Research graph
References from Designs of the clinical trials aiming at evaluating cell and gene therapy products: A critical appraisal from a literature review. Local targets link to admitted publications; unresolved targets remain external evidence.
Challenges with advanced therapy medicinal products and how to meet them
10.1038/nrd3052 · 2010 · External reference
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2023 · External reference
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The state of cell and gene therapy in 2023
10.1016/j.ymthe.2023.11.001 · 2023 · External reference
CAR-T cell therapy: current limitations and potential strategies
10.1038/s41408-021-00459-7 · 2021 · External reference
The Relevance of Advanced Therapy Medicinal Products in the Field of Transplantation and the Need for Academic Research Access: Overcoming Bottlenecks and Claiming a New Time
10.3389/ti.2023.11633 · 2023 · External reference
A decade of marketing approval of gene and cell-based therapies in the United States, European Union and Japan: An evaluation of regulatory decision-making
10.1016/j.jcyt.2018.03.038 · 2018 · External reference
A Worldwide Overview of Regulatory Frameworks for Tissue-Based Products
10.1089/ten.teb.2019.0315 · 2020 · External reference
EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation
10.1016/j.omtm.2019.01.010 · 2019 · External reference
Comparison of the new Japanese legislation for expedited approval of regenerative medicine products with the existing systems in the USA and European Union
10.1002/term.2428 · 2018 · External reference
Mitigating Deficiencies in Evidence during Regulatory Assessments of Advanced Therapies: A Comparative Study with Other Biologicals
10.1016/j.omtm.2020.05.035 · 2020 · External reference
Marketing Regulatory Oversight of Advanced Therapy Medicinal Products (ATMPs) in Europe: The EMA/CAT Perspective
2015 · External reference
Requirements for Clinical Trials with Gene Therapy and Transplant Products in Switzerland
2015 · External reference
Regulation of Clinical Trials with Advanced Therapy Medicinal Products in Germany
2015 · External reference
Overview of the Regulatory Oversight Implemented by the French Regulatory Authorities for the Clinical Investigation of Gene Therapy and Cell Therapy Products
2015 · External reference
Unresolved reference
External reference
Advanced therapy medicinal products: current and future perspectives
2016 · External reference
A pipeline analysis of advanced therapy medicinal products
10.1016/j.drudis.2023.103549 · 2023 · External reference
Clinical holds for cell and gene therapy trials: Risks, impact, and lessons learned
10.1016/j.omtm.2023.101125 · 2023 · External reference
Regulation of advanced therapies in Europe: Are we on the right track?
10.1016/j.stem.2023.07.004 · 2023 · External reference
Unresolved reference
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Unresolved reference
2024 · External reference
Current landscape of clinical development and approval of advanced therapies
10.1016/j.omtm.2021.11.003 · 2021 · External reference
Methodological Characteristics of Clinical Trials Supporting the Marketing Authorisation of Advanced Therapies in the European Union
10.3389/fphar.2021.773712 · 2021 · External reference
A novel adoptive synthetic TCR and antigen receptor (STAR) T-Cell therapy for B-Cell acute lymphoblastic leukemia
10.1002/ajh.26586 · 2022 · External reference
A novel antibody-TCR (AbTCR) T-cell therapy is safe and effective against CD19-positive relapsed/refractory B-cell lymphoma
10.1007/s00432-022-04132-9 · 2023 · External reference
A novel therapeutic management for diabetes patients with chronic limb-threatening ischemia: comparison of autologous bone marrow mononuclear cells versus allogenic Wharton jelly-derived mesenchymal stem cells
10.1186/s13287-023-03427-z · 2023 · External reference
A phase 1 clinical trial of NKTR-255 with CD19-22 CAR T-cell therapy for refractory B-cell acute lymphoblastic leukemia
10.1182/blood.2024024952 · 2024 · External reference
A phase 1/2 clinical trial of invariant natural killer T cell therapy in moderate-severe acute respiratory distress syndrome
10.1038/s41467-024-44905-z · 2024 · External reference
A phase 2 study of axicabtagene ciloleucel in relapsed or refractory large B-cell lymphoma in Japan: 1-year follow-up and biomarker analysis
10.1007/s12185-022-03494-7 · 2023 · External reference
A Phase 2 Study of In Situ Oncolytic Virus Therapy and Stereotactic Body Radiation Therapy Followed by Pembrolizumab in Metastatic Non-Small Cell Lung Cancer
10.1016/j.ijrobp.2023.08.044 · 2024 · External reference
A Phase 2 Trial of Enhancing Immune Checkpoint Blockade by Stereotactic Radiation and In Situ Virus Gene Therapy in Metastatic Triple-Negative Breast Cancer
10.1158/1078-0432.ccr-22-0622 · 2022 · External reference
A phase I clinical trial of oncolytic adenovirus mediated suicide and interleukin-12 gene therapy in patients with recurrent localized prostate adenocarcinoma
10.1371/journal.pone.0291315 · 2023 · External reference
A Phase I Study of Locoregional High-Dose Autologous Natural Killer Cell Therapy With Hepatic Arterial Infusion Chemotherapy in Patients With Locally Advanced Hepatocellular Carcinoma
10.3389/fimmu.2022.879452 · 2022 · External reference
A Phase I Study to Determine the Maximum Tolerated Dose of ex Vivo Expanded Natural Killer Cells Derived from Unrelated, HLA-Disparate Adult Donors. Transplant
2022 · External reference
A phase I trial of autologous RAK cell immunotherapy in metastatic renal cell carcinoma
10.1007/s00262-024-03680-y · 2024 · External reference
A phase I/II study of adoptive SARS-CoV-2-specific T cells in immunocompromised hosts with or at risk of severe COVID-19 infection
10.1016/j.jcyt.2024.05.014 · 2024 · External reference
A Phase I/IIa Randomized Trial Evaluating the Safety and Efficacy of SNK01 Plus Pembrolizumab in Patients with Stage IV Non-Small Cell Lung Cancer
10.4143/crt.2021.986 · 2022 · External reference
A Phase IB/IIA Study of Ex Vivo Expanded Allogeneic Bone Marrow-Derived Mesenchymal Stem Cells for the Treatment of Perianal Fistulizing Crohn’s Disease
10.1097/dcr.0000000000002567 · 2023 · External reference
A phase IB/IIA study of ex vivo expanded allogeneic bone marrow-derived mesenchymal stem cells for the treatment of rectovaginal fistulizing Crohn’s disease
10.1016/j.surg.2023.07.020 · 2024 · External reference
A pilot clinical trial of cell therapy in heart failure with preserved ejection fraction
10.1002/ejhf.2596 · 2022 · External reference
A pilot study of multi-antigen stimulated cell therapy-I plus camrelizumab and apatinib in patients with advanced bone and soft-tissue sarcomas
10.1186/s12916-023-03132-x · 2023 · External reference
A randomized phase II clinical trial of stereotactic body radiation therapy (SBRT) and systemic pembrolizumab with or without intratumoral avelumab/ipilimumab plus CD1c (BDCA-1)+/CD141 (BDCA-3)+ myeloid dendritic cells in solid tumors
10.1007/s00262-024-03751-0 · 2024 · External reference
A randomized placebo-controlled phase 3 study of mesenchymal stem cells induced to secrete high levels of neurotrophic factors in amyotrophic lateral sclerosis
10.1002/mus.27472 · 2022 · External reference
A Randomized Trial of Mesenchymal Stromal Cells for Moderate to Severe Acute Respiratory Distress Syndrome from COVID-19
10.1164/rccm.202201-0157oc · 2023 · External reference
A Randomized, Placebo-Controlled, Phase II Trial of Intravenous Allogeneic Non-HLA Matched, Unrelated Donor, Cord Blood Infusion for Ischemic Stroke
10.1093/stcltm/szad080 · 2024 · External reference
AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patient
10.1038/s41591-024-03078-4 · 2024 · External reference
AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial
10.1016/s0140-6736(23)02874-x · 2024 · External reference
Adeno-Associated Virus Serotype 2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two-Year Interim Results of an Ongoing Phase I/II Gene Therapy Trial
10.1016/j.ophtha.2022.06.006 · 2022 · External reference
Adjuvant dendritic cell therapy in stage IIIB/C melanoma: the MIND-DC randomized phase III trial
10.1038/s41467-024-45358-0 · 2024 · External reference
Administration of an adeno-associated viral vector expressing interferon-β in patients with inflammatory hand arthritis, results of a phase I/II study
10.1016/j.joca.2021.09.013 · 2022 · External reference
Adoptive transfer of CMV-specific TCR-T cells for the treatment of CMV infection after haploidentical hematopoietic stem cell transplantation
10.1136/jitc-2023-007735 · 2024 · External reference
Allogeneic BCMA-targeting CAR T cells in relapsed/refractory multiple myeloma: phase 1 UNIVERSAL trial interim results
10.1038/s41591-022-02182-7 · 2023 · External reference
Allogeneic double-negative T cell therapy for relapsed acute myeloid leukemia patients post allogeneic hematopoietic stem cell transplantation: A first-in-human phase I study
10.1002/ajh.26564 · 2022 · External reference
Allogeneic mesenchymal stem cell therapy for dry eye disease in patients with Sjögren’s syndrome: A randomized clinical trial
10.1016/j.jtos.2023.11.007 · 2024 · External reference
Allogeneic Stem Cell Therapy for Acute Ischemic Stroke: The Phase 2/3 TREASURE Randomized Clinical Trial
10.1001/jamaneurol.2023.5200 · 2024 · External reference
Allogenic mesenchymal stromal cells and their extracellular vesicles in COVID-19 induced ARDS: a randomized controlled trial
10.1186/s13287-023-03402-8 · 2023 · External reference
An international learning collaborative phase 2 trial for haploidentical bone marrow transplant in sickle cell disease
10.1182/blood.2023023301 · 2024 · External reference
An open-label phase 1 clinical trial of the allogeneic side population adipose-derived mesenchymal stem cells in SMA type 1 patients
10.1007/s10072-021-05291-2 · 2022 · External reference
An Open-Label Phase II Study Assessing the Safety of Bilateral, Sequential Administration of Retinal Gene Therapy in Participants with Choroideremia: The GEMINI Study
10.1089/hum.2024.017 · 2024 · External reference
Angiogenic Gene Therapy for Refractory Angina: Results of the EXACT Phase 2 Trial
10.1161/circinterventions.124.014054 · 2024 · External reference
Anti-BCMA CAR T-cell therapy CT103A in relapsed or refractory AQP4-IgG seropositive neuromyelitis optica spectrum disorders: phase 1 trial interim results
10.1038/s41392-022-01278-3 · 2023 · External reference
Anti-CD19 and anti-BCMA CAR T cell therapy followed by lenalidomide maintenance after autologous stem-cell transplantation for high-risk newly diagnosed multiple myeloma
10.1002/ajh.26486 · 2022 · External reference
Anti-G Protein-Coupled Receptor, Class C Group 5 Member D Chimeric Antigen Receptor T Cells in Patients With Relapsed or Refractory Multiple Myeloma: A Single-Arm, Phase Ⅱ Trial
10.1200/jco.22.01824 · 2023 · External reference
Anti-PD-1 Therapy Enhances the Efficacy of CD30-Directed Chimeric Antigen Receptor T Cell Therapy in Patients With Relapsed/Refractory CD30+ Lymphoma
10.3389/fimmu.2022.858021 · 2022 · External reference
Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II interventional trial of pediatric patients with risk factor assessment
10.1038/s41467-024-47057-2 · 2024 · External reference
Artificial Dermis and Autologous Platelet-Rich Plasma for Treatment of Refractory Wounds: A Clinical Study
10.1177/15347346211050710 · 2024 · External reference
Assessment of Visual Function with Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa in the Randomized XIRIUS Phase 2/3 Study
10.1016/j.ophtha.2024.02.023 · 2024 · External reference
Autologous adipose-derived mesenchymal stem cell therapy reverses detrusor underactivity: open clinical trial
10.1186/s13287-023-03294-8 · 2023 · External reference
Autologous bone marrow mononuclear cell infusion for liver cirrhosis after the Kasai operation in children with biliary atresia
10.1186/s13287-022-02762-x · 2022 · External reference
Autologous CD7 CAR-T cells generated without T cell pre-selection in pediatric patients with relapsed/refractory T-ALL: A phase I trial
10.1016/j.ymthe.2024.09.006 · 2025 · External reference
Autologous CD34+ Stem Cell Therapy Increases Coronary Flow Reserve and Reduces Angina in Patients With Coronary Microvascular Dysfunction
10.1161/circinterventions.121.010802 · 2022 · External reference
Autologous mesenchymal stromal cells embedded with Tissucol Duo® for prevention of air leak after anatomical lung resection: results of a prospective phase I/II clinical trial with long-term follow-up
10.1186/s13287-023-03545-8 · 2023 · External reference
Autologous Muscle-Derived Cell Therapy for Swallowing Impairment in Patients Following Treatment for Head and Neck Cancer
10.1002/lary.29606 · 2022 · External reference
Autologous Nanobody-Derived Fratricide-Resistant CD7-CAR T-cell Therapy for Patients with Relapsed and Refractory T-cell Acute Lymphoblastic Leukemia/Lymphoma
10.1158/1078-0432.ccr-21-4097 · 2022 · External reference
Autologous T cell therapy for MAGE-A4+ solid cancers in HLA-A∗02+ patients: a phase 1 trial
10.1038/s41591-022-02128-z · 2023 · External reference
Axicabtagene ciloleucel as first-line therapy in high-risk large B-cell lymphoma: the phase 2 ZUMA-12 trial
10.1038/s41591-022-01731-4 · 2022 · External reference
Axicabtagene Ciloleucel as Second-Line Therapy for Large B-Cell Lymphoma
10.1056/nejmoa2116133 · 2022 · External reference
Axicabtagene ciloleucel in relapsed or refractory indolent non-Hodgkin lymphoma (ZUMA-5): a single-arm, multicentre, phase 2 trial
10.1016/s1470-2045(21)00591-x · 2022 · External reference
B-Cell Maturation Antigen/CD19 Dual-Targeting Immunotherapy in Newly Diagnosed Multiple Myeloma
10.1001/jamaoncol.2024.2172 · 2024 · External reference
BCMA-BBZ-OX40 CAR-T Therapy Using an Instant Manufacturing Platform in Multiple Myeloma
10.1136/jitc-2024-009476 · 2024 · External reference
Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia
10.1056/nejmoa2113206 · 2022 · External reference
Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial
10.1016/s0140-6736(24)01884-1 · 2024 · External reference
Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results
10.1038/s41591-024-03023-5 · 2024 · External reference
Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease
10.1056/nejmoa2117175 · 2022 · External reference
Bispecific CAR T cell therapy targeting BCMA and CD19 in relapsed/refractory multiple myeloma: a phase I/II trial
10.1038/s41467-024-47801-8 · 2024 · External reference
Bispecific CAR-T cells targeting CD19/20 in patients with relapsed or refractory B cell non-Hodgkin lymphoma: a phase I/II trial
10.1038/s41408-024-01105-8 · 2024 · External reference
Bone Marrow-Derived Mesenchymal Stromal Cell Therapy in Severe COVID-19: Preliminary Results of a Phase I/II Clinical Trial
10.3389/fimmu.2022.932360 · 2022 · External reference
CAR-T cell therapy targeting B cell maturation antigen is effective for relapsed/refractory multiple myeloma, including cases with poor performance status
10.1002/ajh.26583 · 2022 · External reference
CD20-specific chimeric antigen receptor-expressing T cells as salvage therapy in rituximab-refractory/relapsed B-cell non-Hodgkin lymphoma
10.1016/j.jcyt.2022.05.001 · 2022 · External reference
CD22-directed CAR T-cell therapy for large B-cell lymphomas progressing after CD19-directed CAR T-cell therapy: a dose-finding phase 1 study
10.1016/s0140-6736(24)00746-3 · 2024 · External reference
CD70-Targeted Allogeneic CAR T-Cell Therapy for Advanced Clear Cell Renal Cell Carcinoma
10.1158/2159-8290.cd-24-0102 · 2024 · External reference
Cell-Based Therapy by Autologous Bone Marrow-Derived Mononuclear Cells for Bone Augmentation of Plate-Stabilized Proximal Humeral Fractures: A Multicentric, Randomized, Open Phase IIa study
10.1093/stcltm/szad067 · 2024 · External reference
Chimeric Antigen Receptor T Cells Targeting CD19 and GCC in Metastatic Colorectal Cancer: A Nonrandomized Clinical Trial
10.1001/jamaoncol.2024.3891 · 2024 · External reference
Cholesterol efflux from C1QB-expressing macrophages is associated with resistance to chimeric antigen receptor T cell therapy in primary refractory diffuse large B cell lymphoma
10.1038/s41467-024-49495-4 · 2024 · External reference
Cilta-cel or Standard Care in Lenalidomide-Refractory Multiple Myeloma
10.1056/nejmoa2303379 · 2023 · External reference
Claudin18.2-specific CAR T cells in gastrointestinal cancers: phase 1 trial interim results
10.1038/s41591-022-01800-8 · 2022 · External reference
Clinical Evaluation of Safety and Efficacy of a Central Current Good Manufacturing Practices Laboratory Produced Autologous Adipose-Derived Stromal Vascular Fraction Cell Therapy Product for the Treatment of Knee Osteoarthritis
10.1089/scd.2024.0008 · 2024 · External reference
Clinical Safety and Efficacy of Locoregional Therapy Combined with Adoptive Transfer of Allogeneic γδ T Cells for Advanced Hepatocellular Carcinoma and Intrahepatic Cholangiocarcinoma
10.1016/j.jvir.2021.09.012 · 2022 · External reference
Coadministration of CD19- and CD22-Directed Chimeric Antigen Receptor T-Cell Therapy in Childhood B-Cell Acute Lymphoblastic Leukemia: A Single-Arm, Multicenter, Phase II Trial
10.1200/jco.22.01214 · 2023 · External reference
Combination Nivolumab, CD137 Agonism, and Adoptive Cell Therapy with Tumor-Infiltrating Lymphocytes for Patients with Metastatic Melanoma
10.1158/1078-0432.ccr-22-2103 · 2022 · External reference
Combined cytotoxic and immune-stimulatory gene therapy for primary adult high-grade glioma: a phase 1, first-in-human trial
10.1016/s1470-2045(23)00347-9 · 2023 · External reference
Combined therapy with CD4+ CD25highCD127- T regulatory cells and anti-CD20 antibody in recent-onset type 1 diabetes is superior to monotherapy: Randomized phase I/II trial
10.1111/dom.14723 · 2022 · External reference
Complementary Gene Therapy after Revascularization with the Saphenous Vein in Diabetic Foot Syndrome
10.3390/genes14101968 · 2023 · External reference
CYAD-01, an autologous NKG2D-based CAR T-cell therapy, in relapsed or refractory acute myeloid leukaemia and myelodysplastic syndromes or multiple myeloma (THINK): haematological cohorts of the dose escalation segment of a phase 1 trial
10.1016/s2352-3026(22)00378-7 · 2023 · External reference
Cytokine-mediated CAR T therapy resistance in AML
10.1038/s41591-024-03271-5 · 2024 · External reference
Danish phase II trial using adipose tissue derived mesenchymal stromal cells for patients with ischaemic heart failure
10.1002/ehf2.14281 · 2023 · External reference
Dendritic cell therapy with CD137L-DC-EBV-VAX in locally recurrent or metastatic nasopharyngeal carcinoma is safe and confers clinical benefit
10.1007/s00262-021-03075-3 · 2022 · External reference
Development and manufacture of novel locally produced anti-BCMA CAR T cells for the treatment of relapsed/refractory multiple myeloma: results from a phase I clinical trial
10.3324/haematol.2022.281628 · 2023 · External reference
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome
10.1126/scitranslmed.adi8214 · 2024 · External reference
Effect of autologous fat transfer in acute burn wound management: A randomized controlled study
10.1016/j.burns.2021.10.011 · 2022 · External reference
Effect of cell therapy with adipose-derived stem cells in the treatment of acute rupture of the Achilles tendon in humans
10.1007/s10561-024-10141-4 · 2024 · External reference
10.1136/jitc-2022-005755
10.1136/jitc-2022-005755 · 2022 · External reference
Efficacy of intrathecal mesenchymal stem cell-neural progenitor therapy in progressive MS: results from a phase II, randomized, placebo-controlled clinical trial
10.1186/s13287-024-03765-6 · 2024 · External reference
Efficacy of Intravesical Nadofaragene Firadenovec for Patients With Bacillus Calmette-Guérin-Unresponsive Nonmuscle-Invasive Bladder Cancer: 5-Year Follow-Up From a Phase 3 Trial
10.1097/ju.0000000000004020 · 2024 · External reference
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial
10.1016/s2352-3026(24)00006-1 · 2024 · External reference
Evaluation of neurotrophic factor secreting mesenchymal stem cells in progressive multiple sclerosis
10.1177/13524585221122156 · 2023 · External reference
Exagamglogene Autotemcel for Severe Sickle Cell Disease
10.1056/nejmoa2309676 · 2024 · External reference
Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia
10.1056/nejmoa2309673 · 2024 · External reference
Feasibility and Safety of Personalized, Multi-Target, Adoptive Cell Therapy (IMA101): First-in-Human Clinical Trial in Patients with Advanced Metastatic Cancer
10.1158/2326-6066.cir-22-0444 · 2023 · External reference
Feasibility of a Novel Academic BCMA-CART (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis
10.1158/1078-0432.ccr-22-0637 · 2022 · External reference
Feasibility of intranasal human milk as stem cell therapy in preterm infants with intraventricular hemorrhage
10.1038/s41372-024-01982-8 · 2024 · External reference
Final Results of Allogeneic Adipose Tissue-Derived Mesenchymal Stem Cells in Acute Ischemic Stroke (AMASCIS): A Phase II, Randomized, Double-Blind, Placebo-Controlled, Single-Center, Pilot Clinical Trial
10.1177/09636897221083863 · 2022 · External reference
First step results from a phase II study of a dendritic cell vaccine in glioblastoma patients (CombiG-vax)
10.3389/fimmu.2024.1404861 · 2024 · External reference
First-in-human dose escalation trial to evaluate the clinical safety and efficacy of an anti-MAGEA1 autologous TCR-transgenic T cell therapy in relapsed and refractory solid tumors
10.1136/jitc-2023-008668 · 2024 · External reference
First-in-Human Gene Therapy Trial of AAV8-hCARp.hCNGB3 in Adults and Children With CNGB3-associated Achromatopsia
10.1016/j.ajo.2023.05.009 · 2023 · External reference
First-in-human phase I study of CLL-1 CAR-T cells in adults with relapsed/refractory acute myeloid leukemia
10.1186/s13045-022-01308-1 · 2022 · External reference
Five-year follow-up of a phase I trial of donor-derived modified immune cell infusion in kidney transplantation
10.3389/fimmu.2023.1089664 · 2023 · External reference
Five-year follow-up of intracoronary autologous cell therapy in acute myocardial infarction: the REGENERATE-AMI trial
10.1002/ehf2.13786 · 2022 · External reference
Follow-up Study to Evaluate the Long-term Safety and Efficacy of Darvadstrocel (Mesenchymal Stem Cell Treatment) in Patients With Perianal Fistulizing Crohn’s Disease: ADMIRE-CD Phase 3 Randomized Controlled Trial
10.1097/dcr.0000000000002325 · 2022 · External reference
From Safety to Benefit in Cell Delivery During Surgical Repair of Ebstein Anomaly: Initial Results
10.1016/j.athoracsur.2020.11.065 · 2022 · External reference
GD2-CAR T cell therapy for H3K27M-mutated diffuse midline gliomas
10.1038/s41586-022-04489-4 · 2022 · External reference
GD2-specific chimeric antigen receptor-modified T cells for the treatment of refractory and/or recurrent neuroblastoma in pediatric patients
10.1007/s00432-021-03839-5 · 2022 · External reference
Gemcitabine, carboplatin, and Epstein-Barr virus-specific autologous cytotoxic T lymphocytes for recurrent or metastatic nasopharyngeal carcinoma: VANCE, an international randomized phase III trial
10.1016/j.annonc.2024.08.2344 · 2024 · External reference
Gene replacement therapy in Bietti crystalline corneoretinal dystrophy: an open-label, single-arm, exploratory trial
10.1038/s41392-024-01806-3 · 2024 · External reference
Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study
10.1016/s0140-6736(24)00310-6 · 2024 · External reference
Gene Therapy in Patients with the Crigler-Najjar Syndrome
10.1056/nejmoa2214084 · 2023 · External reference
Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results
10.1038/s41591-023-02730-9 · 2024 · External reference
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B
10.1056/nejmoa2211644 · 2023 · External reference
Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B
10.1056/nejmoa2302982 · 2024 · External reference
Genetically modified CD7-targeting allogeneic CAR-T cell therapy with enhanced efficacy for relapsed/refractory CD7-positive hematological malignancies: a phase I clinical study
10.1038/s41422-022-00721-y · 2022 · External reference
Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study
10.1182/blood.2022018971 · 2024 · External reference
GPRC5D CAR T cells (OriCAR-017) in patients with relapsed or refractory multiple myeloma (POLARIS): a first-in-human, single-centre, single-arm, phase 1 trial
10.1016/s2352-3026(22)00372-6 · 2023 · External reference
GPRC5D-Targeted CAR T Cells for Myeloma
10.1056/nejmoa2209900 · 2022 · External reference
HA-1-targeted T-cell receptor T-cell therapy for recurrent leukemia after hematopoietic stem cell transplantation
10.1182/blood.2024024105 · 2024 · External reference
Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials
10.1016/s0140-6736(24)01880-4 · 2025 · External reference
High Complete Response Rate in Patients With Metastatic Renal Cell Carcinoma Receiving Autologous Cytokine-Induced Killer Cell Therapy Plus Anti-Programmed Death-1 Agent: A Single-Center Study
2021 · External reference
HLA-mismatched allogeneic adoptive immune therapy in patients with severely immunosuppressed AIDS: a multicenter, open-label, controlled, phase 2a study
10.1080/22221751.2024.2364744 · 2024 · External reference
Human mesenchymal stem cell therapy in severe COVID-19 patients: 2-year follow-up results of a randomized, double-blind, placebo-controlled trial
10.1016/j.ebiom.2023.104600 · 2023 · External reference
Human placenta-derived mesenchymal stem cells transplantation in patients with acute respiratory distress syndrome (ARDS) caused by COVID-19 (phase I clinical trial): safety profile assessment
10.1186/s13287-022-02953-6 · 2022 · External reference
Human Umbilical Cord-Derived Mesenchymal Stem Cells in the Treatment of Multiple Sclerosis Patients: Phase I/II Dose-Finding Clinical Study
10.1177/09636897241233045 · 2024 · External reference
Ide-cel or Standard Regimens in Relapsed and Refractory Multiple Myeloma
10.1056/nejmoa2213614 · 2023 · External reference
Idecabtagene vicleucel for relapsed and refractory multiple myeloma: post hoc 18-month follow-up of a phase 1 trial
10.1038/s41591-023-02496-0 · 2023 · External reference
Iltamiocel Autologous Cell Therapy for the Treatment of Female Stress Urinary Incontinence: A Double-Blind, Randomized, Stratified, Placebo-Controlled Trial
10.1002/nau.25588 · 2024 · External reference
Immunogenicity of personalized dendritic-cell therapy in HIV-1 infected individuals under suppressive antiretroviral treatment: interim analysis from a phase II clinical trial. AIDS Res
2022 · External reference
IMPROvE-CED Trial: Intracoronary Autologous CD34+ Cell Therapy for Treatment of Coronary Endothelial Dysfunction in Patients With Angina and Nonobstructive Coronary Arteries
10.1161/circresaha.121.319644 · 2022 · External reference
In vivo CRISPR gene editing in patients with herpetic stromal keratitis
10.1016/j.ymthe.2023.08.021 · 2023 · External reference
In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial
10.1038/s41591-022-01737-y · 2022 · External reference
Inhaled mRNA therapy for treatment of cystic fibrosis: Interim results of a randomized, double-blind, placebo-controlled phase 1/2 clinical study
10.1016/j.jcf.2023.04.008 · 2023 · External reference
Interim safety and efficacy of gene therapy for RLBP1-associated retinal dystrophy: a phase 1/2 trial
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Intracerebral transplantation of MRI-trackable autologous bone marrow stromal cells for patients with subacute ischemic stroke
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Intrauterine infusion of clinically graded human umbilical cord-derived mesenchymal stem cells for the treatment of poor healing after uterine injury: a phase I clinical trial
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Late Treatment With Autologous Expanded Regulatory T-cell Therapy After Alemtuzumab Induction Is Safe and Facilitates Immunosuppression Minimization in Living Donor Renal Transplantation
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Left Atrial Structural and Functional Response in Kidney Transplant Recipients Treated With Mesenchymal Stromal Cell Therapy and Early Tacrolimus Withdrawal
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Lentiviral Gene Therapy for Artemis-Deficient SCID
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Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy
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Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial
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Lifileucel, an Autologous Tumor-Infiltrating Lymphocyte Monotherapy, in Patients with Advanced Non-Small Cell Lung Cancer Resistant to Immune Checkpoint Inhibitors
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Lisocabtagene maraleucel in follicular lymphoma: the phase 2 TRANSCEND FL study
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Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI
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Long-term remission and survival in patients with relapsed or refractory multiple myeloma after treatment with LCAR-B38M CAR T cells: 5-year follow-up of the LEGEND-2 trial
10.1186/s13045-024-01530-z · 2024 · External reference
Long-term safety and efficacy of gene-corrected autologous keratinocyte grafts for recessive dystrophic epidermolysis bullosa
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Mesenchymal stem cell therapy and cognition in MS: Preliminary findings from a phase II clinical trial
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Mesenchymal Stem Cell Therapy in Acute Intracerebral Hemorrhage: A Dose-Escalation Safety and Tolerability Trial
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Mesenchymal stem cell transplantation in newly diagnosed type-1 diabetes patients: a phase I/II randomized placebo-controlled clinical trial
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Mesenchymal Stem/Stromal Cell Therapy for Radiation-Induced Xerostomia in Previous Head and Neck Cancer Patients: A Phase II Randomized, Placebo-Controlled Trial
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Mesenchymal Stromal Cell Implants for Chronic Motor Deficits After Traumatic Brain Injury: Post Hoc Analysis of a Randomized Trial
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Mesenchymal stromal cells to treat patients with non-ischaemic heart failure: Results from SCIENCE II pilot study
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Modified Kasai operation combined with autologous bone marrow mononuclear cell infusion for biliary atresia
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Motor function and safety after allogeneic cord blood and cord tissue-derived mesenchymal stromal cells in cerebral palsy: An open-label, randomized trial
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Nasal septum-derived chondroprogenitor cells control mandibular condylar resorption consequent to orthognathic surgery: a clinical trial
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Neoantigen-targeted dendritic cell vaccination in lung cancer patients induces long-lived T cells exhibiting the full differentiation spectrum
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Non-viral precision T cell receptor replacement for personalized cell therapy
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Non-viral, specifically targeted CAR-T cells achieve high safety and efficacy in B-NHL
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Novel Autologous Dendritic Cell Therapy AVT001 for Type 1 Diabetes
10.1056/evidoa2300238 · 2024 · External reference
Obecabtagene Autoleucel in Adults with B-Cell Acute Lymphoblastic Leukemia
10.1056/nejmoa2406526 · 2024 · External reference
Off-the-shelf, steroid-resistant, IL13Rα2-specific CAR T cells for treatment of glioblastoma
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OUTREACH: phase 2 study of lisocabtagene maraleucel as outpatient or inpatient treatment at community sites for R/R LBCL
10.1182/bloodadvances.2024013254 · 2024 · External reference
PACE: randomized, controlled, multicentre, multinational, phase III study of PLX-PAD for critical limb ischaemia in patients unsuitable for revascularization: randomized clinical trial
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Phase 1 clinical trial to assess safety and efficacy of NY-ESO-1-specific TCR T cells in HLA-A∗02:01 patients with advanced soft tissue sarcoma
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10.1136/jitc-2022-005145 · 2022 · External reference
Phase 1 Study of CK0801 in Treatment of Bone Marrow Failure Syndromes
10.1056/evidoa2300362 · 2024 · External reference
Phase 1 Study of JNJ-81201887 Gene Therapy in Geographic Atrophy Secondary to Age-Related Macular Degeneration
10.1016/j.ophtha.2024.06.013 · 2024 · External reference
Phase 1 study to determine the safety and dosing of autologous PBMCs modified to present HPV16 antigens (SQZ-PBMC-HPV) in HLA-A∗02+ patients with HPV16+ solid tumors
10.1007/s10637-023-01342-x · 2023 · External reference
Phase 1 TRANSCEND CLL 004 study of lisocabtagene maraleucel in patients with relapsed/refractory CLL or SLL
10.1182/blood.2021011895 · 2022 · External reference
Phase 1 trial of 4-1BB-based adoptive T-cell therapy targeting human telomerase reverse transcriptase in patients with advanced refractory solid tumors
10.1016/j.jcyt.2023.07.006 · 2023 · External reference
Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B
10.1056/nejmoa2119913 · 2022 · External reference
Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-Associated X-Linked Retinitis Pigmentosa
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Phase 2 results of idecabtagene vicleucel (ide-cel, bb2121) in Japanese patients with relapsed and refractory multiple myeloma
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Phase I clinical trial of intracerebroventricular transplantation of allogeneic neural stem cells in people with progressive multiple sclerosis
10.1016/j.stem.2023.11.001 · 2023 · External reference
Phase I non-randomized clinical trial of allogeneic natural killer cells infusion in acute myeloid leukemia patients
10.1186/s12885-023-11610-x · 2023 · External reference
Phase I study of CBM.CD19 chimeric antigen receptor T cell in the treatment of refractory diffuse large B-cell lymphoma in Chinese patients
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Phase I study of expanded natural killer cells in combination with cetuximab for recurrent/metastatic nasopharyngeal carcinoma
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Phase I study of liver depot gene therapy in late-onset Pompe disease
10.1016/j.ymthe.2023.02.014 · 2023 · External reference
Phase I study of safety and efficacy of allogeneic natural killer cell therapy in relapsed/refractory neuroblastomas post autologous hematopoietic stem cell transplantation
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Phase I study targeting newly diagnosed grade 4 astrocytoma with bispecific antibody armed T cells (EGFR BATs) in combination with radiation and temozolomide
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Phase I Study: Safety and Efficacy of an Ex Vivo-Expanded Allogeneic Natural Killer Cell (MG4101) with Rituximab for Relapsed/Refractory B Cell Non-Hodgkin Lymphoma. Transplant
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Phase I Trial of Autologous RNA-electroporated cMET-directed CAR T Cells Administered Intravenously in Patients with Melanoma and Breast Carcinoma
10.1158/2767-9764.crc-22-0486 · 2023 · External reference
Phase I/IIa Feasibility Trial of Autologous Quality- and Quantity-Cultured Peripheral Blood Mononuclear Cell Therapy for Non-Healing Extremity Ulcers
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Phase II Study Shows the Effect of Adenoviral Vascular Endothelial Growth Factor C and Lymph Node Transfer in Lymphedema
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Phase II, Open-Label Study of Ciltacabtagene Autoleucel, an Anti-B-Cell Maturation Antigen Chimeric Antigen Receptor-T-Cell Therapy, in Chinese Patients With Relapsed/Refractory Multiple Myeloma (CARTIFAN-1)
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Phase III clinical trial of autologous CD34 + cell transplantation to accelerate fracture nonunion repair
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Point of care CD19 chimeric antigen receptor (CAR) T-cells for relapsed/refractory acute myeloid leukemia (AML) with aberrant CD19 antigen expression
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Point-of-care anti-CD19 chimeric antigen receptor T-cell therapy for relapsed/refractory follicular lymphoma
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Posoleucel, an Allogeneic, Off-the-Shelf Multivirus-Specific T-Cell Therapy, for the Treatment of Refractory Viral Infections in the Post-HCT Setting
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Progranulin AAV gene therapy for frontotemporal dementia: translational studies and phase 1/2 trial interim results
10.1038/s41591-024-02973-0 · 2024 · External reference
PSCA-CAR T cell therapy in metastatic castration-resistant prostate cancer: a phase 1 trial
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Quality-Adjusted Time without Symptoms or Toxicity: Analysis of Axicabtagene Ciloleucel versus Standard of Care in Patients with Relapsed/Refractory Large B Cell Lymphoma. Transplant
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Randomized Phase II Trial of Dendritic Cell/Myeloma Fusion Vaccine with Lenalidomide Maintenance after Upfront Autologous Hematopoietic Cell Transplantation for Multiple Myeloma: BMT CTN 1401
10.1158/1078-0432.ccr-23-0235 · 2023 · External reference
Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy
10.1093/brain/awac421 · 2023 · External reference
Randomized Trial of Targeted Transendocardial Mesenchymal Precursor Cell Therapy in Patients With Heart Failure
10.1016/j.jacc.2022.11.061 · 2023 · External reference
Renal Autologous Cell Therapy in Type 2 Diabetes with Late Stage 4 Diabetes-Related Chronic Kidney Disease: Trial Design and Early Analysis
10.1159/000527582 · 2023 · External reference
Repeat infusion of autologous bone marrow cells in progressive multiple sclerosis - A phase I extension study (SIAMMS II)
10.1016/j.msard.2022.103782 · 2022 · External reference
Repeated Infusions of Bone-Marrow-Derived Mesenchymal Stem Cells over 8 Weeks for Steroid-Refractory Chronic Graft-versus-Host Disease: A Prospective, Phase I/II Clinical Study
10.3390/ijms25126731 · 2024 · External reference
Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial
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Repeated peripheral infusions of anti-EGFRvIII CAR T cells in combination with pembrolizumab show no efficacy in glioblastoma: a phase 1 trial
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Report of a phase 1 clinical trial for safety assessment of human placental mesenchymal stem cells therapy in patients with critical limb ischemia (CLI)
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Results from a Phase 1 Study of ACTR707 in Combination with Rituximab in Patients with Relapsed or Refractory CD20+ B Cell Lymphoma. Transplant
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Safety and Potential Efficacy of Expanded Umbilical Cord-Derived Mesenchymal Stromal Cells in Luminal Ulcerative Colitis Patients
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Safety and Preliminary Efficacy of Mesenchymal Stromal Cell (ORBCEL-M) Therapy in Diabetic Kidney Disease: A Randomized Clinical Trial (NEPHSTROM)
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Safety evaluation of bi-layered allogenic keratinocyte and fibroblast skin substitute for diabetic foot ulcers-SAFESKIN-DFU: A Phase 1 clinical trial
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Safety of Human Embryonic Stem Cell-derived Mesenchymal Stem Cells for Treating Interstitial Cystitis: A Phase I Study
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Safety of Stromal Vascular Fraction Cell Therapy for Chronic Kidney Disease of Unknown Cause (Mesoamerican Nephropathy)
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Scaphoid Bone Nonunions: Clinical and Functional Outcomes of Collagen/PGA Scaffolds and Cell-Based Therapy
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Second-Line Tisagenlecleucel or Standard Care in Aggressive B-Cell Lymphoma
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Sequential different B-cell antigen-targeted CAR T-cell therapy for pediatric refractory/relapsed Burkitt lymphoma
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Sintilimab plus autologous NK cells as second-line treatment for advanced non-small-cell lung cancer previous treated with platinum-containing chemotherapy
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Sleeping beauty generated CD19 CAR T-Cell therapy for advanced B-Cell hematological malignancies
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Suicide gene therapy using allogeneic adipose tissue-derived mesenchymal stem cell gene delivery vehicles in recurrent glioblastoma multiforme: a first-in-human, dose-escalation, phase I clinical trial
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Survival with Axicabtagene Ciloleucel in Large B-Cell Lymphoma
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Systemic Administration of Allogeneic Cord Blood Mononuclear Cells in Adults with Severe Acute Contusion Spinal Cord Injury: Phase 1/2a Pilot Clinical Study-Safety and Primary Efficacy Evaluation
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Targeting refractory/recurrent neuroblastoma and osteosarcoma with anti-CD3×anti-GD2 bispecific antibody armed T cells
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Transplantation of chemically induced pluripotent stem-cell-derived islets under abdominal anterior rectus sheath in a type 1 diabetes patient
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Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa
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Tumor-Infiltrating Lymphocyte Therapy or Ipilimumab in Advanced Melanoma
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Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A
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Valoctocogene roxaparvovec gene therapy provides durable haemostatic control for up to 7 years for haemophilia A
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