Massachusetts-based gene-editing biotech company Intellia Therapeutics has announced the initiation of a Phase III study of NTLA-2002, its novel gene-editing treatment for hereditary angioedema (HAE). This single-dose therapy is meant to prevent severe swelling attacks in HAE patients.

NTLA-2002 was developed using CRISPR/Cas9 technology to alleviate attacks by deactivating the kallikrein B1 (KLKB1) gene, which codes prekallikrein, a form of the kallikrein protein. The ongoing Phase I/II study claims to have demonstrated that a single-dose treatment can enable a complete response, preventing further recurring attacks. 

Analyst QuickTake: This marks an important developmental milestone as it is the final step towards gaining FDA approval for NTLA-2002, one of the few gene-editing candidates that have progressed to this stage. This notably includes Intellia’s Phase III trial for NTLA-2001, which dosed its first patient in March 2024 . With the recent approval of Casgevy , the first gene-editing treatment, Intellia has the potential to follow suit and be the second gene-editing treatment to enter the market.