公开(公告)号：EP2534262A2

公开(公告)日：2012-12-19

申请号：EP11740543.1

申请日：2011-02-08

申请人： Isis Pharmaceuticals, Inc. ,  The University Of British Columbia

发明人： BENNETT, C., Frank ,  HAYDEN, Michael ,  FREIER, Susan, M. ,  GREENLEE, Sarah ,  CARROLL, Jeffrey ,  WARBY, Simon ,  SWAYZE, Eric, E.

IPC分类号： C12Q1/68 ,  C12N15/11 ,  C07H21/04

CPC分类号： C12Q1/6883 ,  C12N15/113 ,  C12N2310/11 ,  C12N2320/34 ,  C12Q1/6811 ,  C12Q1/6897 ,  C12Q2600/136 ,  C12Q2600/156 ,  C12Q2600/158 ,  C12Q2525/207 ,  C12Q2535/131 ,  C12Q2539/107 ,  C12Q2521/327 ,  C12Q2525/121 ,  C12Q2525/185

摘要： Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a huntingtin gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate Huntington's Disease (HD).

公开(公告)号：EP2534262B1

公开(公告)日：2016-12-14

申请号：EP11740543.1

申请日：2011-02-08

申请人： Ionis Pharmaceuticals, Inc. ,  The University Of British Columbia

发明人： BENNETT, C., Frank ,  HAYDEN, Michael ,  FREIER, Susan, M. ,  GREENLEE, Sarah ,  CARROLL, Jeffrey ,  WARBY, Simon ,  SWAYZE, Eric, E.

IPC分类号： C12Q1/68 ,  C12N15/11 ,  C07H21/04

CPC分类号： C12Q1/6883 ,  C12N15/113 ,  C12N2310/11 ,  C12N2320/34 ,  C12Q1/6811 ,  C12Q1/6897 ,  C12Q2600/136 ,  C12Q2600/156 ,  C12Q2600/158 ,  C12Q2525/207 ,  C12Q2535/131 ,  C12Q2539/107 ,  C12Q2521/327 ,  C12Q2525/121 ,  C12Q2525/185

摘要： Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a huntingtin gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate Huntington's Disease (HD).

公开(公告)号：EP3208347B1

公开(公告)日：2019-08-14

申请号：EP16203872.3

申请日：2011-02-08

申请人： Ionis Pharmaceuticals, Inc. ,  The University Of British Columbia

发明人： BENNETT, C., Frank ,  HAYDEN, Michael ,  FREIER, Susan, M. ,  GREENLEE, Sarah ,  CARROLL, Jeffrey ,  WARBY, Simon ,  SWAYZE, Eric, E.

IPC分类号： C12Q1/68 ,  C12N15/11 ,  C07H21/04 ,  C12Q1/6811

公开(公告)号：EP3628750A1

公开(公告)日：2020-04-01

申请号：EP19191293.0

申请日：2011-02-08

申请人： Ionis Pharmaceuticals, Inc. ,  The University Of British Columbia

发明人： BENNETT, Frank, C. ,  HAYDEN, Michael ,  FREIER, Susan, M. ,  GREENLEE, Sarah ,  SWAYZE, Eric, E. ,  CARROLL, Jeffrey ,  WARBY, Simon

IPC分类号： C12Q1/6897

摘要： Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a huntingtin gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate Huntington's Disease (HD).

公开(公告)号：EP2173358B1

公开(公告)日：2015-10-21

申请号：EP08771759.1

申请日：2008-06-23

申请人： Isis Pharmaceuticals, Inc.

发明人： BHAT, Balkrishen ,  PRAKASH, Thazha, P. ,  ALLERSON, Charles ,  KINBERGER, Garth, A. ,  MARCUSSON, Eric, G. ,  SWAYZE, Eric, E.

IPC分类号： C12N15/113 ,  C12N15/11

CPC分类号： C12N15/111 ,  C12N15/113 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3515 ,  C12N2310/3525 ,  C12N2310/3533 ,  C12N2320/51 ,  C12Y301/03048 ,  C12N2310/3521

公开(公告)号：EP2906258A2

公开(公告)日：2015-08-19

申请号：EP13847957.1

申请日：2013-10-15

申请人： Isis Pharmaceuticals, Inc.

发明人： BENNETT, Frank, C. ,  FREIER, Susan, M. ,  SWAYZE, Eric, E. ,  RIGO, Frank

IPC分类号： A61K48/00

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/313 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3233 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3525 ,  C12N2310/3533

摘要： Disclosed herein are compositions and methods for reducing expression of C90RF72 mRNA and protein in an animal with C90RF72 specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C90RF72 specific inhibitors include antisense compounds. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration C90RF72 specific inhibitors include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), corticalbasal degeneration syndrome (CBD), atypical Parkinsonian syndrome, and olivopontocerellar degeneration (OPCD).

公开(公告)号：EP2906255A2

公开(公告)日：2015-08-19

申请号：EP13844964.0

申请日：2013-10-11

申请人： Isis Pharmaceuticals, Inc.

发明人： KORDASIEWICZ, Holly ,  MAZUR, Curt ,  SETH, Punit, P. ,  SWAYZE, Eric, E.

IPC分类号： A61K48/00 ,  C07H21/02

CPC分类号： C12N15/113 ,  C07H21/00 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3525 ,  C12N2310/3533

摘要： The present disclosure provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.

公开(公告)号：EP2023939B1

公开(公告)日：2012-06-27

申请号：EP07811874.2

申请日：2007-05-07

申请人： Isis Pharmaceuticals, Inc.

发明人： BHANOT, Sanjay ,  GEARY, Richard, S. ,  MCKAY, Robert ,  MONIA, Brett, P. ,  SETH, Punit, P. ,  SIWKOWSKI, Andrew, M. ,  SWAYZE, Eric, E. ,  WANCEWICZ, Edward

IPC分类号： C12N15/113 ,  A61K31/712 ,  A61P3/00 ,  A61P9/00

CPC分类号： C12N15/113 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/341 ,  C12N2310/3515 ,  C12Y203/0102 ,  C12N2310/3521

摘要： The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.

公开(公告)号：EP2176280B1

公开(公告)日：2011-12-21

申请号：EP08781239.2

申请日：2008-07-01

申请人： Isis Pharmaceuticals, Inc.

发明人： SWAYZE, Eric, E. ,  SETH, Punit, P.

IPC分类号： C07H21/00 ,  C07H19/04

CPC分类号： C07H21/00 ,  C07H19/00

摘要： The present disclosure describes 6-disubstituted bicyclic nucleosides, oligomeric compounds prepared therefrom and methods of using the oligomeric compounds. More particularly, the 6-disubstituted bicyclic nucleosides each comprise a 2'-O-C(Ri)(R2)-4' or 2'-O- C=(R3)(R.4)-4' bridge wherein each R is, independently a substituent group and Ri and R2 include H. The 6-disubstituted bicyclic nucleosides are useful for enhancing properties of oligomeric compounds including nuclease resistance. In certain embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.

公开(公告)号：EP2176280A2

公开(公告)日：2010-04-21

申请号：EP08781239.2

申请日：2008-07-01

申请人： Isis Pharmaceuticals, Inc.

发明人： SWAYZE, Eric, E. ,  SETH, Punit, P.

IPC分类号： C07H21/00 ,  C07H19/04

CPC分类号： C07H21/00 ,  C07H19/00

摘要： The present disclosure describes 6-disubstituted bicyclic nucleosides, oligomeric compounds prepared therefrom and methods of using the oligomeric compounds. More particularly, the 6-disubstituted bicyclic nucleosides each comprise a 2'-O-C(Ri)(R2)-4' or 2'-O- C=(R3)(R.4)-4' bridge wherein each R is, independently a substituent group and Ri and R2 include H. The 6-disubstituted bicyclic nucleosides are useful for enhancing properties of oligomeric compounds including nuclease resistance. In certain embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.