Intellia to Advance Development of ATTR Amyloidosis, Angioedema, AATD Gene Therapies


The company is kicking off 2023 by announcing planned advancements for several gene therapies in its pipeline.

Intellia Therapeutics has announced its intention to initiate global pivotal trials of its CRISPR-based therapies NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis and NTLA-2002 for hereditary angioedema (HAE) and submit an investigational new drug application (IND) for NTLA-3001 for alpha-1 antitrypsin deficiency (AATD)-associated liver disease.1

For NTLA-2001, Intellia plans to submit an IND to include US sites in the pivotal study and to also present updated clinical data from the ongoing phase 1 study (NCT04601051). The study previously reported positive data from the study in September 2022 that showed deep reductions in serum TTR over a 28-day period after 9 patients in the low dose cohort received the therapy.2 The company also plans to prepare for a phase 3 study for treating ATTR amyloidosis with polyneuropathy.

Similarly, Intellia plans to submit an IND for NTLA-2002 to include US sites in the pivotal phase 2 study and to provide updated data from the ongoing first-in-human study (NCT05120830). Previously, NTLA-2002 demonstrated reductions in plasma kallikrein and HAE attack rates in data presented in November 2022.3

“2022 proved to be another outstanding year for Intellia, with several significant clinical milestones achieved across our pipeline, further reinforcing the ability of our modular CRISPR genome editing platform to target a broad range of diseases,” John Leonard, MD, president and chief executive officer, Intellia, said in a statement.1 “These accomplishments reflect steady execution against our core strategy: to harness the immense power of genome editing, both for in vivo and ex vivo applications. As we look ahead, our highest priority will be to prepare for the initiation of global pivotal trials for our first two investigational in vivo CRISPR-based therapies, NTLA-2001 for ATTR amyloidosis and NTLA-2002 for hereditary angioedema. As these programs continue to progress, we believe we are moving closer to setting a new standard of care for people living with these and other serious diseases.

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NTLA-3001 is Intellia’s next candidate positioned to enter trials, with IND-enabling activities set to complete this year. It is also the company’s first CRISPR-based gene insertion therapy to head to trials. Intellia also made announcements concerning new platform capabilities, including next-generation allogeneic and CRISPR and other gene editing techniques. Intellia also announced its intention to seek collaboration opportunities to advance the development of its ex vivo candidate NTLA-6001 for CD30+ lymphomas.

“In addition, we are advancing the next wave of platform capabilities, such as in vivo gene insertion and our proprietary allogeneic solution. Importantly, while the possibilities to apply our industry-leading genome editing technology are expansive, we are taking a disciplined approach with our portfolio by deploying resources on high-impact opportunities and collaborating with a network of other scientific leaders to expand the applications of our innovative technologies,” Leonard added to the statement.1

1. Intellia Therapeutics highlights strategic priorities and anticipated 2023 key milestones. News release. Intellia Therapeutics. January 5, 2023.
2. Intellia Therapeutics Presents New Interim Data from First-in-Human Study of NTLA-2002 for the Treatment of Hereditary Angioedema (HAE) at the American College of Allergy, Asthma & Immunology 2022 Annual Scientific Meeting/ November 12, 2022.
3. Intellia and Regeneron Announce Initial Data from the Cardiomyopathy Arm of Ongoing Phase 1 Study of NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis. News release. Intellia Therapeutics. September 16, 2022.
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