Intellia's NTLA-2002 Phase 1 CRISPR-Based Treatment Shows Promising Results in HAE

Jun 3, 2024, 12:49 AM

Intellia Therapeutics has announced positive long-term data from its ongoing Phase 1 study of NTLA-2002, a CRISPR-based treatment for hereditary angioedema (HAE). The data, presented at the EAACI 2024 Congress, showed a dramatic reduction in patients' swelling attacks. Despite the promising results, there are discussions about the appropriate dosing for pivotal trials. Some analysts, including RNAiAnalyst, suggest that a 75mg dose would be clearly superior due to its efficacy with a -95% reduction in PKK, though Intellia is currently only considering 25mg and 50mg doses due to safety concerns.