Intellia (NTLA) Shares Skyrocket 29.81% on Pivotal Phase 3 Trial Progress for CRISPR HAE Therapy

Intellia Therapeutics (NTLA) shares surged 29.81% on September 18, marking a six-day winning streak with a cumulative gain of 42.69%. The stock reached its highest level since September 2025, with an intraday increase of 31.99%, driven by progress in its pivotal Phase 3 HAELO trial for lonvoguran ziclumeran, an in vivo CRISPR therapy for hereditary angioedema (HAE).

The Phase 3 HAELO study, which enrolled 60 participants in nine months, is a critical milestone for Intellia. The therapy’s potential to address an unmet medical need—offering a one-time treatment for a rare genetic disorder—has intensified investor optimism. Lonvo-z, the investigational drug, demonstrated durable reductions in kallikrein levels in earlier trials, reinforcing its therapeutic promise. Anticipated topline data in early 2026 and a Biologics License Application (BLA) filing by mid-2026 provide clear catalysts for future growth.

Regulatory momentum further supports the stock’s upward trajectory. Lonvo-z has secured Orphan Drug and RMAT designations from the FDA, along with accelerated pathways from the EMA and UK MHRA. These designations offer financial incentives and expedited review, reducing approval risks and enhancing commercial potential. Analysts, meanwhile, project an average price target of $34.14, implying over 127% upside from recent levels, as 26 firms maintain an “Outperform” rating.

Intellia’s leadership in CRISPR technology and a diversified pipeline strengthen its long-term outlook. The company’s in vivo platform targets rare diseases, while ex vivo programs expand into oncology and autoimmune disorders. With a strong balance sheet and strategic partnerships, Intellia is positioned to capitalize on its first-mover advantage in gene editing, solidifying its role as a transformative player in genomic medicine.