AHA Conference 2024 - Safety and efficacy outcomes of nexiguran ziclumeran (NTLA-2001) in patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM).

Dr Marianna Fontana (University College London, London, UK) joins us onsite at AHA Conference to discuss the findings from the nexiguran ziclumeran for ATTR-CM trial (NCT04601051).

Dr Fontana discusses a phase 1, ongoing, open-label, single-arm trial investigating the use of NTLA-2001 using CRISPR-based technology in patients with ATTR-CM. 36 patients were enrolled in the trial and received an intravenous infusion of NTLA-2001. The primary outcome measure assessed the safety and tolerability of NTLA-2001, including the serum transthyretin level.

Interview Questions:
1.     What is the importance behind the study?
2.    Could you tell us about the unique features of the CRISPR gene-editing system?
3.    What was the study design and patient population?
4.    What were the key findings?
5.    What further research is needed in this area, and what are the next steps?

Recorded on-site at AHA Conference in Chicago, 2024.

Editors: Yazmin Sadik, Jordan Rance.
Videographers: Mike Knight, Dan Brent, Oliver Miles, Tom Green, David Ben-Harosh.

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