公开(公告)号：US11952585B2

公开(公告)日：2024-04-09

申请号：US17147974

申请日：2021-01-13

申请人： HOMOLOGY MEDICINES, INC.

发明人： Omar L. Francone ,  Deborah A. Kinch ,  Diana Lamppu ,  Albert Barnes Seymour ,  Teresa Leah Wright

IPC分类号： C12N15/864 ,  A61K48/00 ,  C12N7/00 ,  C12N7/01 ,  C12N9/02 ,  C12N15/52 ,  C12N15/86 ,  A61K31/573

CPC分类号： C12N15/86 ,  C12N7/00 ,  C12N9/0071 ,  A61K31/573 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/50 ,  C12Y114/16001

摘要： Provided herein are methods of treating a subject having a disease or disorder associated with a PAH gene mutation. The methods generally comprise administering to the subject a therapeutically effective does of a recombinant adeno-associated virus (rAAV) that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell and thereby restore PAH gene function in the subject.

公开(公告)号：US20210261983A1

公开(公告)日：2021-08-26

申请号：US17147974

申请日：2021-01-13

申请人： HOMOLOGY MEDICINES, INC.

发明人： Omar L. Francone ,  Deborah A. Kinch ,  Diana Lamppu ,  Albert Barnes Seymour ,  Teresa Leah Wright

IPC分类号： C12N15/86 ,  C12N7/00 ,  C12N9/02

摘要： Provided herein are methods of treating a subject having a disease or disorder associated with a PAH gene mutation. The methods generally comprise administering to the subject a therapeutically effective does of a recombinant adeno-associated virus (rAAV) that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell and thereby restore PAH gene function in the subject.