AHA Scientific Sessions 2025 Late-Breaking and Featured Science Collection

  • Published:  05 November 2025
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AHA Scientific Sessions 2025 Late-Breaking and Featured Science Collection

  • Published:  05 November 2025
  • Views: 

    Views Icon

    510

  • Likes: 

    Heart Icon

    2

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About the episode

AHA Scientific Sessions 2025 - Phase 1 results show the CRISPR-Cas9 gene editing therapy is a safe treatment option targeting ANGPTL3 in patients with lipid disorders.

 

Prof Stephen Nicholls (Monash Victorian Heart Institute, Clayton, AU) joins us to share results from a first-in-human phase 1 trial investigating a one-time infusion of CTX310, a CRISPR-Cas9 gene editing therapy, at different doses targeting angiopoietin-like protein 3 (ANGPTL3). 15 patients with high cholesterol levels or high triglyceride levels.

 

Findings showed that within two to four weeks after the one-time infusion of CTX310, LDL cholesterol levels decreased by almost 50% and triglyceride levels decreased by almost 55%. These reduced levels were sustained for at least 60 days of follow-up.

 

Interview Questions:
1. Can you explain the rationale behind targeting the ANGPTL3 gene for patients with lipid disorders?
2. How does this CRISPR-Cas9 therapy differ from existing treatments?
3. What was the study design and patient population?
4. What were the key findings?
5. What are the take-home messages, and what further research is required?

 

Recorded on-site at AHA Conference in New Orleans, 2025.
Editor: Yazmin Sadik.
Video Specialist: David Ben-Harosh and Dan Brent.

Support: This is an independent interview produced by Radcliffe Cardiology.

Overview

Stay at the forefront of cardiovascular medicine with our comprehensive video collection from the American Heart Association's Scientific Sessions 2025. Access expert analysis of the most impactful late-breaking trials and featured science shaping clinical practice.

  • Late-Breaker Discussions: Watch Dr Harriette Van Spall break down pivotal clinical trial data and explore its implications for advancing cardiovascular research and patient care.
  • Expert Interviews: Our focused interview series distills essential insights, featuring key study designs, primary endpoints, and clinical take-home messages from leading faculty presenting at the conference.

More from this programme

Faculty Biographies

Stephen Nicholls

Stephen Nicholls

Monash Victorian Heart Institute Director and Professor of Cardiology

Prof Stephen Nicholls is the Director of the Monash Victorian Heart Institute in Melbourne, AU and a Professor of Cardiology at Monash University. He also serves as the Deputy Director and Heart Health Theme Leader at the South Australian Health and Medical Research Institute, Adelaide, AU.

Prof Nicholls completed his cardiology training at the John Hunter Hospital and received his Doctor of Philosophy (PhD) in Medical Biochemistry from the University of Adelaide.

He has written over 770 manuscripts, book chapters and conference proceedings. His research is focused on strategies to reduce the risk of heart disease, novel vascular imaging and generating an understanding of the factors that promote plaque formation. He has raised over $100 million in research funding and has received multiple awards, including the Cleveland Clinic Innovator Award in 2011, the NHMRC Research Excellence Award in 2016, the RT Hall Price in 2018 and the Eric Susman Prize in…

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