CRISPR Therapeutics reports positive Phase 1b CTX310 data
CRISPR Therapeutics presented Phase 1b data for CTX310 on November 9, 2026, showing a 63% mean triglyceride decrease and a 65% mean reduction in ANGPTL3 at Day 30. The study involved 10 people with severe hypertriglyceridemia, and no treatment-related serious adverse events were reported. The FDA has granted CTX310 Regenerative Medicine Advanced Therapy designations for severe hypertriglyceridemia and elevated LDL cholesterol.
Why it matters
The findings provide an early clinical data point for CTX310 as CRISPR Therapeutics continues the Phase 1b study with additional cohorts. The FDA designations may support the therapy's development in both targeted indications, while longer-term effects remain to be established.
Key facts
- 1At Day 30, CTX310 produced mean reductions of 63% in triglycerides and 65% in circulating ANGPTL3 protein from baseline. investing.com
- 2The Phase 1b study included 10 participants, each receiving one intravenous infusion at a dose of 40 mg or 50 mg based on body weight. investing.com
- 3Mean reductions at Day 30 were 51% for non-HDL cholesterol, 23% for apolipoprotein B, and 16% for LDL cholesterol from a mean LDL baseline of 60 mg/dL. investing.com
- 4Seven participants had reached at least 90 days of follow-up by the data cutoff. investing.com
- 5The FDA granted CTX310 Regenerative Medicine Advanced Therapy designations for severe hypertriglyceridemia and elevated LDL cholesterol. investing.com
Open questions
- The materials do not establish CTX310's long-term efficacy or safety profile.
Summary written by AlphAI AI Desk from 2 of 2 sources. Not investment advice. Figures are as stated by the linked sources.