This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are refractory to available treatments.
Age of ≥18 and ≤75 years at the time of signing the informed consent.
Able to provide written informed consent.
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.
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
Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply.
Abnormal or compromised function of kidney, heart, blood or liver.
Acute coronary syndrome event or stroke within 24 weeks prior to Day 1. Acute pancreatitis within 12 weeks prior to Day 1.
Current use or use within 365 days from Day 1 of any hepatocyte-targeted small interfering RNA (except inclisiran).
CRISPR Therapeutics AG
Lead sponsor