CRSP-CVD-400
Research type
Research Study
Full title
A Phase 1 Open-label, Multicenter, First-in-human, Ascending Single-dose Study Evaluating the Safety and Tolerability of a Lipid Nanoparticle Formulation of CRISPR–Guide RNA–Cas9 Nuclease (CTX310) for In Vivo Editing of the Angiopoietin-like 3 (ANGPTL3) Gene in Subjects With Refractory Dyslipidemias
IRAS ID
1010804
Contact name
Laurie Kelliher
Contact email
Sponsor organisation
CRISPR Therapeutics AG
Research summary
Dyslipidaemia is a condition of unhealthy level of lipids (fats) in the blood. Lipids are waxy, fatty particles that move in the blood. There are different types of blood lipids, such as bad cholesterol (LDL-C), triglycerides and good cholesterol (HDL-C). High levels of blood lipids, especially LDL-C and triglycerides, can lead to build-up of these particles, which can create plaques in the blood vessels. Plaques can lead to heart disease, stroke, and other serious health conditions. For many individuals, diet, exercise, and medications can help lower lipids, but for a significant population who may or may not have an underlying genetic disorder, diet, exercise, and available treatment options are not sufficient to lower blood lipids to a healthy range. The therapy being investigated in this study, CTX310, is a medication developed by CRISPR Therapeutics AG. CTX310 may be able to help people who have not responded to lifestyle changes and currently available treatments to lower the lipid levels. CTX310 works through a process called ‘gene editing’ and is designed to block the production of a protein called ANGPTL3 by disrupting the related gene. ANGPTL3 is made in the liver and controls the breakdown of cholesterol and triglyceride (lipids). The reduction of this protein is expected to reduce lipid levels in the blood. This is a Phase 1 open-label, first-in-human, ascending single dose study of CTX310 in patients with dyslipidaemia that have not responded adequately to the currently available treatments. Participants will receive a one-time infusion of CTX310 and be closely monitored to learn if CTX310 is safe in people and can help treat dyslipidaemias. The study will include a total of approximately 24 participants between the ages of 18 to 75 years in Canada, Australia, New Zealand and the United Kingdom.
REC name
North East - York Research Ethics Committee
REC reference
24/NE/0214
Date of REC Opinion
20 Feb 2025
REC opinion
Further Information Favourable Opinion