The Food and Drug Administration (FDA) has granted Orphan Drug designation to NTLA-2002 for the treatment of hereditary angioedema.

Using CRISPR/Cas9 technology, the investigational therapy is designed to edit disease-causing genes inside the human body. NTLA-2002 inactivates the kallikrein B1 (KLKB1) gene, which reduces kallikrein activity, thereby preventing attacks in patients with hereditary angioedema. It is administered as a single intravenous infusion.

The Company is currently investigating the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or II hereditary angioedema in a phase 1/2 clinical study (ClinicalTrials.gov Identifier: NCT05120830).

“We hope to demonstrate in our ongoing clinical trial that NTLA-2002 can result in deep and sustained kallikrein activity reduction following a single dose, and potentially prevent the unpredictable swelling attacks caused by this genetic disease,” said Intellia President and CEO John Leonard, MD. “We look forward to presenting interim data on September 16 at the 2022 Bradykinin Symposium, including safety, kallikrein reduction and HAE attack rate data.”

The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare disease or disorders that affect fewer than 200,000 individuals.

Reference

Intellia Therapeutics receives US FDA Orphan Drug designation for NTLA-2002, an investigational CRISPR therapy for the treatment of hereditary angioedema. News release. Intellia Therapeutics, Inc. Accessed September 2, 2022. https://www.globenewswire.com/news-release/2022/09/01/2508902/0/en/Intellia-Therapeutics-Receives-U-S-FDA-Orphan-Drug-Designation-for-NTLA-2002-an-Investigational-CRISPR-Therapy-for-the-Treatment-of-Hereditary-Angioedema.html