A Precision Gene Engineered B Cell Medicine Producing Sustained Levels of Active Factor IX for Hemophilia B Therapy
Hanlan Liu, Swati Singh, Timothy Mullen, Caroline P. Bullock, Sean Keegan, Troy Patterson, Sakshisingh Thakur, Amy Lundberg, Sol Shenker, Ryan Couto, Charuta Yadav, Shamael R. Dastagir, Lily Yuanzhi Li, Wayne Bainter, Ella Liberzon, , Richard A. Morgan
This paper describes a gene-engineered B Cell Medicine (BE-101) that produces sustained active factor IX for hemophilia B therapy, showing durable and redosable effects in mice.
Molecular Therapy · Sep 5, 2025Peer-reviewedPharmacology & Toxicology