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2. 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

Author

Giermasz, Adam, primary, Visweshwar, Nathan, additional, Harrington, Thomas J., additional, Leavitt, Andrew D, additional, Konkle, Barbara, additional, Rupon, Jeremy, additional, Di Russo, Gregory, additional, Tseng, Li-Jung, additional, de los Angeles Resa, Maria, additional, Ganne, Florence, additional, Agathon, Delphine, additional, Plonski, Frank, additional, Rouy, Didier, additional, Cockroft, Bettina M., additional, Fang, Annie, additional, and Arkin, Steven, additional

Published
2022
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3. 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

Author

Visweshwar, Nathan, primary, Harrington, Thomas J., additional, Leavitt, Andrew D, additional, Konkle, Barbara A., additional, Giermasz, Adam, additional, Stine, Kimo, additional, Rupon, Jeremy, additional, Di Russo, Gregory, additional, Tseng, Li-Jung, additional, de los Angeles Resa, Maria, additional, Ganne, Florence, additional, Agathon, Delphine, additional, Plonski, Frank, additional, Rouy, Didier, additional, Cockroft, Bettina M., additional, Fang, Annie, additional, and Arkin, Steven, additional

Published
2021
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4. 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

Author

Harrington, Thomas J., Giermasz, Adam, Visweshwar, Nathan, Leavitt, Andrew D., Konkle, Barbara A., Rupon, Jeremy, Dirusso, Gregory, Tseng, Li-Jung, de los Angeles Resa, Maria, Ganne, Florence, Agathon, Delphine, Plonski, Frank, Rouy, Didier, Cockroft, Bettina M., Fang, Annie F., and Arkin, Steven

Abstract

Introduction:Hemophilia A is an X-linked ( F8gene) recessive disorder of hemostasis that results in insufficient factor VIII (FVIII) activity. Adeno-associated virus (AAV)-based gene therapy enables delivery of a modified F8cDNA, allowing synthesis of functional endogenous FVIII, which prevents bleeding events. We present updated results with nearly 4 years of follow-up on an ongoing gene therapy study in participants with severe hemophilia A (FVIII activity <1%).

Published
2023
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