Gene-editing Therapy NTLA-2001 Given Orphan Drug Status by FDA

Gene-editing Therapy NTLA-2001 Given Orphan Drug Status by FDA

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Gene-editing Therapy NTLA-2001 Given Orphan Drug Status by FDA

The U.S. Food and Drug Administration (FDA) has given an orphan drug designation to NTLA-2001, an investigational gene-editing therapy for familial amyloid polyneuropathy (FAP) and other forms of transthyretin (ATTR) amyloidosis. This designation is given to experimental medicines that have the potential to improve care for people with rare diseases — conditions that affect fewer than 200,000 people in the U.S. It gives therapy developers (Intellia Therapeutics in this case) certain incentives, most notably the…

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