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Share Name | Share Symbol | Market | Type |
---|---|---|---|
Intellia Therapeutics Inc | NASDAQ:NTLA | NASDAQ | Common Stock |
Price Change | % Change | Share Price | Bid Price | Offer Price | High Price | Low Price | Open Price | Shares Traded | Last Trade | |
---|---|---|---|---|---|---|---|---|---|---|
-0.07 | -0.30% | 23.65 | 23.64 | 23.69 | 23.845 | 23.29 | 23.77 | 117,241 | 15:18:07 |
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)
Copyright (c) 2021 Dow Jones & Company, Inc.
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