NEW YORK – Intellia Therapeutics on Wednesday said it has dosed the first hereditary angioedema patient within the Phase III HAELO trial of the investigational in vivo CRISPR-based gene-editing therapy, NTLA-2002.
Intellia expects to submit a biologics license application to the US Food and Drug Administration for NTLA-2002 next year to support its plans for its first US commercial launch in 2027.
"We are pleased to have initiated dosing in the HAELO Phase III study as we are in our final lap of clinical development for NTLA-2002," Intellia President and CEO John Leonard said in a statement.
NTLA-2002, which is wholly owned by Cambridge, Massachusetts-based Intellia, is designed as a one-time treatment for hereditary angioedema, a rare genetic disease characterized by severe and unpredictable inflammatory attacks. NTLA-2002 aims to prevent these attacks by inactivating the KLKB1 gene, which plays a critical role in the body's inflammatory response.
In the global randomized, controlled HAELO trial, investigators will evaluate the efficacy and safety of NTLA-2002 in 60 adults with type I or type II hereditary angioedema, monitor the number of hereditary angioedema attacks patients experience, and track the number of patients who achieve attack-free status through 28 weeks posttreatment. Intellia expects to complete enrollment later this year.
Patients who receive a placebo within the HAELO trial will be eligible to receive NTLA-2002 within an optional crossover portion of the study after 28 weeks.