Intellia Genome Candidate Receives FDA's Orphan Drug Designation

 

Intellia Therapeutics Inc. said Thursday that the Food and Drug Administration granted NTLA-2002 orphan drug designation. The clinical stage genome editing company said NTLA-2002, its genome editing candidate, is currently being evaluated in a Phase 1/2 study in adults with Type I or Type II hereditary angioedema, a genetic disorder characterized by recurring and unpredictable inflammatory attacks. Intellia said NTLA-2002 is designed to knock out the target gene kallikrein B1 to reduce plasma kallikrein activity and thus prevent hereditary angioedema attacks. Chief Executive John Leonard said the company will present interim data on safety, kallikrein reduction and hereditary angioedema attack rate data on Sept. 16 at the 2022 Bradykinin Symposium. https://www.marketscreener.com/quote/stock/INTELLIA-THERAPEUTICS-IN-27506682/news/Intellia-Therapeutics-NTLA-2002-Genome-Candidate-Receives-FDA-s-Orphan-Drug-Designation-41675278/