公开(公告)号：US20170268000A1

公开(公告)日：2017-09-21

申请号：US15039188

申请日：2014-12-02

Applicant: IONIS PHARMACEUTICALS ,INC. ,  ROSALIND FRANKLIN UNIVERSITY OF MEDICINE AND SCIENCE

Inventor： Frank RIGO ,  Michelle L. HASTINGS

IPC: C12N15/113

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

Abstract: 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.

公开(公告)号：US20250049838A1

公开(公告)日：2025-02-13

申请号：US18933256

申请日：2024-10-31

Applicant: Rosalind Franklin University of Medicine and Science

Inventor： Michelle L. HASTINGS ,  Wren E. MICHAELS

IPC: A61K31/712 ,  C12N15/113

Abstract: 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 and/or expression 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：US20180119152A1

公开(公告)日：2018-05-03

申请号：US15835995

申请日：2017-12-08

Applicant: Rosalind Franklin University of Medicine and Science

Inventor： Michelle L. HASTINGS

IPC: C12N15/113

Abstract: 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：US20180094266A1

公开(公告)日：2018-04-05

申请号：US15837926

申请日：2017-12-11

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

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

IPC: C12N15/113

CPC classification number: 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

Abstract: 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：US20160244767A1

公开(公告)日：2016-08-25

申请号：US15045999

申请日：2016-02-17

Applicant: Rosalind Franklin University of Medicine and Science

Inventor： Michelle L. HASTINGS

IPC: C12N15/113

CPC classification number: 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

Abstract: 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.

Abstract translation: 本公开一般涉及包含与囊性纤维化跨膜传导调节子（CFTR）RNA转录物互补的寡核苷酸的化合物。 某些这样的化合物可用于与CFTR RNA转录物杂交，包括但不限于细胞中的CFTR RNA转录物。 在某些实施方案中，这种杂交导致调节CFTR转录物的剪接。 在某些实施方案中，这些化合物用于治疗与囊性纤维化相关的一种或多种症状。

公开(公告)号：US20150315590A1

公开(公告)日：2015-11-05

申请号：US14705579

申请日：2015-05-06

Applicant: ROSALIND FRANKLIN UNIVERSITY OF MEDICINE AND SCIENCE

Inventor： Michelle L. HASTINGS

IPC: C12N15/113

CPC classification number: C12N15/113 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  C12N2310/11 ,  C12N2320/33

Abstract: The present invention provides a method for treating Usher's syndrome in a human subject including administering to the human subject an oligonucleotide having 8 to 30 linked nucleosides having a nucleobase sequence comprising a complementary region comprising at least 8 contiguous nucleobases complementary to a target region of equal length within exon 3 of an Usher RNA transcript.

Abstract translation: 本发明提供了一种治疗人类受试者的Usher综合征的方法，包括向人类受试者施用具有8至30个连接核苷的寡核苷酸，所述核苷酸具有包含至少8个连续核苷酸序列的互补区的互补区，该互补区与等长的靶区域互补 在Usher RNA转录本的外显子3内。