CTX310 is an investigational in vivo CRISPR-Cas9 gene-editing therapy developed by CRISPR Therapeutics for the treatment of severe or refractory dyslipidemia. CTX310 uses a lipid nanoparticle-delivered CRISPR-Cas9 system to introduce a loss-of-function edit in the ANGPTL3 gene in hepatocytes following a single intravenous infusion, aiming to durably lower triglycerides and LDL cholesterol.
Disease: Refractory Dyslipidemias (NCT07491172)
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Candidate summary:
Disease/Disease category:
- Endocrine, nutritional or metabolic diseases
Official title:
A Phase 1 Open-label, Multicenter, First-in-human, Ascending Dose Trial 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 Participants With Refractory Dyslipidemias
Phases:
- Phase 1
Status:
Active recruiting
Country:
- United States
- United Kingdom
- Australia
- New Zealand
