As the company announced on its investor relations page, Intellia Therapeutics (Intellia) has reported positive Phase 3 results for its CRISPR-based drug Lonvoguran Ziclumeran (Lonvo-z) for the treatment of hereditary angioedema (HAE). This marks a global first success for in vivo gene editing, according to the company.
The Phase 3 study showed that the one-time treatment significantly reduced the number of swelling attacks in patients with HAE. HAE is a rare, genetic disorder characterized by recurrent, painful swelling of the skin and mucous membranes. The disease is caused by a deficiency or dysfunction of the C1 inhibitor protein, leading to uncontrolled inflammatory reactions.
Lonvo-z is an in vivo gene therapy based on CRISPR/Cas9 technology. It is administered intravenously and aims to correct the defective gene responsible for HAE. According to Intellia, the one-time treatment could offer a permanent solution for patients, rather than lifelong therapy with regular medications.
The company has also initiated a rolling submission process for a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for Lonvo-z, as reported by Yahoo Finance. The rolling submission process allows the company to submit parts of the application as they are completed, rather than waiting for the full submission.
The positive Phase 3 data were also picked up by other media. CNBC reports that Intellia's CRISPR-based drug succeeded in the pivotal study. According to Statnews.com, the treatment reduced swelling attacks in the registration trial. CoinCentral, on the other hand, comments that Intellia's stock (NTLA) fell despite the groundbreaking Phase 3 success – a classic "buy the rumor, sell the news" phenomenon.
The results mark a significant advance in gene therapy, as this is the first successful Phase 3 study for an in vivo gene editing treatment. Previously, gene therapies were mostly performed ex vivo, where cells are modified outside the body and then returned. Intellia's in vivo method allows direct correction of the gene in the body, potentially making the procedure simpler and more accessible to more patients.
Hereditary angioedema affects an estimated 1 in 50,000 people worldwide. The disease can cause life-threatening swelling in the throat that blocks the airways. Current treatments include prophylactic medications such as C1 inhibitor concentrates or bradykinin receptor antagonists, which must be administered regularly. A one-time gene therapy could significantly improve quality of life.
