CRISPR Therapeutics Announces Positive Phase 1 Clinical Data for CTX310® Demonstrating Deep and Durable ANGPTL3 Editing, Triglyceride and Lipid Lowering

CRISPR Therapeutics Announces Positive Phase 1 Clinical Data for CTX310® Demonstrating Deep and Durable ANGPTL3 Editing, Triglyceride and Lipid Lowering -Data presented in a late-breaking presentation at the American Heart Association (AHA) Scientific Sessions 2025- https://ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-announces-positive-phase-1-clinical-data A Glimpse Into the Future: Gene Editing for LDL Cholesterol One of the most groundbreaking moments of AHA 2025 came from the presentation of a first-in-human trial using CRISPR-Cas9 gene editing to target the ANGPTL3 gene — a key regulator of LDL metabolism. A single infusion of CTX310 from CRISPR Therapeutics safely reduced LDL-C by up to 87%, with a mean reduction of 49% at Day 60, suggesting the possibility of a one-time, durable treatment for people with severe or genetic lipid disorders like FH and HoFH. Why it matters: This is early science — but it signals a bold future where LDL control may no longer depend on lifelong medication. For families living with inherited hypercholesterolemia, gene-editing research represents true hope for a cure. The Family Heart Foundation at AHA 2025 https://familyheart.org/family-heart-at-2025-aha-sessions Beyond the science, the Family Heart Foundation played a powerful role on the ground at AHA 2025 — sharing research, elevating patient voices, and advancing awareness of FH and elevated Lp(a). Our presence at AHA included: FIND FH® Initiative: Shani Bardach, Director of Quality and Research Operations, presented “Novel Strategies to FIND People Living with Genetic Dyslipidemias,” highlighting how the Foundation’s Flag, Identify, Network, and Deliver™ (FIND FH) program uses machine learning to find undiagnosed FH in health systems. Complementary work from Emory University showcased how AI-driven outreach helps clinicians detect FH earlier — an essential step in preventing heart disease before it happens. Understanding Gaps in Cholesterol Management and FH Diagnosis: Diane MacDougall, Vice President of Research, presented new data from the Family Heart Database® showing persistent gaps in cholesterol management across the U.S., including disparities by sex, race, and age. Findings from Emory doctoral student Jeffrey Osei reinforced these insights, demonstrating that while FH diagnoses are increasing, there is still progress to be made. Real Stories at the Novartis Expert Theater: Ambassador Brian Farrington shared his lived experience with high Lp(a), bringing humanity to the science and reminding attendees that behind every number is a family and a future we’re working to protect. On the Exhibit Floor: The Family Heart booth was a hub for connection, education, and collaboration — empowering clinicians and advocates alike to learn, partner, and take action for better diagnosis and care. Together, these efforts underscored the Foundation’s leadership in research, advocacy, and community engagement — uniting science and stories to drive change. Read more about our impact and presentations in our recent blog >