| # | Title | Journal | Year | Citations |
|---|
| 1 | Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template | Science Translational Medicine | 2015 | 204 |
| 2 | Biallelic loss of BCMA as a resistance mechanism to CAR T cell therapy in a patient with multiple myeloma | Nature Communications | 2021 | 173 |
| 3 | Progresses towards safe and efficient gene therapy vectors | Oncotarget | 2015 | 163 |
| 4 | Human GUCY2C-Targeted Chimeric Antigen Receptor (CAR)-Expressing T Cells Eliminate Colorectal Cancer Metastases | Cancer Immunology Research | 2018 | 100 |
| 5 | First-in-human multicenter study of bb2121 anti-BCMA CAR T-cell therapy for relapsed/refractory multiple myeloma: Updated results. | Journal of Clinical Oncology | 2017 | 76 |
| 6 | bb2121 anti-BCMA CAR T-cell therapy in patients with relapsed/refractory multiple myeloma: Updated results from a multicenter phase I study. | Journal of Clinical Oncology | 2018 | 50 |
| 7 | Pimodivir Treatment in Adult Volunteers Experimentally Inoculated with Live Influenza Virus: A Phase IIa, Randomized, Double-Blind, Placebo-Controlled Study | Antiviral Therapy | 2018 | 45 |
| 8 | Single-Cell Profiling of an In Vitro Model of Human Interneuron Development Reveals Temporal Dynamics of Cell Type Production and Maturation | Neuron | 2017 | 43 |
| 9 | Idecabtagene vicleucel (ide-cel, bb2121), a BCMA-directed CAR T cell therapy, in relapsed and refractory multiple myeloma: Updated KarMMa results. | Journal of Clinical Oncology | 2021 | 38 |
| 10 | An assessment of the hospital exemption landscape across European Member States: regulatory frameworks, use and impact | Cytotherapy | 2020 | 19 |
| 11 | 2021 White Paper on Recent Issues in Bioanalysis: ISR for Biomarkers, Liquid Biopsies, Spectral Cytometry, Inhalation/Oral & Multispecific Biotherapeutics, Accuracy/LLOQ for Flow Cytometry (Part 2 – Recommendations on Biomarkers/CDx Assays Development & Validation, Cytometry Validation & Innovation, Biotherapeutics PK LBA Regulated Bioanalysis, Critical Reagents & Positive Controls Generation) | Bioanalysis | 2022 | 10 |
| 12 | Economic evaluation of betibeglogene autotemcel (Beti-cel) gene addition therapy in transfusion-dependent β-thalassemia | Journal of Market Access & Health Policy | 2021 | 9 |
| 13 | KarMMa-4: Idecabtagene vicleucel (ide-cel, bb2121), a BCMA-directed CAR T-cell therapy in high-risk newly diagnosed multiple myeloma. | Journal of Clinical Oncology | 2021 | 9 |
| 14 | Early MRD negativity to predict deepening myeloma response in relapsed/refractory multiple myeloma (RRMM) patients treated with bb2121 anti-BCMA CAR T cells. | Journal of Clinical Oncology | 2018 | 7 |
| 15 | A First-in-Human Study To Assess the Safety and Pharmacokinetics of LYS228, a Novel Intravenous Monobactam Antibiotic in Healthy Volunteers | Antimicrobial Agents and Chemotherapy | 2019 | 6 |
| 16 | T-SIGn tumor reengineering therapy and CAR T cells synergize in combination therapy to clear human lung tumor xenografts and lung metastases in NSG mice | OncoImmunology | 2022 | 5 |
| 17 | An analysis of health-related quality of life in the phase III PROSELICA and FIRSTANA studies assessing cabazitaxel in patients with metastatic castration-resistant prostate cancer | ESMO Open | 2021 | 4 |
| 18 | Heterogeneity of the long-term economic burden of severe sickle cell disease: a 5-year longitudinal analysis | Journal of Medical Economics | 2022 | 4 |
| 19 | Interleukin-2 Therapy Induces CD4 Downregulation, Which Decreases Circulating CD4 T Cell Counts, in African Green Monkeys | Journal of Virology | 2016 | 3 |
| 20 | Treatment Patterns and Costs of Care in Commercially-Insured and Medicaid Patients with Transfusion-Dependent SS-Thalassemia | Value in Health | 2018 | 3 |
| 21 | Characteristics of neurotoxicity associated with idecabtagene vicleucel (ide-cel, bb2121) in patients with relapsed and refractory multiple myeloma (RRMM) in the pivotal phase II KarMMa study. | Journal of Clinical Oncology | 2021 | 3 |
| 22 | 250. A Phase 2/3 Study of the Efficacy and Safety of Ex Vivo Gene Therapy with Lenti-D TM Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy | Molecular Therapy | 2016 | 2 |
| 23 | US immigration order strikes against biotech | Nature Biotechnology | 2017 | 2 |
| 24 | 229. PGE2 Increases Lentiviral Vector Transduction Efficiency of Human HSC | Molecular Therapy | 2016 | 1 |
| 25 | 761. Targeted Killing of HIV Infected Cells Using CCR5-Disrupted Anti-HIV-CAR T Cells | Molecular Therapy | 2016 | 1 |
| 26 | PSY33 - FEASIBILITY OF EVALUATING THE BURDEN OF B-THALASSEMIA WITH GERMAN CLAIMS DATA | Value in Health | 2018 | 1 |
| 27 | The transfer of genetically engineered lymphocytes in melanoma patients: a Phase I dose escalation study | Value in Health | 2015 | 0 |
| 28 | 44. Novel Combination of megaTAL Nuclease-Driven Genome Engineering with a Drug Selection Cassette Increases Efficiency of HIV Gene Therapy | Molecular Therapy | 2016 | 0 |
| 29 | 277. Small Molecule-Regulated Antigen Recognition System for Inducible T Cell Targeting of Cancer Cells | Molecular Therapy | 2016 | 0 |
| 30 | LentiGlobin gene therapy for transfusion-dependent β-Thalassemia: Update from the northstar HGB-204 phase 1/2 clinical study | Cytotherapy | 2017 | 0 |
| 31 | Abstract MS01-1: Using T cell receptor engineered T cells for the treatment of cancer | Cytotherapy | 2013 | 0 |
| 32 | Association of high baseline ferritin with tocilizumab administration for CRS in relapsed/refractory multiple myeloma patients treated with bb2121 anti-BCMA CAR T cells. | Journal of Clinical Oncology | 2018 | 0 |
| 33 | Abstract 414: A Canine Myocardial Slice Model of Doxorubicin Induced Cardiotoxicity to Study Autophagy Modulation and Its Effect on Extracellular Vesicle Associated Mirna | Circulation Research | 2020 | 0 |
| 34 | IMMU-02. CHIMERIC ANTIGEN RECEPTOR (CAR) T CELL NEUROTOXICITY CORRELATES WITH PRETREATMENT AND ACUTE CSF NEUROFILAMENT LIGHT CHAIN (NFL) LEVELS | Neuro-Oncology | 2020 | 0 |
| 35 | Abstract 2324: Preclinical safety, biodistribution, and tumor infiltration analysis of CAR T cell targets using in situ hybridization technology | Neuro-Oncology | 2019 | 0 |
| 36 | Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop | Human Gene Therapy | 2023 | 0 |
