Intellia Therapeutics, Inc. (NTLA), a clinical-stage gene editing company, Monday said that the U.S. Food and Drug Administration or FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its nexiguran ziclumeran (nex-z) for the treatment of hereditary transthyretin (ATTR) amyloidosis with polyneuropathy. Intellia shares were up more than 4 percent in pre-market.
Hereditary ATTR amyloidosis, caused by a mutation in the transthyretin (TTR) gene, is a progressive disorder characterized by the buildup of abnormal protein deposits in body's organs and tissues.
Nex-z is a CRISPR-based investigational therapy designed as a single-dose treatment for ATTR amyloidosis with polyneuropathy.
RMAT designation was granted based on interim data from the Phase 1 study which showed one-time treatment with nex-z led to rapid, deep and durable TTR reduction in patients with ATTR amyloidosis with polyneuropathy.
RMAT designation includes benefits such as early interactions with the FDA, eligibility for priority review and accelerated approval.
As part of a multi-target collaboration with Regeneron, development and commercialization of nex-z is led by Intellia.
Intellia stock had closed at $14.30, up 3.62 percent on Friday. It has traded in the range of $12.82 - $34.87 in the last 1 year.
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