Gene-editing Therapy NTLA-2001 Given Orphan Drug Status by FDA The U.S. Food and Drug Administration (FDA) has given orphan drug designation to NTLA- 2001, an investigational gene-editing therapy for familial amyloid polyneuropathy (FAP) and other forms of transthyretin (ATTR) amyloidosis. … The aim of NTLA-2001 is to disrupt the TTR gene, thus decreasing transthyretin production and preventing the buildup of these harmful protein […]