Intellia Therapeutics’ Investigational CRISPR Treatment NTLA-2001 Receives European Union Orphan Drug Designation for ATTR Amyloidosis
“This news is a significant milestone for NTLA-2001 and the ATTR patient community,” said Intellia President and Chief Executive Officer
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
NTLA-2001 is the first experimental CRISPR therapy to be administered systemically, or through a vein, to edit a gene inside the human body. Intellia’s proprietary non-viral platform utilizes lipid nanoparticles designed to deliver to the liver a simple, two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 protein. Intellia’s robust preclinical data showing deep and long-lasting transthyretin (TTR) reduction following knockout of the target gene in vivo support NTLA-2001’s potential as a single dose of treatment. NTLA-2001 is part of a co-development/co-promotion agreement between Intellia, the lead development and commercialization party, and Regeneron Pharmaceuticals, Inc.
About Transthyretin Amyloidosis (ATTR)
Transthyretin amyloidosis, or ATTR, is a rare, progressive and fatal disease. Hereditary ATTR (hATTR) occurs when a person is born with a specific DNA mutation in the TTR gene, which causes the liver to produce a protein called transthyretin (TTR) in a misfolded form and build up in the body. hATTR can manifest as polyneuropathy (hATTR-PN), which can lead to nerve damage, or cardiomyopathy (hATTR-CM), which involves heart muscle disease that can lead to heart failure. In addition, non-mutated, or wild-type TTR protein, can also accumulate in the body, leading to wild-type ATTR (wtATTR). There are an estimated 50,000 hATTR patients worldwide and between 200,000 and 500,000 people with wtATTR.
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company, focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by both producing therapeutics that permanently edit and/or correct disease-associated genes in the human body with a single treatment course, and creating enhanced engineered cells that can treat oncological and immunological diseases. Intellia’s combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts it in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create new classes of therapeutic products. Learn more about Intellia and CRISPR/Cas9 at intelliatx.com. Follow us on Twitter @intelliatweets.
Forward Looking Statements
This press release contains “forward-looking statements” of
Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain its intellectual property position; risks related to Intellia’s relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Intellia’s product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the risk that Intellia’s collaborations with Regeneron or its other collaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the
Interim Head of
Source: Intellia Therapeutics, Inc.