公开(公告)号：US12168776B2

公开(公告)日：2024-12-17

申请号：US16636583

申请日：2018-08-09

Applicant: BIOVERATIV THERAPEUTICS INC.

Inventor： Alexey Seregin ,  Tongyao Liu ,  Susannah Patarroyo-White ,  Douglas Drager ,  Robert T. Peters ,  Jiayun Liu

IPC: C12N15/00 ,  C07K14/755 ,  C12N15/86

Abstract: The present disclosure provides nucleic acid molecules comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette encoding a miRNA and/or a therapeutic protein. In certain embodiments, the therapeutic protein comprises a clotting factor, e.g., a FVIII polypeptide, a FIX polypeptide, or a fragment thereof. In some embodiments, the first ITR and/or the second ITR is an ITR of a non-adeno-associated virus (AAV). The present disclosure also provides methods of treating bleeding disorders such as hemophilia comprising administering to the subject the nucleic acid molecule or a polypeptide encoded thereby.