Clinical trial results published by Intellia Therapeutics

Intellia's CRISPR Treatment for 36 ATTR-CM Patients Shows Superior Results, Reports High Adverse Events Over 12 Months

Nov 16, 2024, 03:30 PM

Intellia Therapeutics has announced that its CRISPR treatment for transthyretin amyloid cardiomyopathy (ATTR-CM) has outperformed conventional therapies. In a Phase 1 open-label study involving 36 patients, the treatment demonstrated a consistent reduction in serum TTR levels over 12 months. However, the study also reported a high rate of adverse events, attributed to a combination of the patients' advanced underlying conditions and the therapy itself. The median six-minute walk test (6MWT) results indicated a gain over baseline at the 12-month mark, suggesting improved patient outcomes. This development is significant for patients suffering from ATTR-CM, a condition that can lead to severe heart complications.