Posted (Actual): January 20, 2026

Study Record Updates

• Last Update Posted (Actual): March 17, 2026
• Last Update Submitted That Met QC Criteria: March 15, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: HeFH; LNP; ART002g1
• Other Study ID Numbers: IIT-ATMP-2026001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No