A single infusion of NTLA-2001, an experimental gene-editing therapy, generally is safe and leads to a marked reduction in blood levels of the harmful transthyretin (TTR) protein in people with familial amyloid polyneuropathy (FAP) and ATTR amyloidosis with cardiomyopathy (ATTR-CM). These updated, interim data cover 65 of 72 patients…

Intellia Therapeutics is launching a pivotal Phase 3 clinical trial in the U.S. to evaluate the safety and effectiveness of its investigational gene-editing therapy NTLA-2001 in people with ATTR amyloidosis with cardiomyopathy (ATTR-CM). The study, which is expected to start by the end of the year, follows the recent…

The U.S. Food and Drug Administration (FDA) has rejected Alnylam Pharmaceuticals‘ application seeking the expansion of its Onpattro (patisiran) label to include treatment of people with ATTR amyloidosis with cardiomyopathy (ATTR-CM), or heart damage. Based on the regulatory rejection, Alnylam has decided to no longer pursue an approval of…

ATTR amyloidosis with cardiomyopathy (ATTR-CM), noting damage to the heart, is usually considered to be an irreversible condition. But in a first known reporting, the disease spontaneously resolved for three men in the U.K. Accompanying ATTR-CM’s resolution was the presence in the bloodstream of antibodies targeted against the insoluble transthyretin…

A single dose of NTLA-2001, Intellia Therapeutics’ investigational gene-editing therapy, safely and effectively leads to rapid and sustained reductions in the levels of the damaging transthyretin (TTR) protein in people with familial amyloid polyneuropathy (FAP), according to interim data from a Phase 1 trial. Notably, maximum TTR reductions…

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…

In focus groups, transthyretin (ATTR) amyloidosis patients and families reported challenges and concerns related to the odyssey of getting a diagnosis, the effect of symptoms on quality of life, and the familial impact of the rare inherited disease. The study, which included people with…

Eidos Therapeutics announced that the Phase 3 clinical trial evaluating its lead candidate acoramidis (formerly known as AG10) in treating adults with transthyretin amyloidosis cardiomyopathy (ATTR-CM) is fully enrolled. Top-line results from part A of the trial, called ATTRibute-CM (NCT03860935), are expected in late 2021 or early 2022, and from…

The European Commission (EC) has approved the once-daily oral medication Vyndaqel (tafamidis) for the treatment of cardiomyopathy — a disease of the heart muscle that makes it harder for the heart to pump blood to the rest of the body — in adults with transthyretin…