By Colin Kellaher

 

Intellia Therapeutics Inc. on Thursday said the U.S. Food and Drug Administration granted orphan-drug designation to NTLA-2001 for the treatment of transthyretin, or ATTR, amyloidosis.

The Cambridge, Mass., clinical-stage genome-editing company said NTLA-2001 is the first CRISPR therapy to be administered systemically to edit a disease-causing gene inside the human body.

The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S. and provides for an extended marketing-exclusivity period against competition.

ATTR amyloidosis is a rare condition that can impact a number of organs and tissues within the body through the accumulation of misfolded transthyretin protein deposits.

Intellia said NTLA-2001 has the potential to be the first single-dose treatment for ATTR amyloidosis, as it may be able to halt and reverse complications of the disease.

 

Write to Colin Kellaher at colin.kellaher@wsj.com

 

(END) Dow Jones Newswires

October 21, 2021 07:58 ET (11:58 GMT)

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