公开(公告)号：EP3724335A1

公开(公告)日：2020-10-21

申请号：EP18830581.7

申请日：2018-12-10

申请人： Rosalind Franklin University of Medicine and Science ,  The McLean Hospital Corporation

发明人： HASTINGS, Michelle, L. ,  ISACSON, Ole ,  KORECKA-ROET, Joanna, A.

IPC分类号： C12N15/113

公开(公告)号：EP3377629A1

公开(公告)日：2018-09-26

申请号：EP16806342.8

申请日：2016-11-11

申请人： Rosalind Franklin University of Medicine and Science ,  The McLean Hospital Corporation

发明人： HASTINGS, Michelle, L. ,  ISACSON, Ole ,  KORECKA-ROET, Joanna, A.

IPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/7125 ,  C07H21/00 ,  A61P25/28

CPC分类号： C12N15/1137 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/346 ,  C12N2320/33 ,  C12Y207/11001 ,  C12N2310/321 ,  C12N2310/3521 ,  C12N2310/3525 ,  C12N2310/322 ,  C12N2310/3533

摘要： The present disclosure relates generally to compounds comprising oligonucleotides complementary to a Leucine-Rich-Repeat-Kinase (LRRK2) RNA transcript. Certain such compounds are useful for hybridizing to a LRRK2 RNA transcript, including but not limited to a LRRK2 RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the LRRK2 transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Parkinson's disease.

公开(公告)号：EP3077510B1

公开(公告)日：2020-05-06

申请号：EP14867872.5

申请日：2014-12-02

申请人： Ionis Pharmaceuticals, Inc. ,  Rosalind Franklin University of Medicine and Science

发明人： RIGO, Frank ,  HASTINGS, Michelle, L.

IPC分类号： C12N15/113 ,  A61K31/7088

公开(公告)号：EP3259356A1

公开(公告)日：2017-12-27

申请号：EP16714069.8

申请日：2016-02-17

申请人： Rosalind Franklin University of Medicine and Science ,  Hastings, Michelle L.

发明人： HASTINGS, Michelle, L.

IPC分类号： C12N15/113

CPC分类号： C12N15/1137 ,  C12N15/1138 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/351 ,  C12N2310/3521 ,  C12N2310/3525 ,  C12N2310/3533 ,  C12N2320/30 ,  C12N2320/33

摘要： The present disclosure relates generally to compounds comprising oligonucleotides complementary to a cystic fibrosis transmembrane conductance regulator (CFTR) RNA transcript. Certain such compounds are useful for hybridizing to a CFTR RNA transcript, including but not limited to a CFTR RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the CFTR transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Cystic Fibrosis.

公开(公告)号：EP3849564A1

公开(公告)日：2021-07-21

申请号：EP19859762.7

申请日：2019-09-10

申请人： Ionis Pharmaceuticals, Inc. ,  Rosalind Franklin University of Medicine and Science

发明人： HASTINGS, Michelle, L. ,  RIGO, Frank

IPC分类号： A61K31/712 ,  A61K31/7125 ,  A61K31/713 ,  C12N15/113

公开(公告)号：EP3077510A2

公开(公告)日：2016-10-12

申请号：EP14867872.5

申请日：2014-12-02

申请人： Ionis Pharmaceuticals, Inc. ,  Rosalind Franklin University of Medicine and Science

发明人： RIGO, Frank ,  HASTINGS, Michelle, L.

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  A61K31/7088 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/3341 ,  C12N2310/3525

摘要： The present invention provides compounds comprising oligonucleotides complementary to a CLN3 transcript. Certain such compounds are useful for hybridizing to a CLN3 transcript, including but not limited to a CLN3 transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the CLN3 transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Batten Disease.

摘要翻译： 本发明提供了包含与CLN3转录物互补的寡核苷酸的化合物。 某些此类化合物可用于与CLN3转录物杂交，包括但不限于细胞中的CLN3转录物。 在某些实施方案中，这种杂交导致CLN3转录物剪接的调节。 在某些实施方案中，这些化合物用于治疗与巴滕病相关的一种或多种症状。