First U.S. Patient Enrolls in MAGNITUDE Phase 3 Gene-Editing Trial at MedStar

In a groundbreaking development, the MedStar Washington Hospital Center has enrolled the first American heart patient in the MAGNITUDE study, a Phase 3 clinical trial investigating the potential of gene-editing therapy for transthyretin amyloidosis with cardiomyopathy (ATTR-CM). This condition, which is often overlooked, is a significant cause of heart failure characterized by the stiffening of the heart muscle due to the accumulation of the TTR protein, which hampers the organ's ability to circulate blood effectively.

The MAGNITUDE study, a global, multicenter initiative, employs a double-blind, placebo-controlled design to assess the safety and efficacy of a novel gene-editing treatment. This treatment aims to halt the synthesis of the abnormal TTR protein by utilizing the CRISPR/Cas9 gene-editing system within the NTLA-2001 medication. The drug, delivered via a single intravenous infusion, targets the liver, the primary site of TTR protein production.

ATTR-CM is a severe and life-threatening manifestation of ATTR, where the TTR protein forms deposits known as amyloidosis in the heart and nervous system. The study, led by Dr. Farooq Sheikh, medical director of the Advanced Heart Failure Program at MedStar Washington Hospital Center, has garnered significant interest due to the potential impact on the lives of patients suffering from this condition.

Preliminary data from Phase 1 and 2 trials indicate that NTLA-2001 has successfully led to a sustained reduction in TTR protein levels. This marks the first time an investigational CRISPR therapy has been used intravenously for gene editing within the human body. The study is anticipated to include around 765 participants globally, contributing to the extensive cardiovascular research portfolio at MedStar Health.

It is estimated that 50,000 individuals globally are affected by hereditary ATTR amyloidosis, while between 200,000 and 500,000 people suffer from the wild-type form, which predominantly affects the heart, resulting in ATTR-CM. Currently, there is no cure for these conditions.

The MAGNITUDE study represents a significant step forward in the exploration of gene-editing therapies for heart diseases and could offer new hope for patients battling this debilitating condition.