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 deposits in different parts of the body. The experimental therapy is thought to accomplish this by removing the mutated TTR gene from patients’ liver cells, the main producers of TTR, using the CRISPR/Cas-9 gene-editing tool.

… NTLA-2001 uses Intellia’s proprietary tiny particles, or nanoparticles, to deliver CRISPR/Cas- 9 components to cells.

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