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Bettina M. Cockroft
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Journal Articles

Giroctocogene fitelparvovec gene therapy for severe hemophilia A: 104-week analysis of the phase 1/2 Alta study

Clinical Trials & Observations
Andrew D. Leavitt, Barbara A. Konkle, Kimo C. Stine, Nathan Visweshwar, Thomas J. Harrington, Adam Giermasz, Steven Arkin, Annie Fang, Frank Plonski, Anne Yver, Florence Ganne, Delphine Agathon, Maria de los Angeles Resa, Li-Jung Tseng, Gregory Di Russo, Bettina M. Cockroft, Liching Cao, Jeremy Rupon
Journal: Blood
Blood (2024) 143 (9): 796–806.
https://doi.org/10.1182/blood.2022018971
Published: 2024
Includes: Supplemental data
Journal Articles

Four-Year Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia Α

Thomas J. Harrington, Adam Giermasz, Nathan Visweshwar, Andrew D. Leavitt, Barbara A. Konkle, Jeremy Rupon, Gregory Dirusso, Li-Jung Tseng, Maria de los Angeles Resa, Florence Ganne, Delphine Agathon, Frank Plonski, Didier Rouy, Bettina M. Cockroft, Annie F. Fang, Steven Arkin
Journal: Blood
Blood (2023) 142 (Supplement 1): 1054.
https://doi.org/10.1182/blood-2023-179422
Published: 2023
Journal Articles

Interim Safety and Efficacy Results from a Phase 1/2 Study of Zinc Finger Nuclease-Modified Autologous Hematopoietic Stem Cells for Sickle Cell Disease (PRECIZN-1)

Asif Alavi, Mehrdad Abedi, Suhag Parikh, Richard Boismenu, Michael Chen, Ben L. Hsu, Bettina M. Cockroft, Isobelle Galeon, Pablo Rendo, Mark C. Walters
Journal: Blood
Blood (2022) 140 (Supplement 1): 4907–4909.
https://doi.org/10.1182/blood-2022-163725
Published: 2022
Journal Articles

Updated Results of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia Α

Adam Giermasz, Nathan Visweshwar, Thomas J. Harrington, Andrew D Leavitt, Barbara Konkle, Jeremy Rupon, Gregory Di Russo, Li-Jung Tseng, Maria de los Angeles Resa, Florence Ganne, Delphine Agathon, Frank Plonski, Didier Rouy, Bettina M. Cockroft, Annie Fang, Steven Arkin
Journal: Blood
Blood (2022) 140 (Supplement 1): 7776–7777.
https://doi.org/10.1182/blood-2022-159421
Published: 2022
Journal Articles

Updated Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia

Mark C. Walters, Angela R. Smith, Gary J. Schiller, Erica B. Esrick, David A. Williams, Tatiana Gogoleva, Didier Rouy, Bettina M. Cockroft, Gregory M. Vercellotti
Journal: Blood
Blood (2021) 138 (Supplement 1): 3974.
https://doi.org/10.1182/blood-2021-147907
Published: 2021
Journal Articles

Updated Results of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (PF-07055480/SB-525) Gene Therapy in Adults with Severe Hemophilia a

Nathan Visweshwar, Thomas J. Harrington, Andrew D Leavitt, Barbara A. Konkle, Adam Giermasz, Kimo Stine, Jeremy Rupon, Gregory Di Russo, Li-Jung Tseng, Maria de los Angeles Resa, Florence Ganne, Delphine Agathon, Frank Plonski, Didier Rouy, Bettina M. Cockroft, Annie Fang, Steven Arkin
Journal: Blood
Blood (2021) 138 (Supplement 1): 564.
https://doi.org/10.1182/blood-2021-148651
Published: 2021
Journal Articles

Updated Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (SB-525) Gene Therapy in Adults with Severe Hemophilia a

Andrew D. Leavitt, MD, Barbara A. Konkle, MD, Kimo Stine, MD, Nathan Visweshwar, MD, Thomas J. Harrington, MD, Adam Giermasz, MD PhD, Steven Arkin, MD, Annie Fang, MD PhD, Frank Plonski, RN, MA, Lynne Smith, MBA, Li-Jung Tseng, PhD MBA, Gregory Di Russo, MD, Bettina M Cockroft, MD, Jeremy Rupon, MD, Didier Rouy, MD PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 12.
https://doi.org/10.1182/blood-2020-137648
Published: 2020