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公开(公告)号:EP4053277A1
公开(公告)日:2022-09-07
申请号:EP21213766.5
申请日:2016-12-26
发明人: BOITANO, Anthony Edward , COOKE, Michael , KLICKSTEIN, Lloyd B. , LESCARBEAU, Reynald , MICKANIN, Craig Stephen , MULUMBA, Kabungo , POLICE, Seshidhar Reddy , SNEAD, Jennifer , STEVENSON, Susan C. , STEWART, Morag , YANG, Yi
IPC分类号: C12N15/113
摘要: The present invention is directed to genome editing systems, reagents and methods for the treatment of hemoglobinopathies.
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公开(公告)号:EP2788476B1
公开(公告)日:2019-05-22
申请号:EP12806795.6
申请日:2012-12-07
IPC分类号: C12N5/0789 , A61K35/12
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公开(公告)号:EP2350078B1
公开(公告)日:2019-04-17
申请号:EP09748626.0
申请日:2009-10-29
发明人: BOITANO, Anthony , COOKE, Michael , PAN, Shifeng , SCHULTZ, Peter G. , TELLEW, John , WAN, Yongqin , WANG, Xing
IPC分类号: C07D473/34 , C07D487/04 , A61K31/52 , A61K31/519 , A61P37/00
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4.
公开(公告)号:EP3624806A1
公开(公告)日:2020-03-25
申请号:EP18730857.2
申请日:2018-05-17
申请人: Novartis AG
IPC分类号: A61K31/675 , A61P35/00 , A61K39/00 , A61K9/19 , A61K45/06
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公开(公告)号:EP3524604A1
公开(公告)日:2019-08-14
申请号:EP19164770.0
申请日:2009-10-29
发明人: BOITANO, Anthony , COOKE, Michael , PAN, Shifeng , SCHULTZ, Peter G. , TELLEW, John , WAN, Yongqin , WANG, Xing
IPC分类号: C07D473/34 , C07D487/04 , A61K35/28 , C12N5/0789 , A61P7/00 , A61P43/00 , A61P35/00 , A61K31/52 , A61K31/519 , A61P37/00
摘要: The present invention relates to compounds and compositions for expanding the number of CD34+ cells for transplantation. The invention further relates to a cell population comprising expanded hematopoietic stem cells (HSCs) and its use in autologous or allogeneic transplantation for the treatment of patients with inherited immunodeficient and autoimmune diseases and diverse hematopoietic disorders to reconstitute the hematopoietic cell lineages and immune system defense.
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