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Journal Articles

RP-L301, a Lentiviral-Mediated Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase 2 Clinical Trial Design

Ami J Shah, José Luis López Lorenzo, Rachael F Grace, E J van Beers, M Bartels, Andreas Glenthoej, Susana Navarro, Lucia Llanos, Begoña Pérez de Camino Gaisse, Sol Sanchez, Josune Zubicaray, May Chien, Oscar Quintana Bustamante, M González-Vicent, June Iriondo, Miriam Zeini, Grace Choi, Eileen Nicoletti, Maria Chitty-Lopez, Gayatri R Rao, Juan Bueren, Jonathan D Schwartz, Jose Carlos Segovia, Julián Sevilla
Journal: Blood
Blood (2024) 144 (Supplement 1): 3583.1.
https://doi.org/10.1182/blood-2024-201734
Published: 2024
Journal Articles

Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group a]: Updated Results from Global RP-L102 Clinical Trials

Agnieszka D. Czechowicz, Julián Sevilla, Claire Booth, Josune Zubicaray, Paula Rio, Susana Navarro, Kritika Cherry, Gráinne O'Toole, Jinhua Xu-Bayford, Philip Ancliff, Elena Sebastián, Michael Rothe, Antonella L Bastone, Philipp John-Neek, Axel Schambach, Wei Wang, Grace Choi, Miriam Zeini, Eileen Nicoletti, Cindy Eide, John E Wagner, Gayatri R Rao, Adrian J. Thrasher, Maria-Grazia Roncarolo, Jonathan D Schwartz, Juan A Bueren, Rajni Agarwal
Journal: Blood
Blood (2024) 144 (Supplement 1): 7463.
https://doi.org/10.1182/blood-2024-194638
Published: 2024
Journal Articles

Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT) Outcomes in Leukocyte Adhesion Deficiency Type -I

Claire Booth, Patrick Gilbert, Luuk Gras, Hawazen Alsaedi, Amir Ali Hamidieh, Mor Seny Gueye, Mary Slatter, Ashrafsadat Mousavi, Amos Toren, Musa Karakukcu, Abdelghani Tbakhi, Polina Stepensky, Gulyuz Ozturk, Peter Bader, Henrik Sengeloev, Tayfun Güngör, R.F. Wynn, Franco Locatelli, Maria Chitty-Lopez, Joana E Matos, Stuart Turner, Gayatri R Rao, Jonathan D Schwartz, Michael H Albert, Bénédicte Neven
Journal: Blood
Blood (2024) 144 (Supplement 1): 5079.
https://doi.org/10.1182/blood-2024-203643
Published: 2024
Journal Articles

Lentiviral-mediated Gene Therapy for Adults and Children with Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study

Ami J. Shah, José Luis López Lorenzo, Julián Sevilla, Susana Navarro, Lucía Llanos, Begoña Pérez de Camino Gaisse, Sol Sanchez, Josune Zubicaray, Bert Glader, May Chien, Oscar Quintana Bustamante, Miriam Zeini, Grace Choi, Eileen Nicoletti, Gayatri R. Rao, Maria Grazia Roncarolo, Juan A. Bueren, Jonathan D. Schwartz, José C. Segovia
Journal: Blood
Blood (2022) 140 (Supplement 1): 4902–4903.
https://doi.org/10.1182/blood-2022-170948
Published: 2022
Journal Articles

Lentiviral-mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials

Agnieszka Czechowicz, Julián Sevilla, Claire Booth, Rajni Agarwal, Josune Zubicaray, Paula Río, Susana Navarro, Kritika Chetty, Grainne O'Toole, Jinhua Xu-Bayford, Philip Ancliff, Elena Sebastián, Grace Choi, Miriam Zeini, Eileen Nicoletti, John E Wagner, Gayatri R. Rao, Adrian J. Thrasher, Jonathan D. Schwartz, Maria Grazia Roncarolo, Juan A. Bueren
Journal: Blood
Blood (2022) 140 (Supplement 1): 10646–10647.
https://doi.org/10.1182/blood-2022-168342
Published: 2022
Journal Articles

Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)

Claire Booth, Julián Sevilla, Gayatri R. Rao, Maria Chitty Lopez, Elena Almarza, Dayna Terrazas, Josune Zubicaray, Marta González Vicent, Kritika Chetty, Grainne O'Toole, Jinhua Xu-Bayford, Eileen Nicoletti, Augustine Fernandes, Caroline Kuo, Satiro de Oliveira, Theodore B. Moore, Grace Choi, Miriam Zeini, Cristina Mesa-Núñez, Adrian J. Thrasher, Juan A. Bueren, Jonathan D. Schwartz, Donald B. Kohn
Journal: Blood
Blood (2022) 140 (Supplement 1): 7774–7775.
https://doi.org/10.1182/blood-2022-158578
Published: 2022
Journal Articles

Hematopoietic and Immunological Assessment in Fanconi Anemia after Ex Vivo Lentiviral FANCA Gene Therapy with RP-L102

Rofida Nofal, Yan Yi Chan, Sushmita Sen, Ulises Juarez Figueroa, Hana Willner, Matthias Felber, Mark Krampf, Supawat Thongthip, Grace Choi, Eileen Nicoletti, Jonathan D. Schwartz, Kenneth I. Weinberg, Alfredo Rodriguez, Rajni Agarwal, Maria Grazia Roncarolo, Agnieszka Czechowicz
Journal: Blood
Blood (2022) 140 (Supplement 1): 7772–7773.
https://doi.org/10.1182/blood-2022-157840
Published: 2022
Journal Articles

Gene Therapy for Fanconi Anemia [Group A]: Interim Results of RP-L102 Clinical Trials

Agnieszka Czechowicz, Julián Sevilla, Rajni Agarwal, Claire Booth, Josune Zubicaray, Paula Río, Susana Navarro, Philip Ancliff, Elena Sebastian, Brian C Beard, Kenneth M Law, Grace Choi, Miriam Zeini, Camilla Duran-Persson, Eileen Nicoletti, John E Wagner, Gayatri R Rao, Adrian J. Thrasher, Jonathan D Schwartz, Juan A Bueren, Maria Grazia Roncarolo
Journal: Blood
Blood (2021) 138 (Supplement 1): 3968.
https://doi.org/10.1182/blood-2021-147071
Published: 2021
Journal Articles

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Interim Results of a Global Phase 1 Study for Adult and Pediatric Patients

Ami J Shah, José Luis López Lorenzo, Susana Navarro, Julián Sevilla, Lucía Llanos, Begoña Pérez de Camino Gaisse, Sol Sanchez, Bert Glader, May Chien, Oscar Quintana Bustamante, Brian C Beard, Kenneth M Law, Miriam Zeini, Grace Choi, Eileen Nicoletti, Gayatri R Rao, Maria Grazia Roncarolo, Juan A Bueren, Jonathan D Schwartz, José C Segovia
Journal: Blood
Blood (2021) 138 (Supplement 1): 563.
https://doi.org/10.1182/blood-2021-148161
Published: 2021
Journal Articles

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results

Donald B. Kohn, Claire Booth, Julián Sevilla, Gayatri R Rao, Elena Almarza, Dayna Terrazas, Eileen Nicoletti, Augustine Fernandes, Caroline Kuo, Satiro de Oliveira, Theodore B. Moore, Kenneth M Law, Brian C Beard, Grace Choi, Miriam Zeini, Cristina Mesa-Núñez, Adrian J. Thrasher, Juan A Bueren, Jonathan D Schwartz
Journal: Blood
Blood (2021) 138 (Supplement 1): 2932.
https://doi.org/10.1182/blood-2021-151898
Published: 2021
Journal Articles

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients

José Luis López Lorenzo, MD, Susana Navarro, PhD, Ami J Shah, MD, Maria Grazia Roncarolo, MD, Julián Sevilla, MD PhD, Lucía Llanos, MD PhD, Begoña Pérez Camino de Gaisse, MD, Sol Sanchez, MD, Bertil Glader, MD PhD, May Chien, MD, Oscar Quintana Bustamante, PhD, Brian C Beard, PhD, Kenneth M Law, PhD, Miriam Zeini, PhD, Grace Choi, BS, Eileen Nicoletti, MD, Juan A Bueren, PhD, Gayatri R Rao, MD JD, Jonathan D Schwartz, MD, José C Segovia, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 47.
https://doi.org/10.1182/blood-2020-137246
Published: 2020
Journal Articles

Gene Therapy for Fanconi Anemia, Complementation Group a: Updated Results from Ongoing Global Clinical Studies of RP-L102

Agnieszka Czechowicz, MDPhD, Rajni Agarwal, MD, Julián Sevilla, MD PhD, Paula Río, PhD, Susana Navarro, PhD, Brian C Beard, PhD, Kenneth M Law, PhD, Grace Choi, BS, Miriam Zeini, PhD, Eileen Nicoletti, MD, John E Wagner, MD, Claire Booth, MBBS, PhD MSc, Jonathan D Schwartz, MD, Juan A Bueren, PhD, Maria Grazia Roncarolo, MD
Journal: Blood
Blood (2020) 136 (Supplement 1): 14.
https://doi.org/10.1182/blood-2020-137106
Published: 2020
Journal Articles

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1

Donald B. Kohn, MD, Gayatri R Rao, MD JD, Elena Almarza, PhD, Dayna Terrazas, RN, Eileen Nicoletti, MD, Augustine Fernandes, PhD, Caroline Kuo, MD, Satiro N. De Oliveira, MD, Theodore B. Moore, MD, Kenneth M Law, PhD, Brian C Beard, PhD, Julian Sevilla, MD PhD, Cristina Mesa-Núñez, PhD, Juan A Bueren, PhD, Jonathan D Schwartz, MD
Journal: Blood
Blood (2020) 136 (Supplement 1): 15.
https://doi.org/10.1182/blood-2020-142484
Published: 2020
Journal Articles

Gene Therapy for Lad-I Immunodeficiency: Preclinical Evaluation of HSC Transduction Under Optimized GMP-Conditions

Elena Almarza, PhD, Cristina Mesa-Núñez, PhD, Carlos Damián, María Fernández-García, Begoña Díez-Cabezas, PhD, M Luz Lozano, Paula Rio, PhD, Rosa M Yañez, PhD, Ken Law, PhD, Brian C Beard, PhD, Jonathan D Schwartz, MD, Juan A. Bueren, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 5751.
https://doi.org/10.1182/blood-2019-122878
Published: 2019
Journal Articles

Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy

Paula Rio, PhD, Susana Navarro, PhD, Rebeca Sanchez-Dominguez, PhD, Jose C Segovia, PhD, Wei Wang, PhD, Eva Merino, Rosa M Yañez, PhD, Jose Antonio Casado, PhD, Yari A. Gimenez, Francisco J Roman-Rodriguez, PhD, Omaira Alberquilla, Eva Galvez, MD, Raquel Hladun, MD, Jordi Barquinero, PhD, Anne Galy, PharmD, PhD, Nagore García de Andoín, MD, Ricardo López, MD PhD, Albert Catalá, MD PhD, Marina Cavazzana, Francois Lefrere, MD, Thierry Leblanc, MD, Jonathan D Schwartz, MD, Roser Pujol, PhD, Jordi Surrallés, PhD, Jean Soulier, MD PhD, Manfred Schmidt, PhD, Cristina Díaz de Heredia, MD PhD, Julian Sevilla, MD PhD, Juan A. Bueren, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4627.
https://doi.org/10.1182/blood-2019-129686
Published: 2019
Journal Articles

Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells

Agnieszka Czechowicz, MD PhD, Maria Grazia Roncarolo, MD, Brian C Beard, PhD, Ken Law, PhD, Eileen Nicoletti, MD, Paula Río, PhD, Juan A. Bueren, PhD, Jonathan D Schwartz, MD, Sandeep Soni, MBBS
Journal: Blood
Blood (2019) 134 (Supplement_1): 3350.
https://doi.org/10.1182/blood-2019-127352
Published: 2019
Journal Articles

Advances in the Gene Therapy of Patients with Fanconi Anemia

Juan A. Bueren, PhD, Susana Navarro, PhD, Wei Wang, PhD, Rebeca Sanchez-Dominguez, PhD, Eva Merino, Jose C Segovia, PhD, Anne Galy, PharmD, PhD, María L. Lamana, MD PhD, Rosa M Yañez, PhD, Jose A Casado, PhD, Yari A. Gimenez, Francisco J Roman-Rodriguez, Lara A. Alvarez, Roser Pujol, PhD, Jordi Surralles, PhD, Ricardo López, MD PhD, Nagore García de Andoín, MD, Albert Catala, MD PhD, Eva Galvez, MD, Raquel Hladun, MD, Jonathan D Schwartz, MD, Manfred Schmidt, PhD, Cristina Díaz de Heredia, MD PhD, Paula Rio, PhD, Julian Sevilla, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 1022.
https://doi.org/10.1182/blood-2018-99-111160
Published: 2018
Journal Articles

Preclinical Evaluation for the Gene Therapy of Patients with Leukocyte Adhesion Deficiency Type I

Elena Almarza, PhD, Cristina Mesa-Núñez, Carlos Damián, María Fernández-García, Begoña Díez-Cabezas, Jonathan D Schwartz, MD, Juan A Bueren
Journal: Blood
Blood (2018) 132 (Supplement 1): 5798.
https://doi.org/10.1182/blood-2018-99-111136
Published: 2018
Journal Articles

Leukocyte Adhesion Deficiency-I (LAD-I): A Comprehensive Review of Published Cases

Sanchali Kasbekar, PharmD, Juan Bueren, PhD, Adrian J Thrasher, MD PhD FMedSci, Donald B Kohn, MD, Julian Sevilla, MD PhD, Jonathan D Schwartz, MD, Elena Almarza, PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 4821.
https://doi.org/10.1182/blood.V130.Suppl_1.4821.4821
Published: 2017
Journal Articles

Lead-in Stage Results of a Pivotal Trial of SL-401, an Interleukin-3 Receptor (IL-3R) Targeting Biologic, in Patients with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) or Acute Myeloid Leukemia (AML)

Kendra L. Sweet, MD, Naveen Pemmaraju, MD, Andrew A Lane, MD PhD, Anthony S. Stein, MD, Sumithira Vasu, MBBS, William Blum, MD, David A. Rizzieri, MD, Eunice S. Wang, MD, Eric K. Rowinsky, MD, Michael Szarek, PhD, Christopher L. Brooks, PhD, Sarah Disalvatore, MPH, David Liu, Madeleine Duvic, MD, Jonathan D. Schwartz, MD, Marina Konopleva, MD PhD
Journal: Blood
Blood (2015) 126 (23): 3795.
https://doi.org/10.1182/blood.V126.23.3795.3795
Published: 2015