Intellia Therapeutics has released updated information highlighting results on conditions such as hereditary angioedema (HAE), among other rare diseases in its portfolio.

Intellia’s technologies hold the potential to offer curative therapies for life-threatening conditions such as HAE, transthyretin amyloidosis (ATTR), and alpha-1 antitrypsin deficiency (AATD).

“We continue to make significant and rapid progress in the development of our first two in vivo, CRISPR [clustered regularly interspaced short palindromic repeats]-based therapeutic candidates,” said John Leonard, MD, president and chief executive officer of Intellia.

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“For NTLA-2002, we are thrilled to see the excitement for this investigational therapy from the HAE community. In just a handful of months, we were able to identify all patients required to fully enroll the ongoing phase 2 study and now expect to initiate the pivotal phase 3 program next year. For NTLA-2001, our team has been working diligently to prepare for an IND [Investigational New Drug] submission in September and to begin the phase 3 study for the cardiomyopathy manifestation of ATTR amyloidosis before the end of this year. Overall, we continue to believe that NTLA-2001 and NTLA-2002 represent only the beginning for our pipeline and platform as additional in vivo and ex vivo candidates advance toward the clinic.”

Read more about HAE therapies

NTLA-2002 is an in vivo CRISPR-based therapy that is meant to prevent HAE attacks through a reduction of plasma kallikrein activity after only 1 dose. It also aims to eliminate the high treatment burden that is associated with other HAE therapies. The drug has the potential to permanently reduce total plasma kallikrein protein and activity, which is a key mediator of HAE. It is being evaluated in a phase 1/2 study in adults with type I or II HAE.

Intellia announced that all patients have been identified for the global phase 2 portion of the study, and enrollment is expected to be completed in the second half of 2023.

Positive interim results from the phase 1 portion of the ongoing phase 1/2 study of NTLA-2002 were released earlier this year. The data showed a 95% mean reduction in monthly attack rate after a single dose of NTLA-2002. The drug has been well tolerated at each of 3 doses, and most adverse events were mild.


Intellia Therapeutics announces second quarter 2023 financial results and highlights recent company progress. News release. Intellia Therapeutics, Inc.; August 3, 2023.