Intellia Therapeutics Doses First Patient in Landmark CRISPR/Cas9 Clinical Trial of NTLA-2001 for the Treatment of Transthyretin Amyloidosis
Following safety assessment and dose optimization, Intellia intends to further evaluate NTLA-2001 in a broader ATTR patient population of both polyneuropathy and cardiomyopathy patients.
- Following safety assessment and dose optimization, Intellia intends to further evaluate NTLA-2001 in a broader ATTR patient population of both polyneuropathy and cardiomyopathy patients.
- Once weve assessed safety and established an optimal dose, we intend to rapidly initiate trials for the clinical manifestations of ATTR.
- Intellias global Phase 1 trial is an open-label, multi-center, two-part study of NTLA-2001 in adults with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN).
- Intellia Therapeuticsis a leading genome editing company, focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system.