NTLA-2001 Gene Editing Trial to Include Patients With Heart Disease

NTLA-2001 Gene Editing Trial to Include Patients With Heart Disease

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NTLA-2001 Gene Editing Trial to Include Patients With Heart Disease

A clinical trial of the gene-editing therapy NTLA-2001 in people with familial amyloid polyneuropathy (FAP) is expanding to include those with a related condition, called ATTR amyloidosis with cardiomyopathy (ATTR-CM), Intellia Therapeutics, which is developing the therapy, reported. FAP is a form of ATTR amyloidosis — a group of disorders caused by the buildup of protein clumps, called amyloids, made up of a misfolded version of the protein transthyretin. FAP is characterized by neurological symptoms (polyneuropathy) and…

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