公开(公告)号：US20250002868A1

公开(公告)日：2025-01-02

申请号：US18294941

申请日：2022-08-03

Applicant: TAKEDA PHARMACEUTICAL COMPANY LIMITED

Inventor： Christian Fiedler ,  Dominik MITERGRADNEGGER ,  Meinhard HASSLACHER ,  Thomas GATTERNIG ,  Daniela VASINA ,  Michael FELDHOFER ,  Renate PFANDL

IPC: C12N7/00

Abstract: The present disclosure provides for a method of purifying adeno-associated virus comprising purifying full AAV capsids from a concentrated AAV fraction or preparation comprising empty AAV capsids and full AAV capsids. The present disclosure also provides for a method of purifying adeno-associated virus comprising purifying empty AAV capsids from a concentrated AAV fraction or preparation comprising empty AAV capsids and full AAV capsids. The method utilizes one or more monovalent and one or more divalent cations to effect the separation resulting in purified full AAV capsids or empty AAV capsids.

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.

公开(公告)号：US20220267796A1

公开(公告)日：2022-08-25

申请号：US17622044

申请日：2020-06-26

Applicant: TAKEDA PHARMACEUTICAL COMPANY LIMITED

Inventor： Christian FIEDLER ,  Marcus SCHEINDEL ,  Meinhard HASSLACHER ,  Jadranka KOEHN

IPC: C12N15/86 ,  B01D15/16 ,  B01D15/38

Abstract: Provided herein are methods of producing an adeno-associated virus (AAV) product and methods of purifying adeno-associated virus. AAV is loaded onto an affinity resin, wash steps are undertaken at room temperature, and AAV is eluted from the affinity resin at a lower temperature. Various buffers are disclosed for use in the wash steps and elution.