公开(公告)号：US20230241139A1

公开(公告)日：2023-08-03

申请号：US18096853

申请日：2023-01-13

Applicant: TAKEDA PHARMACEUTICAL COMPANY LIMITED

Inventor： Christian FIEDLER ,  Eva FRITSCHER ,  Meinhard HASSLACHER ,  Dominik MITTERGRADNEGGER ,  Tanvir TABISH

IPC: A61K35/76 ,  A61P7/04 ,  A61K9/00 ,  A61K9/19 ,  A61K47/18 ,  A61K47/26 ,  A61P7/00 ,  A61K31/195

CPC classification number: A61K35/76 ,  A61P7/04 ,  A61K9/0019 ,  A61K9/0053 ,  A61K9/19 ,  A61K47/183 ,  A61K47/26 ,  A61P7/00 ,  A61K31/195

Abstract: Adeno-associated liquid and lyophilized pharmaceutical compositions are provided herein. In exemplary aspects, the pharmaceutical compositions comprise about 5 mM to about 25 mM L-histidine, about 0 mM to about 150 mM sodium chloride, about 0.001% (w/v) to about 0.01% (w/v) polysorbate 80 (PS80), and about 1% to about 10% (w/v) sucrose, trehalose, or combination thereof to AAV. In exemplary aspects, the pharmaceutical compositions further comprise glycine or mannitol. Methods of preparing a pharmaceutical composition comprising AAV, methods of treating a bleeding disorder in a subject, and methods of storing AAV compositions are also provided.