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Intellia Therapeutics Receives U.S. FDA Orphan Drug Designation for NTLA-2002, an Investigational CRISPR Therapy for the Treatment of Hereditary Angioedema
NTLA-2002, an in vivo genome editing candidate designed to prevent angioedema attacks in patients with hereditary angioedema (HAE) after a single dose, is
About this update from Intellia Therapeutics, Inc.
[{"type":"text","content":"NTLA-2002, an in vivo genome editing candidate designed to prevent angioedema attacks in patients with hereditary angioedema (HAE) after a single dose, is currently being evaluated in a Phase 1/2 study CAMBRIDGE, Mass., Sept. 01, 2022 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing potentially curative therapies leveraging CRISPR-based technologies, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for Intellia’s in vivo CRISPR/Cas9 genome editing candidate, NTLA-2002, for the treatment of hereditary angioedema (HAE). NTLA-2002 is a systemically administered investigational therapy designed to knockout the target gene kallikrein B1 (KLKB1) to reduce plasma kallikrein activity and thus prevent HAE attacks. NTLA-2002 is currently being evaluated in a Phase 1/2 study in adults with Type I or Type II HAE. “Orphan drug designation represents an important milestone in the development of NTLA-2002 and underscores the importance of developing innovative, new treatment options for people living with HAE,” said Intellia President and Chief Executive Officer John Leonard, M.D. “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. 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 Program provides orphan status to drugs intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user-fee exemptions and seven-year marketing exclusivity upon FDA approval. About the NTLA-2002 Clinical Program Intellia’s multi-national Phase 1/2 study is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in adults with Type I or Type II hereditary angioedema (HAE). This includes the measurement of plasma kallikrein protein levels a...