Lead Product(s): BAY2599023
Therapeutic Area: Genetic Disease Product Name: DTX201
Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Ultragenyx Pharmaceutical
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 24, 2020
Two of the six patients treated to date in this clinical study, both given the therapy at lowest dose, are still showing FVIII activity at more than one year post-treatment, its investigators report.