Intellia Therapeutics Announces Expansion of Ongoing Phase 1 Study of NTLA-2001 to Include Adults with Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)
- Approved protocol amendment enables enrollment of ATTR-CM patients in the ongoing first-in-human study of NTLA-2001, a systemically delivered CRISPR/Cas9-based therapy
The first investigational therapy of its kind, NTLA-2001 is an in vivo CRISPR/Cas9-based genome editing candidate being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis. It is designed to inactivate the TTR gene in liver cells to prevent the production of transthyretin (TTR) protein and is being developed to potentially be the first single-dose ATTR amyloidosis treatment to not only halt but also reverse disease progression in both ATTRv-PN and ATTR-CM patients. NTLA-2001 has received orphan drug designation for the treatment of ATTR amyloidosis by both the
“ATTR amyloidosis is a chronic, fatal disease that can impact different organs and tissues within the body, often manifesting as either polyneuropathy or cardiomyopathy. At Intellia, our goal is to develop a potentially curative treatment that could benefit as many patients living with this disease as possible,” said Intellia President and Chief Executive Officer
The protocol amendment to the Phase 1 study allows for enrollment of up to 36 adults in the
Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially be the first curative treatment for ATTR amyloidosis. NTLA-2001 is the first investigational CRISPR therapy candidate to be administered systemically, or intravenously, to edit genes inside the human body. Intellia’s proprietary non-viral platform deploys lipid nanoparticles to deliver to the liver a two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which carries out the precision editing. Robust preclinical data, showing deep and long-lasting transthyretin (TTR) reduction following in vivo inactivation of the target gene, supports NTLA-2001’s potential as a single-administration therapeutic. Interim Phase 1 clinical data released in
About the NTLA-2001 Clinical Program
The global Phase 1 trial is an open-label, multi-center, two-part study of NTLA-2001 in adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) or cardiomyopathy (ATTR-CM). The trial’s primary objectives are to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2001. Patients receive a single dose of NTLA-2001 via intravenous administration. The study will enroll up to 38 ATTRv-PN participants (ages 18-80 years) and up to 36 ATTR-CM participants (ages 18-90 years) and consists of a single-ascending dose phase followed by a dose-expansion phase. Visit clinicaltrials.gov (NCT04601051) for more details.
About Transthyretin (ATTR) Amyloidosis
Transthyretin amyloidosis, or ATTR amyloidosis, is a rare, progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis occurs when a person is born with mutations in the TTR gene, which causes the liver to produce structurally abnormal transthyretin (TTR) protein with a propensity to misfold. These damaged proteins build up as amyloid in the body, causing serious complications in multiple tissues, including the heart, nerves and digestive system. ATTRv amyloidosis predominantly manifests as polyneuropathy (ATTRv-PN), which can lead to nerve damage, or cardiomyopathy (ATTRv-CM), which can lead to heart failure. Some individuals without the genetic mutation produce non-mutated, or wild-type TTR proteins that become unstable over time, misfolding and aggregating in disease-causing amyloid deposits. This condition, called wild-type ATTR (ATTRwt) amyloidosis, primarily affects the heart. There are an estimated 50,000 people worldwide living with ATTRv amyloidosis and between 200,000 and 500,000 people with ATTRwt amyloidosis.
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
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Source: Intellia Therapeutics, Inc.