CRISPR Therapeutics to Unveil Late-Breaking CTX310 Data at AHA 2026…

Event summary

CRISPR Therapeutics will present Phase 1b trial data for CTX310, its CRISPR/Cas9 in vivo gene editing therapy targeting ANGPTL3, at the AHA Scientific Sessions 2026.
The presentation, scheduled for November 9, 2026, focuses on severe hypertriglyceridemia treatment.
CTX310 is part of CRISPR's in vivo liver editing portfolio, which includes programs for cardiovascular diseases and rare conditions.
CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) delivery platform for liver gene editing.

The big picture

CRISPR Therapeutics is advancing its leadership in CRISPR-based therapies with the presentation of late-breaking data for CTX310. This follows the approval of CASGEVY, the world’s first CRISPR-based therapy, and underscores the company’s pivot from research-stage to commercial-stage biopharmaceutical. The data could validate CRISPR’s in vivo gene editing approach, potentially opening new avenues for treating metabolic and cardiovascular diseases.

What we're watching

Clinical Efficacy: The effectiveness of CTX310 in reducing triglycerides and its potential to address severe hypertriglyceridemia will be closely watched.
Regulatory Pathway: The data could influence the regulatory strategy for CTX310 and similar gene editing therapies.
Competitive Positioning: CRISPR Therapeutics' progress in cardiovascular gene editing will be compared to competitors in the space.