公开(公告)号：EP3763814A1

公开(公告)日：2021-01-13

申请号：EP20185077.3

申请日：2016-05-06

申请人： The Children's Medical Center Corporation ,  The Broad Institute, Inc. ,  Massachusetts Institute of Technology

发明人： BAUER, Daniel E. ,  ORKIN, Stuart H. ,  SANJANA, Neville ,  SHALEM, Ophir ,  ZHANG, Feng

IPC分类号： C12N15/10 ,  C12N5/078 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

摘要： Provided herein are nucleic acid molecules that target the BCL11A enhancer functional regions, compositions comprising the nucleic acid molecules and methods for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels. In particular, the nucleic acid molecules target the +62, +58, and/or the +55 enhancer functional regions.

公开(公告)号：EP3208339A1

公开(公告)日：2017-08-23

申请号：EP16194659.5

申请日：2009-09-14

申请人： The Children's Medical Center Corporation ,  President and Fellows of Harvard College

发明人： ORKIN, Stuart H. ,  SANKARAN, Vijay G.

IPC分类号： C12N15/63 ,  C12N15/11 ,  A61K48/00

CPC分类号： C12N15/113 ,  A01K67/0275 ,  A01K2217/052 ,  A01K2217/15 ,  A01K2227/105 ,  A01K2267/0381 ,  A61K31/713 ,  A61K35/28 ,  A61K49/00 ,  C07K14/4705 ,  C07K14/805 ,  C12N5/0647 ,  C12N15/8509 ,  C12N15/86 ,  C12N2310/14 ,  C12N2310/531 ,  C12N2740/15043 ,  C12N2800/206

摘要： The invention relates to methods and uses of modulating fetal hemoglobin expression (HbF) in a hematopoietic progenitor cells via inhibitors of BCL11A expression or activity, such as RNAi and antibodies.

摘要翻译： 本发明涉及通过BCL11A表达或活性抑制剂如RNAi和抗体调节造血祖细胞中胎儿血红蛋白表达（HbF）的方法和用途。

公开(公告)号：EP3208339B1

公开(公告)日：2019-05-01

申请号：EP16194659.5

申请日：2009-09-14

申请人： The Children's Medical Center Corporation ,  President and Fellows of Harvard College

发明人： ORKIN, Stuart H. ,  SANKARAN, Vijay G.

IPC分类号： C12N15/63 ,  C12N15/11 ,  A61K48/00

公开(公告)号：EP2925864B1

公开(公告)日：2018-10-31

申请号：EP13858006.3

申请日：2013-11-27

申请人： The Children's Medical Center Corporation

发明人： ORKIN, Stuart H. ,  BAUER, Daniel E. ,  XU, Jian

IPC分类号： C12N5/10 ,  C12N5/0789 ,  C12N15/09 ,  A61K35/12 ,  A61K38/46 ,  C07K14/805 ,  C12N15/10 ,  C12N15/63 ,  C07K14/47 ,  C12N15/01 ,  C12N9/22

CPC分类号： C12N15/01 ,  A61K35/12 ,  A61K38/465 ,  C07K14/4702 ,  C07K14/805 ,  C12N9/22 ,  C12N15/102 ,  C12N15/63

摘要： Provided herein are methods and compositions for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels.

公开(公告)号：EP3294873B1

公开(公告)日：2020-08-19

申请号：EP16793259.9

申请日：2016-05-06

申请人： The Children's Medical Center Corporation ,  The Broad Institute, Inc. ,  Massachusetts Institute of Technology

发明人： BAUER, Daniel E. ,  ORKIN, Stuart H. ,  SANJANA, Neville ,  SHALEM, Ophir ,  ZHANG, Feng

IPC分类号： C12N15/10 ,  C12N5/078 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

公开(公告)号：EP3502240A1

公开(公告)日：2019-06-26

申请号：EP18203436.3

申请日：2013-11-27

申请人： The Children's Medical Center Corporation

发明人： ORKIN, Stuart H. ,  BAUER, Daniel E. ,  XU, Jian

IPC分类号： C12N5/10 ,  C12N5/0789 ,  C12N15/09 ,  A61K35/12 ,  A61K38/46 ,  C07K14/805 ,  C12N15/10 ,  C12N15/63 ,  C07K14/47 ,  C12N15/01 ,  C12N9/22

摘要： Provided herein are methods and compositions for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels.