Preliminary Safety and Efficacy Results from Precizn-1: An Ongoing Phase 1/2 Study on Zinc Finger Nuclease-Modified Autologous CD34+ HSPCs for Sickle Cell Disease (SCD)
Alavi A, Krishnamurti L, Abedi M, Galeon I, Reiner D, Smith S, Wang L, Ramezi A, Rendo P, Walters M. Preliminary Safety and Efficacy Results from Precizn-1: An Ongoing Phase 1/2 Study on Zinc Finger Nuclease-Modified Autologous CD34+ HSPCs for Sickle Cell Disease (SCD). Blood 2021, 138: 2930. DOI: 10.1182/blood-2021-151650.Peer-Reviewed Original ResearchSickle cell diseaseVaso-occlusive crisisAdverse eventsAutologous CD34Apheresis cyclesWeek 26Cell diseaseSpeakers bureauTotal HbOngoing phase 1/2 studySevere sickle cell diseaseF cellsPhase 1/2 studyBaseline patient characteristicsRelated adverse eventsHealth-related qualityPeripheral blood WBCCurrent unmet needPotential therapeutic valueEarly termination visitMinimum cell doseStem cell engraftmentTrend of improvementCurrent employmentElevated fetal hemoglobin