Intellia Highlights New Early Data for Single-Dose HAE Therapy

A revolutionary gene-editing therapy developed by Intellia Therapeutics has shown remarkable promise in treating hereditary angioedema (HAE), according to data from an early-stage study. The therapy, known as NTLA-2002, uses in vivo CRISPR-based gene editing technology and has demonstrated a significant reduction in the frequency of attacks in HAE patients.

In the initial part of a Phase I/II clinical trial, a single dose of NTLA-2002 resulted in a 98% average reduction in the monthly attack rates among participants. The trial's median follow-up duration was 20.1 months. John Leonard, CEO of Intellia Therapeutics, highlighted the "unprecedented data," noting that eight out of ten study participants were attack-free for 18 months or longer following just one dose of NTLA-2002.

Presented at the European Academy of Allergy and Clinical Immunology (EAACI) congress, the study findings also revealed a 99% average reduction in moderate-to-severe attacks. Leonard emphasized that the reduction in attack rates was consistent even among patients suffering from the most severe symptoms. He suggested that NTLA-2002 could potentially serve as a one-time, functional cure for HAE, given the durability of its effects.

Intellia Therapeutics is now preparing to initiate a pivotal Phase III trial for NTLA-2002 in the latter half of the year. The upcoming trial aims to validate the promising results observed in the earlier phases and possibly bring this groundbreaking treatment closer to clinical use.