The genome editing company Intellia Therapeutics has administered NTLA-2002, an investigational clustered regularly interspaced short palindromic repeats (CRISPR)-based therapy, to the first patient in its phase 1/2 study in adults with type I or type II hereditary angioedema (HAE).
“Today, we are pleased to announce dosing has begun in the phase 2 study of NTLA-2002, and based on strong interest from investigators and patients, we expect to complete enrollment in the second half of this year,” said John Leonard, MD, president and chief executive officer of Intellia.
“Our rapid progression of the clinical development of NTLA-2002 supports our goal to bring forth a potential functional cure for the treatment of HAE. Simultaneously, we are working toward submitting our second in vivo IND [Investigational New Drug] application and initiating a global pivotal trial for NTLA-2001. We look forward to sharing new interim data from both the NTLA-2001 and NTLA-2002 first-in-human studies in the months to come.”
Intellia has recently reported operational highlights and financial results for the first quarter of 2023 that included updates for in vivo programs in HAE, hereditary transthyretin amyloidosis (hATTR), and alpha-1 antitrypsin deficiency (AATD).
Read more about HAE therapies
“We’ve had a productive start to the year with the successful clearance of our first IND application for an investigational in vivo CRISPR-based therapy alongside broad pipeline and platform progress,” said Leonard.
The US Food and Drug Administration (FDA) has cleared the NTLA-2002 phase 2 IND application and granted Regenerative Medicine Advanced Therapy designation to NTLA-2002 for the treatment of HAE. Moreover, NTLA-2002 was awarded the Innovation Passport of the UK Medicines and Healthcare Products Regulatory Agency.
Intellia plans to divulge additional clinical data, such as updated safety, pharmacodynamic data, and attack-rate measures, from the phase 1 portion of the first-in-human study in 2023.
Regarding NTLA-2001, an investigational CRISPR-based therapy designed to inactivate the TTR gene in liver cells, Intellia is currently preparing for a global pivotal study in hATTR with polyneuropathy (hATTRv-PN). The enrollment of the dose-expansion portion of the hATTRv-PN arm in the phase 1 study has been concluded and the company has begun redosing patients.
Read more about hATTR therapies
Furthermore, Intellia plans to submit a US IND application in mid-2023 and expects to initiate a global pivotal trial for ATTR with cardiomyopathy (ATTR-CM) by the end of the year.
Additional data from the hATTR-PN and ATTR-CM arms of the phase 1 study is expected in 2023.
Intellia is conducting IND-enabling activities for NTLA-3001 and NTLA-2003, candidate therapies for the treatment of AATD-associated lung disease and AATD-associated liver disease, respectively. The company plans to submit an IND or IND-equivalent filing for NTLA-3001 in the second half of 2023.
Intellia Therapeutics announces first quarter 2023 financial results and highlights recent company progress. News release. Intellia Therapeutics, Inc.; May 4, 2023