Intellia Therapeutics Receives U.S. FDA Orphan Drug Designation for NTLA-2001 See Also: Important News from Ultragenyx Pharmaceutical

Intellia Therapeutics Granted Orphan Drug Designation for NTLA-2001
Intellia Therapeutics (NTLA) announced that the U.S. FDA has granted orphan drug designation to NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis. This investigational therapy is the first CRISPR therapy to be administered systemically to edit a disease-causing gene inside the human body. NTLA-2001 has the potential to be the first single-dose treatment for ATTR amyloidosis as it may be able to halt and reverse the devastating complications of this disease. ATTR amyloidosis is a rare condition that can impact a number . . .

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