2021
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
2020
Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy (GT): Updated Results in Group C Patients from the Phase 1/2 Hgb-206 Study
Walters M, Kanter J, Kwiatkowski J, Krishnamurti L, Mapara M, Schmidt M, Miller A, Pierciey F, Bonner M, Huang W, Ribeil J, Thompson A, Tisdale J. Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy (GT): Updated Results in Group C Patients from the Phase 1/2 Hgb-206 Study. Transplantation And Cellular Therapy 2020, 26: s1-s2. DOI: 10.1016/j.bbmt.2019.12.136.Peer-Reviewed Original ResearchPeripheral blood mononuclear cellsGroup C patientsAdverse eventsC patientsMedian HbTotal HbPlerixafor mobilizationNon-hematologic gradeGene therapySerious adverse eventsBlood mononuclear cellsLentiviral vectorsStrong therapeutic benefitFebrile neutropeniaSevere SCDConclusions PatientsGraft failureInitial patientsLast visitMononuclear cellsBusulfan conditioningHemolysis markersAutologous CD34Colony-forming unit assaysTherapeutic benefit
2019
Lentiglobin Gene Therapy in Patients with Sickle Cell Disease: Updated Interim Results from Hgb-206
Mapara M, Tisdale J, Kanter J, Kwiatkowski J, Krishnamurti L, Schmidt M, Miller A, Pierciey F, Shi W, Ribeil J, Asmal M, Thompson A, Walters M. Lentiglobin Gene Therapy in Patients with Sickle Cell Disease: Updated Interim Results from Hgb-206. Transplantation And Cellular Therapy 2019, 25: s64-s65. DOI: 10.1016/j.bbmt.2018.12.147.Peer-Reviewed Original ResearchSickle cell diseaseGrade 3 adverse eventsAdverse eventsHematopoietic stem cellsGrp BC patientsCell diseaseSevere sickle cell diseaseGene therapyNon-hematologic gradeVaso-occlusive painPhase 1 studyLentiviral vectorsAutologous hematopoietic stem cellsGrp CFebrile neutropeniaMyeloablative conditioningClinical effectsHb levelsLast visitBusulfan conditioningAutologous CD34Treatment characteristicsMethods AdultsPatients
2015
Initial Results from Study Hgb-206: A Phase 1 Study Evaluating Gene Therapy By Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the Lentiglobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease
Kanter J, Walters M, Hsieh M, Thompson A, Krishnamurti L, Kwiatkowski J, Kamble R, von Kalle C, Kuypers F, Cavazzana M, Leboulch P, Sandler L, Soni S, Tisdale J. Initial Results from Study Hgb-206: A Phase 1 Study Evaluating Gene Therapy By Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the Lentiglobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease. Blood 2015, 126: 3233. DOI: 10.1182/blood.v126.23.3233.3233.Peer-Reviewed Original ResearchSevere SCDSevere sickle cell diseaseSickle cell diseaseCell diseaseBluebird BioAdverse eventsAutologous CD34Additional subjectsLentiviral vectorsBone marrow harvestVector copy numberIntegration site analysisAdvisory CommitteeGene therapyHematologic engraftmentPRBC transfusionIntravenous busulfanChronic transfusionHb levelsSafety profileAutologous transplantationCell doseStudy visitMonth postProduct infusion