Strong Enrollment in Phase III Trials
Enrollment in Phase III studies for hereditary angioedema (HAE) and transthyretin amyloidosis (ATTR) is progressing well, with expectations to exceed 550 patients by year-end in the MAGNITUDE trial.
Positive Data from Phase I Trials
Phase I data for NTLA-2001 showed a mean serum TTR reduction of 90% at 12 months, indicating potential disease stabilization or improvement for ATTR amyloidosis patients.
Robust Financial Position
Intellia reported approximately $861.7 million in cash, cash equivalents, and marketable securities as of December 31, 2024, providing a solid financial foundation to fund operations into 2027.
Regenerative Medicine Advanced Therapy Designation
Nex-z received FDA's RMAT designation for ATTR amyloidosis, facilitating close collaboration with the FDA towards a BLA filing by 2028.