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Gene Therapy Trial Report

Summary

A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias


NCTID NCT07491172 (View at clinicaltrials.gov)
Description
Development Status 🔄 Active
Indication 🔄 Hypertriglyceridemia, Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Uncontrolled Hypercholesterolemia, Mixed Dyslipidemia
Disease Ontology Term 🔄 DOID:13810; DOID:13809; DOID:3146
Drug Product Name 🔄 CTX310
Sponsor CRISPR Therapeutics AG
Funder Type Industry
Recruitment Status
Enrollment Count 90 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant 🔄 ANGPTL3
Therapeutic Modality 🔄 Gene editing
Therapy Route 🔄 in vivo
Mechanism of Action 🔄 Gene inactivation
Route of Administration 🔄 Intravenous
Drug Product Type 🔄 Encapsulated mRNA
Target Tissue/Cell 🔄 Hepatocytes
Gene Delivery System Type 🔄 Endocytosis
Non-Viral Vector Type LNP (LDLR)
Editor Type 🔄 Cas9 mRNA
Dose 1 🔄 Phase 1: 0.1 mg/kg (n=3); 0.3 mg/kg (n=3), 0.6mg/kg (n=3), 0.7 mg/kg (n=2), 0.8 mg/kg (n=4)

Study Record Dates


Current Phase Phase1
Submit Date 2026-03-18
Completion Date 2028-06
Last Update 🔄 2026-07-23

Participation Criteria


Eligible Age 18 Years - 75 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Key Inclusion Criteria: 1. Age of ≥18 and ≤75 years at the time of signing the informed consent. 2. Able to provide written informed consent. 3. Participants diagnosed with persistent dyslipidemias defined by TG ≥150 mg/dL - and LDL-C ≥70 mg/dL in participants with ASCVD, or LDL-C ≥70 or 100mg/dL in participants with or without ASCVD respectively, or TG ≥500 mg/dL. 4. Refractory to the maximal intensity or MTD of standard of care lines of lipid-lowering therapies available through routine clinical care, for at least 12 weeks prior to screening 5. Female participants must be postmenopausal or surgically sterile. 6. All male participants and their female partners must agree to the use of an acceptable method of effective contraception for the duration of the study. Exclusion Criteria: 1. Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply. 2. Evidence of liver disease, defined as but not limited to: LFTS \>2 × upper limit of normal (ULN), or total bilirubin \>2 × ULN, or INR \>1.5 × ULN, or liver stiffness measured by liver elastography 3. Abnormal or compromised function of kidney, heart, blood or liver. 4. Acute coronary syndrome event or stroke within 24 weeks prior to Day 1. Acute pancreatitis within 12 weeks prior to Day 1. 5. Current use or use within 365 days from Day 1 of any hepatocyte-targeted small interfering RNA (except inclisiran). 6. Positive serology for HIV, hepatitis B or hepatitis C (antibody, surface antigen orNAT). Serology consistent with prior immunization will be eligible for the trial. 7. Any prior malignancy within the past 5 years, or current malignancy (exceptions for resected or removed basal cell carcinoma, squamous cell carcinoma in situ and carcinoma in situ of the cervix or breast). 8. Women of childbearing potential. Note: Other protocol defined Inclusion/Exclusion criteria may apply. Note: The inclusion and exclusion criteria listed represent the global protocol. Additional or modified eligibility criteria may apply in certain countries in accordance with local regulatory and ethics committee requirements and the approved country-specific protocol.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 🔄 19
Locations New Zealand,United States,United Kingdom,Australia

Regulatory Information


Has US IND True
FDA Designations
Recent Updates IND was cleared in 1Q26

Resources/Links