Intellia Therapeutics’ Investigational CRISPR Treatment NTLA-2001 Receives European Union Orphan Drug Designation for ATTR Amyloidosis

CAMBRIDGE, Mass., March 30, 2021 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, announced today that the European Commission (EC) has granted orphan drug designation to NTLA-2001. This investigational medicinal product is being developed as a treatment for transthyretin amyloidosis (ATTR), a rare condition that can impact a number of organs and tissues within the body through the accumulation of misfolded transthyretin (TTR) protein deposits. NTLA-2001 is the first experimental CRISPR therapy – a Nobel Prize-winning technology – to be administered systemically via intravenous dosing to edit a gene, specifically the TTR gene, that encodes the production of TTR protein, inside the human body. In addition to being the first therapy of its kind, it also has the potential to be the first curative treatment and it may be able to halt and reverse ATTR progression.

“This news is a significant milestone for NTLA-2001 and the ATTR patient community,” said Intellia President and Chief Executive Officer John Leonard, M.D. “We are pleased that the EC recognizes the potential significant benefit of NTLA-2001 in the treatment of patients with this debilitating disease where there is no cure. We look forward to advancing the global development of this genome editing product in collaboration with Regeneron.”

This decision by the EC follows the initiation of Intellia’s global Phase 1 study to evaluate NTLA-2001 for hereditary ATTR with polyneuropathy (hATTR-PN). The trial aims to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2001. Following safety assessment and dose optimization, Intellia intends to further evaluate NTLA-2001 in both polyneuropathy and cardiomyopathy patients. Orphan drug designation is granted to therapies that are intended for the treatment, prevention, or diagnosis of life threatening or chronically debilitating rare diseases where there are either no treatments or no satisfactory therapeutic options. The designation provides regulatory, financial and commercial incentives to develop therapies for rare diseases defined as having a prevalence of less than five in 10,000 people in the European Union.