Ahmad Masri, MD, MS: Reaching New Milestones in Cardiovascular Gene Therapy Development

Intellia Therapeutics has reached the milestone of developing the first gene therapy in the cardiovascular field to reach phase 3 trials. The company announced the first patients have been dosed in its phase 3 MAGNITUDE trial in March 2024.¹

MAGNITUDE is evaluating NTLA-2001, or as per its new name, nexiguran ziclumeran (nex-z), a gene editing therapy, as a potential disease-modifying therapy for transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM). The therapy is also still being evaluated in a phase 1/2 trial, data from which are set to be presented at the American Heart Associations 2024 Scientific Sessions Meeting, to be held in Chicago, Illinois, on November 16-19.²

“While this therapy NTLA-2001 does not necessarily target the myocardium, it is for a cardiac indication. So, this makes it essentially the first phase 3 trial that we’ve run across... It does signal an important milestone for us is that we are crossing over to that to that space of development [for which there have been many years of development] for different products in the hematology space and in other areas, but not really in cardiology,” Ahmad Masri, MD, MS, director of the Hypertrophic Cardiomyopathy Center at Oregon Health and Science University, who is an investigator on the nex-z program, told HCPLive.

In this clip, Masri discussed how nex-z's progress ties into the growing trajectory of gene therapy in the cardiovascular space, and how each program adds to progress in the space as a whole. He also discussed how platform development for gene therapy may help facilitate therapy development.

REFERENCES

1. Intellia Therapeutics Announces First Patient Dosed in the Phase 3 MAGNITUDE Study of NTLA-2001 as a Single-Dose CRISPR-Based Treatment for Transthyretin Amyloidosis with Cardiomyopathy. News release. March 18, 2024. https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-first-patient-dosed-phase-3

2. Intellia Therapeutics to Present New Clinical Data from the Phase 1 Study of nexiguranziclumeran (nex-z) for the Treatment of Transthyretin (ATTR) Amyloidosis at the 2024 AHA Scientific Sessions. News release. Intellia Therapeutics. October 1, 2024. https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-present-new-clinical-data-phase-1-study