Ahmad Masri, MD, MS: Establishing Proof-of-Concept of Gene Editing in ATTR-CM

Intellia Therapeutics hopes to develop 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.

Data from the phase /21 trial of nex-z are set to be presented at the American Heart Associations 2024 Scientific Sessions Meeting, to be held in Chicago, Illinois, on November 16-19.

HCPLive spoke with Ahmad Masri, MD, MS, director of the Hypertrophic Cardiomyopathy Center at Oregon Health and Science University, about how nex-z's phase 1 tiral data has provided proof-of-concept for the therapy. Masri also shared his excitement for the future findings of the ongoing phase 3 MAGNITUDE trial.

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