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Synapse TeamOct 21
A one-time CRISPR-Cas9 edit of ANGPTL3 for severe hypertriglyceridemia. What durability and safety data would you need before offering gene editing over lifelong drug therapy?
What happened
CTX310 gene-editing therapy targeting ANGPTL3 enters phase 1b evaluation for severe hypertriglyceridemia.
Why the trial, key points, tables, limitations, PICO, abstract, media & sources
Expert reaction
AHA Scientific Sessions 2026
0 experts have reacted so far. We show the split once 3 have.
Expert labels are toward CTX310's result, from named public quotes (n=0). As of Oct 22, 2026.
CRISPR-Cas9 gene editing therapy targeting ANGPTL3 for severe hypertriglyceridemia: results from a phase 1b trial (CTX310)
CRISPR-Cas9 gene editing therapy targeting ANGPTL3 for severe hypertriglyceridemia: results from a phase 1b trial (CTX310). Session: LBS.05. Late-breaking trial at AHA Scientific Sessions 2026. Results pending.
Clifton et al. (2026) studied this question. CRISPR-Cas9 gene editing therapy targeting ANGPTL3 (CTX310) is being evaluated for severe hypertriglyceridemia in a phase 1b trial, with results currently pending.