公开(公告)号：EP3363903A1

公开(公告)日：2018-08-22

申请号：EP18159400.3

申请日：2014-11-07

申请人： Editas Medicine, Inc. ,  The Broad Institute Inc. ,  Massachusetts Institute of Technology ,  The University of Iowa Research Foundation

发明人： PALESTRANT, Deborah ,  MATA-FINK, Jordi ,  BORISY, Alexis ,  ZHANG, Feng ,  DAVIDSON, Beverly ,  RODRIGUEZ, Edgardo

IPC分类号： C12N15/63

CPC分类号： C12N15/902 ,  C12N9/22 ,  C12N15/11 ,  C12N15/111 ,  C12N15/1137 ,  C12N15/63 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2800/80

摘要： Disclosed herein are methods and compositions useful in targeting a payload to, or editing a target nucleic acid, where a governing gRNA molecule is used to target, optionally inactivate, a Cas9 molecule or a Cas9 molecule/gRNA complex.

公开(公告)号：EP4372090A2

公开(公告)日：2024-05-22

申请号：EP23219520.6

申请日：2014-11-07

申请人： Editas Medicine, Inc. ,  The Broad Institute, Inc. ,  Massachusetts Institute of Technology ,  The University of Iowa Research Foundation

发明人： PALESTRANT, Deborah ,  MATA-FINK, Jordi ,  BORISY, Alexis ,  ZHANG, Feng ,  DAVIDSON, Beverly ,  RODRIGUEZ, Edgardo

IPC分类号： C12N15/63

CPC分类号： C12N15/63 ,  C12N15/111 ,  C12N15/1137 ,  C12N2310/1020130101 ,  C12N2310/2020170501 ,  C12N9/22

摘要： Disclosed herein are methods and compositions useful in targeting a payload to, or editing a target nucleic acid, where a governing gRNA molecule is used to target, optionally inactivate, a Cas9 molecule or a Cas9 molecule/gRNA complex.

公开(公告)号：EP3080257A1

公开(公告)日：2016-10-19

申请号：EP14821429.9

申请日：2014-12-12

申请人： The Broad Institute Inc. ,  Massachusetts Institute of Technology ,  University of Iowa Research Foundation

发明人： ZHANG, Feng ,  DAVIDSON, Beverly ,  LIN, Chie-yu ,  RODRIGUEZ, Edgardo

IPC分类号： C12N9/22 ,  C12N15/10 ,  C12N15/85

CPC分类号： C12N9/22 ,  A01K67/0276 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/0318 ,  A61K31/713 ,  A61K38/465 ,  A61K48/005 ,  B82Y5/00 ,  C12N7/00 ,  C12N15/102 ,  C12N15/1082 ,  C12N15/113 ,  C12N15/63 ,  C12N15/86 ,  C12N15/907 ,  C12N2740/15043 ,  C12N2750/14143 ,  C12Y301/00

摘要： The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences especially for use as to nucleotide repeat disorders. Provided are delivery systems and tissues or organ which are targeted as sites for delivery especially for use as to nucleotide repeat disorders. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex or system especially for use as to nucleotide repeat disorders, as well as methods for the design and of such. Also provided are methods of directing CRISPR complex or system formation in eukaryotic cells especially for use as to nucleotide repeat disorders including with consideration of specificity for target recognition and avoidance of toxicity and editing or modifying a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.

摘要翻译： 本发明提供了系统，方法，和组合物的序列和/或靶序列的活动的操作输送，工程和优化爱尤其用作核苷酸重复障碍。 提供了哪些有针对性的网站进行传递真爱特别是用作核苷酸重复障碍输送系统和组织或器官。 因此，提供了用于设计和追求的载体和载体系统其中的一些编码复杂或CRISPR系统的爱情一个或多个组件特别是用作核苷酸重复障碍，以及方法。 所以提供了在真核细胞中指导CRISPR复合物或系统形成方法特别爱用作到核苷酸重复障碍考虑特异性目标识别和毒性和编辑的避免或在感兴趣的基因组基因座修饰的靶位点，以改变或包括 提高疾病或症状的状态。

公开(公告)号：EP3080258A1

公开(公告)日：2016-10-19

申请号：EP14821430.7

申请日：2014-12-12

申请人： The Broad Institute, Inc. ,  Massachusetts Institute of Technology ,  University of Iowa Research Foundation

发明人： DAVIDSON, Beverly ,  LIN, Chie-yu ,  RODRIGUEZ, Edgardo ,  ZHANG, Feng

IPC分类号： C12N9/22 ,  C12N15/10 ,  C12N15/85

CPC分类号： C12N9/22 ,  A01K67/0276 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/0318 ,  A61K31/713 ,  A61K38/465 ,  A61K48/005 ,  B82Y5/00 ,  C12N7/00 ,  C12N15/102 ,  C12N15/1082 ,  C12N15/113 ,  C12N15/63 ,  C12N15/86 ,  C12N15/907 ,  C12N2740/15043 ,  C12N2750/14143 ,  C12Y301/00

摘要： The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences especially for use as to nucleotide repeat disorders. Provided are delivery systems and tissues or organ which are targeted as sites for delivery especially for use as to nucleotide repeat disorders. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex or system especially for use as to nucleotide repeat disorders, as well as methods for the design and of such. Also provided are methods of directing CRISPR complex or system formation in eukaryotic cells especially for use as to nucleotide repeat disorders including with consideration of specificity for target recognition and avoidance of toxicity and editing or modifying a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.

公开(公告)号：EP3066201A1

公开(公告)日：2016-09-14

申请号：EP14802772.5

申请日：2014-11-07

申请人： Editas Medicine, Inc. ,  The Broad Institute Inc. ,  Massachusetts Institute of Technology ,  The University of Iowa Research Foundation

发明人： GLUCKSMANN, Alexandra ,  PALESTRANT, Deborah ,  TARTAGLIA, Louis, Anthony ,  MATA-FINK, Jordi ,  CZECHOWICZ, Agnieszka, Dorota ,  BORISY, Alexis ,  ZHANG, Feng ,  DAVIDSON, Beverly ,  RODRIGUEZ, Edgardo

IPC分类号： C12N15/63

CPC分类号： C12N15/902 ,  C12N9/22 ,  C12N15/11 ,  C12N15/111 ,  C12N15/1137 ,  C12N15/63 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2800/80

摘要： Disclosed herein are methods and compositions useful in targeting a payload to, or editing a target nucleic acid, where a governing gRNA molecule is used to target, optionally inactivate, a Cas9 molecule or a Cas9 molecule/gRNA complex.

摘要翻译： 本文公开了可用于将有效载荷靶向或编辑靶核酸的方法和组合物，其中使用治疗性gRNA分子靶向，任选地灭活Cas9分子或Cas9分子/ gRNA复合物。

公开(公告)号：EP3066201B1

公开(公告)日：2018-03-07

申请号：EP14802772.5

申请日：2014-11-07

申请人： Editas Medicine, Inc. ,  The Broad Institute Inc. ,  Massachusetts Institute of Technology ,  The University of Iowa Research Foundation

发明人： PALESTRANT, Deborah ,  MATA-FINK, Jordi ,  BORISY, Alexis ,  ZHANG, Feng ,  DAVIDSON, Beverly ,  RODRIGUEZ, Edgardo

IPC分类号： C12N15/63

CPC分类号： C12N15/902 ,  C12N9/22 ,  C12N15/11 ,  C12N15/111 ,  C12N15/1137 ,  C12N15/63 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2800/80

摘要： Disclosed herein are methods and compositions useful in targeting a payload to, or editing a target nucleic acid, where a governing gRNA molecule is used to target, optionally inactivate, a Cas9 molecule or a Cas9 molecule/gRNA complex.

公开(公告)号：EP4372090A3

公开(公告)日：2024-08-07

申请号：EP23219520.6

申请日：2014-11-07

申请人： Editas Medicine, Inc. ,  The Broad Institute, Inc. ,  Massachusetts Institute of Technology ,  The University of Iowa Research Foundation

发明人： PALESTRANT, Deborah ,  MATA-FINK, Jordi ,  BORISY, Alexis ,  ZHANG, Feng ,  DAVIDSON, Beverly ,  RODRIGUEZ, Edgardo

IPC分类号： C12N15/63 ,  C12N15/11 ,  C12N9/22

CPC分类号： C12N15/63 ,  C12N15/111 ,  C12N15/1137 ,  C12N2310/1020130101 ,  C12N2310/2020170501 ,  C12N9/22

摘要： Disclosed herein are methods and compositions useful in targeting a payload to, or editing a target nucleic acid, where a governing gRNA molecule is used to target, optionally inactivate, a Cas9 molecule or a Cas9 molecule/gRNA complex.