Author Archives: David Melamed, PhD

UK Approves Phase 1 Trial of Potential Gene Editing Therapy, NTLA-2001

A Phase 1 trial of NTLA-2001, a potential gene editing therapy for  familial amyloid polyneuropathy (FAP), was approved to open by the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA). Intellia Therapeutics is planning to launch the open-label and two-part trial in people with hereditary transthyretin amyloidosis (ATTR) with polyneuropathy before…