公开(公告)号：WO2010103475A2

公开(公告)日：2010-09-16

申请号：PCT/IB2010/051038

申请日：2010-03-10

Applicant: MED DISCOVERY SA ,  UNITECTRA ,  FONTANA, Adriano ,  RECHER, Mike ,  KUNDIG, Christoph

Inventor： FONTANA, Adriano ,  RECHER, Mike ,  KUNDIG, Christoph

IPC: A61K35/14 ,  A61K38/55 ,  C12N5/0787 ,  C12Q1/37 ,  A61P7/00

CPC classification number: A61K38/57 ,  A61K35/15 ,  A61K35/28 ,  A61K38/1709 ,  A61K2300/00

Abstract: The invention relates to therapeutic compounds which are inhibitors of serine proteases, to pharmaceutical compositions thereof and to their use in the treatment of the human or animal body. More specifically, the present invention relates to a method for the treatment of neutropenia comprising the administration to a subject in need thereof of a therapeutically effective amount of a serine protease inhibitor. The invention also comprises prevention of apoptosis of myeloid cells (1) during and after transfection of bone marrow cells performed for gene therapy, (2) during blood stem cell mobilization performed for reconstitution of hematopoiesis and (3) during infusion of cells of the myeloid lineage for reconstitution of hematopoiesis for gene therapy or for treatment of neutropenia by infusion of neutrophils.

Abstract translation: 本发明涉及作为丝氨酸蛋白酶抑制剂的治疗性化合物，其药物组合物以及它们在治疗人体或动物体中的用途。 更具体地说，本发明涉及用于治疗嗜中性白血球减少症的方法，其包括向有需要的受试者施用治疗有效量的丝氨酸蛋白酶抑制剂。 本发明还包括在用于基因治疗的骨髓细胞转染期间和之后预防骨髓细胞凋亡（1），（2）在用于重建造血作用的血液干细胞动员期间和（3）在输注髓细胞期间 用于重建基因疗法的造血作用或通过输注嗜中性粒细胞治疗嗜中性白细胞减少症的谱系。

公开(公告)号：WO2010103475A3

公开(公告)日：2010-11-25

申请号：PCT/IB2010051038

申请日：2010-03-10

Applicant: MED DISCOVERY SA ,  UNIV ZUERICH ,  FONTANA ADRIANO ,  RECHER MIKE ,  KUNDIG CHRISTOPH

Inventor： FONTANA ADRIANO ,  RECHER MIKE ,  KUNDIG CHRISTOPH

IPC: A61K35/14 ,  A61K38/55 ,  A61P7/00 ,  C12N5/0787 ,  C12Q1/37

CPC classification number: A61K38/57 ,  A61K35/15 ,  A61K35/28 ,  A61K38/1709 ,  A61K2300/00

Abstract: The invention relates to therapeutic compounds which are inhibitors of serine proteases, to pharmaceutical compositions thereof and to their use in the treatment of the human or animal body. More specifically, the present invention relates to a method for the treatment of neutropenia comprising the administration to a subject in need thereof of a therapeutically effective amount of a serine protease inhibitor. The invention also comprises prevention of apoptosis of myeloid cells (1) during and after transfection of bone marrow cells performed for gene therapy, (2) during blood stem cell mobilization performed for reconstitution of hematopoiesis and (3) during infusion of cells of the myeloid lineage for reconstitution of hematopoiesis for gene therapy or for treatment of neutropenia by infusion of neutrophils.

Abstract translation: 本发明涉及作为丝氨酸蛋白酶抑制剂的治疗化合物，其药物组合物及其在治疗人或动物体中的用途。 更具体地说，本发明涉及一种治疗嗜中性粒细胞减少症的方法，其包括向有需要的受试者施用治疗有效量的丝氨酸蛋白酶抑制剂。 本发明还包括在用于基因治疗的骨髓细胞转染期间和之后预防骨髓细胞（1）的凋亡，（2）在用于重建造血细胞的血液干细胞移动过程中和（3）在髓样细胞输注期间 重建血液造影用于基因治疗或通过输注嗜中性粒细胞治疗中性粒细胞减少症的血统。