Intellia Becomes First To Show Potential For CRISPR Therapy Re-Dosing

The company presented data showing it is possible to safely and effectively re-dose its ATTR amyloidosis CRISPR/Cas9-based therapy.

The common description of gene therapies as “one-time treatments” may need a bit of reassessment as new data from Intellia Therapeutics, Inc.’s Phase I study of NTLA-2001 in transthyretin amyloidosis (ATTR) show that it is possible to safely administer more than one dose of a CRISPR/Cas9 in vivo gene-editing therapy. The findings especially have significant implications for CRISPR/Cas9 therapies for diseases that originate outside the liver.

Intellia presented proof-of-concept data for redosing NTLA-2001 on 25 June at the Peripheral Nerve Society annual meeting in Montreal. NTLA-2001,...