Early Human Trial of Gene Editing Targeting ANGPTL3

A total of 15 participants received CTX310, with no dose-limiting toxic effects observed.

• Serious adverse events occurred in two participants (13%), including one death 179 days after treatment.
• Infusion-related reactions were reported in three participants (20%).
• One participant with elevated aminotransferases experienced a transient increase that peaked on day 4 and returned to baseline by day 14.
• The mean percent change in ANGPTL3 levels varied with dosage, showing a reduction of -79.7% at 0.7 mg per kilogram and -73.2% at 0.8 mg per kilogram.

Simplified