Positive Phase 1 NTLA-2002 Interim Data for Hereditary Angioedema Patients Published in NEJM

A recent study has shown that NTLA-2002, an experimental gene editing treatment for hereditary angioedema (HAE), significantly reduced the frequency of swelling attacks in patients. The research, published in the New England Journal of Medicine, indicated that a single administration of the CRISPR-based therapy led to a 95% decrease in the average monthly rate of HAE attacks. Notably, 90% of the participants remained free from attacks during the 16-week primary observation period and beyond. The drug was found to be well-tolerated, with the most common side effects being mild and transient, such as infusion-related reactions and fatigue. This marks the second time that initial clinical data for one of Intellia Therapeutics' in vivo CRISPR therapies has been featured in the prestigious medical journal.
Intellia Therapeutics, a company at the forefront of gene editing with CRISPR technology, is developing NTLA-2002 as a potential one-time treatment for HAE, a rare and potentially life-threatening genetic disorder. The company's president, John Leonard, M.D., expressed optimism about the interim data and mentioned plans to present further findings from the Phase 1 and Phase 2 trials later in the year. They are also preparing to start a pivotal global study for NTLA-2002 in 2024, pending regulatory feedback.
The Phase 1/2 clinical trial of NTLA-2002 is assessing its safety and efficacy in adults with Type I or Type II HAE. The open-label, international study aims to determine an appropriate dosage for a subsequent Phase 3 trial. Enrollment for this phase is complete, and more information can be found on clinicaltrials.gov under the identifier NCT05120830.
Hereditary angioedema is a rare genetic disease that causes severe, unpredictable, and potentially life-threatening swelling attacks. It affects an estimated one in 50,000 individuals. While there is no cure, there are treatments available to manage the condition, which may include long-term prophylactic therapies. Despite these treatments, patients often experience breakthrough attacks. NTLA-2002 works by inactivating the KLKB1 gene, which is responsible for producing the kallikrein precursor protein, thereby preventing HAE attacks.
Intellia Therapeutics is a clinical-stage company that is pioneering the use of CRISPR-based therapies to treat various diseases. Their in vivo programs focus on precise gene editing within the human body, while their ex vivo programs involve engineering human cells to combat cancer and autoimmune diseases. The company is committed to advancing the capabilities of its gene editing platform and is exploring novel editing and delivery technologies to enhance its effectiveness. For more information, visit intelliatx.com or follow them on social media.

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