CRISPR Gene Editing Therapy Can Be Administered Twice, Intellia Study Suggests

1. Intellia Study: A small study by Intellia Therapeutics suggests that CRISPR gene editing therapy can be given twice to patients.
2. Transthyretin Amyloidosis: The study involved patients with transthyretin amyloidosis, a condition that can cause cardiomyopathy and polyneuropathy.
3. Re-dosing: Three patients who initially received a low dose of the CRISPR therapy were given a second, higher dose, which resulted in a significant reduction in serum TTR protein levels.
4. Safety: The re-dosing was generally well tolerated, with one patient experiencing a mild infusion-related reaction.
5. Implications: This study provides the first clinical data showing that a gene editing therapy can be administered twice, opening up possibilities for treating other diseases where multiple doses may be needed.