Intellia Gets FDA Orphan Designation for NTLA-2001 in ATTR Amyloidosis
October 21 2021 - 8:13AM
Dow Jones News
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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