CTX001 Clinical Trial: Patient Data Shows Promising Results The promising progress of Victoria Gray, the first trial participant for sickle cell disease treatment, a year after her treatment this July had already kindled hopes of CTX001 as an effective therapy. As per the data presented at the...
“What we have been able to do through this study is a tremendous achievement,” said investigator Haydar Frangoul of the Sarah Cannon Research Institute, who presented the results at ASH. “By gene editing the patient’s own stem cells we may have the potential to make this therapy an op...
We present preliminary results from this Phase 2 study. Methods: This open-label, multi-center, single-arm Phase 2 study investigated pts with unresectable advanced, metastatic or recurrent BTC in advanced 2L or 3L setting. Eligible pts were treated with CTX-009 (10 mg/kg IV biweekly) in ...