A genome editing therapy designed to inactivate a mutated gene in the liver has reported positive Phase 1 data – the first therapy of its kind to achieve an effect inside the human body. The drug, NTLA-2001, is intended as a one-time treatment for people living with transthyretin (ATTR) amyloidosis, a rare disease caused by misfolded proteins. The study results were reported on 26 June by Intellia Therapeutics Inc and its partner Regeneron Pharmaceuticals Inc, and simultaneously published in The New England Journal of Medicine.
