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Intellia Therapeutics: NTLA-2002 CRISPR-based therapy reduces rates of Hereditary Angioedema (HAE) attacks
- CRISPR gene editing based treatment reduced HAE attacks rates by up to 80%.
- NTLA-2002 was well tolerated by patients.
The Latest
In a phase 2, randomized, double-blind, placebo-controlled study done by Intellia Therapeutics the efficacy and safety of NTLA-2002 was tested. The study involved 27 participants who were randomized to receive either a 25mg dose, 50mg dose or placebo via intravenous infusion. Participants were then observed for a 16-week period. The mean monthly attack rate compared to placebo was reduced by 75% and 77% for 25mg and 50mg dose, respectively during weeks 1-16 and reduced by 80% and 81% during weeks 5-16. 8 of 11 participants given the 50mg dose reported no attacks at all in the 16-week observation period after a single dose of NTLA-2002. In comparison, 4 of the 10 patients given 25mg doses experienced a complete response. Furthermore, patients receiving the 50mg dose achieved a kallikrein protein reduction of 86% from baseline compared to 55% reduction in the 25mg dose group at week 16. The placebo group experienced no appreciable freedom of HAE attacks. At both dose levels, NTLA-2002 was well-tolerated.
Physicians Perspective
HAE is a rare, genetic disease characterized by severe, recurring and unpredictable inflammatory attacks to various organs and tissues of the body, causing severe, debilitating pain that can be life-threatening. It is estimated that 1 in 50,000 people are affected by HAE. There is no known cure for HAE. Current preventative treatment options include long and short-term prophylaxis that prevents swelling attacks. These therapies are life-long and require chronic intravenous or subcutaneous administration as often as twice per week or daily oral administration. Current options for controlling attacks include BioCryst Orladeyo (once a day) and Takeda’s Takhzyro (every 2 to 4 weeks). Moreover, even with chronic administration, breakthrough attacks can still occur. Intellia Therapeutics developed NTLA-2002 with the goal of developing a one-time therapy for patients with HAE with the potential to completely eliminate HAE attacks.
Molecular Targets
HTLA-2002 utilizes CRISPR/Cas9 technology to reduce HAE attacks. The drug works by inactivating the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein; the precursor for kallikrein protein. NTLA-2002 attacks are caused by increased bradykinin production which leads to fluid leakage, tissue swelling, and painful, unpredictable angioedema episodes. Kallikrein is an enzyme that activates bradykinin by cleaving kininogen. However, people with HAE have a deficiency in C1 inhibitor, a protein that usually controls kallikrein activity. Without C1-INH, kallikrein becomes overactive leading to excessive bradykinin. By knocking down KLKB1, NTLA-2002 reduces production of kallikrein, thereby decreasing bradykinin levels and recurrent swelling.
Company History
Intellia Therapeutics is leading clinical gene editing company based in Cambridge, Massachusettes focused on CRISPR-based therapies. The company is also working to utilize CRISPR/as-9 technology to develop therapies for transthyretin amyloidosis, alpha-1 antitrypsin deficiency, and hemophilia A/B.
Further Reading: https://ir.intelliatx.com/news-releases/news-release-details/intellia-presents-positive-results-phase-2-study-ntla-2002
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