Intellia Therapeutics to Present Data from Clinical Trial of NTLA-2002 Aimed at Treating Hereditary Angioedema

Intellia Therapeutics to Present Interim Clinical Data from NTLA-2002 Trial for Hereditary Angioedema

An abstract featuring interim clinical data from the Phase 1/2 study of Intellia Therapeutics’ (NTLA) product NTLA-2002 has been selected for an oral presentation at the 2022 Bradykinin Symposium, taking place September 15-16 in Berlin, Germany.


NTLA-2002 is an investigational in vivo CRISPR/Cas9 therapy in development as a single-dose treatment to prevent angioedema attacks in people with hereditary angioedema (HAE). The presentation will include interim safety, kallikrein reduction and attack rate data from the ongoing dose-escalation portion of Intellia’s first-in-human study of NTLA-2002. 

Presentation Details

Title: “In vivo CRISPR/Cas9 editing of KLKB1 in Patients with HAE” 

Date and Time: Friday, September 16, 2022 from 10:55 – 11:05 a.m. 

Session: Session V — Short Oral Presentations

Presenter: Dr. Hilary Longhurst, M.D., Ph.D., FRCP, FRCPath, Senior Medical Officer at Auckland District Health Board and Honorary Associate Professor at University of Auckland, New Zealand, the trial’s principal investigator in New Zealand 

Intellia Therapeutics Investor Event: Intellia plans to host a webcast to review the presented clinical data at the 2022 Bradykinin Symposium. The Company will announce details for the event in September.

About the NTLA-2002 Clinical Program

Intellia’s multi-national Phase 1/2 study is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II HAE. This includes the measurement of plasma kallikrein protein levels and activity as determined by HAE attack rate measures. The Phase 1 portion of the study is an open-label, single-ascending dose design used to identify up to two dose levels of NTLA-2002 that will be further evaluated in the randomized, placebo-controlled Phase 2 portion of the study. This Phase 1/2 study will identify the dose of NTLA-2002 for use in future studies.

Visit (NCT05120830) for more details. 

The Product

Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2002 is the first single-dose investigational treatment being explored in clinical trials for the potential to continuously reduce kallikrein activity and prevent attacks in people living with HAE.

NTLA-2002 is a wholly owned investigational CRISPR therapeutic candidate designed to inactivate the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein. NTLA-2002 is Intellia’s second investigational CRISPR therapeutic candidate to be administered systemically, by intravenous infusion, to edit disease-causing genes inside the human body with a single dose of treatment. 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 together carry out the precision editing

Hereditary Angioedema

HAE is a rare, genetic disorder characterized by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body, which can be painful, debilitating and life-threatening. It is estimated that one in 50,000 people are affected by HAE, and current treatment options often include life-long therapies, which may require chronic intravenous (IV) or subcutaneous (SC) administration as often as twice per week, or daily oral administration to ensure constant pathway suppression for disease control. Despite chronic administration, breakthrough attacks still occur. Kallikrein inhibition is a clinically validated strategy for the preventive treatment of HAE attacks.

About Intellia Therapeutics

Intellia Therapeutics, a clinical-stage genome editing company. It is developing novel, potentially curative therapeutics leveraging CRISPR-based technologies. To fully realize the transformative potential of CRISPR-based technologies, Intellia is pursuing two primary approaches.

The company’s in vivo programs use intravenously administered CRISPR as the therapy, in which proprietary delivery technology enables highly precise editing of disease-causing genes directly within specific target tissues.

Intellia’s ex vivo programs use CRISPR to create the therapy by using engineered human cells to treat cancer and autoimmune diseases. Intellia’s deep scientific, technical, and clinical development experience, along with its robust intellectual property portfolio, have enabled the company to take a leadership role in harnessing the full potential of genome editing to create new classes of genetic medicine.

Prohost Observations 

Intellia’s value comes mainly from the novel genome editing Clustered Regularly Interspaced Short Palindromic Repeat (CRiSPR). This technology is adapted from the natural defense mechanisms of microorganisms. These organisms use CRISPR-derived RNA, a molecular cousin to DNA, in addition to various Cas proteins to foil attacks by viruses.    

Regarding the firm’s value, the most important factor is not the revenues or incomes, as the firm is still clinical stage, it is the CRiSPR technology and the success of the firm’s in vivo program, which enables a systemic administration of the CRiSPR therapy. This success is expected to lead to a precise editing of disease-causing genes directly within specific target tissues.

NTLA is way undervalued. Why? Because many investors are still causing selloffs based on the incomes, rather than on the future income. This future is near, as current trials have reached Phase 3. The firm is further undervalued because of the current bad economy, the abnormally high inflation and the high price of oil and gas.

All these problems will not deny Intellia’s stock price from soaring, which is going to happen sooner rather than later.

Click here to read more about Intellia Therapeutics.

Leave a Reply