Results from a proof-of-concept study of Orchard Therapeutics' (ORTX) gene therapy candidate OTL-300 in transfusion-dependent patients with an inherited blood disorder called beta thalassemia showed a positive effect. Reductions in transfusions at month 12 were achieved in eight of the nine participants. 67% (n=4/6) of the pediatric patients treated achieved transfusion independence while one experienced a lower need for transfusions. Source: Seeking Alpha 29/4/2019