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Gene therapy for hemophilia B: results from the phase 1/2 101HEMB01/02 studies

Open Access
Clinical Trials & Observations
Steven Pipe, Allen Poma, Anita Rajasekhar, Tamara Everington, Serap Sankoh, Jack Allen, Jason Cataldo, Eric Crombez
Journal: Blood Advances
Blood Adv (2025) 9 (12): 2980–2987.
https://doi.org/10.1182/bloodadvances.2024015184
Published: 2025