公开(公告)号：US20190309304A1

公开(公告)日：2019-10-10

申请号：US16438015

申请日：2019-06-11

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

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：US20210401869A1

公开(公告)日：2021-12-30

申请号：US17472887

申请日：2021-09-13

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：US10787669B2

公开(公告)日：2020-09-29

申请号：US16438015

申请日：2019-06-11

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

Inventor： Michelle L. Hastings ,  Ole Isacson ,  Joanna A. Korecka-Roet

IPC: C07H21/02 ,  C07H21/04 ,  C12N15/113

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：US20200216846A1

公开(公告)日：2020-07-09

申请号：US16536981

申请日：2019-08-09

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

Inventor： Frank Rigo ,  Michelle L. Hastings

IPC: C12N15/113 ,  A61K31/7088

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：US10370667B2

公开(公告)日：2019-08-06

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: A61K48/00 ,  C07H21/02 ,  C07H21/04 ,  C12N15/113

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：US20180117073A1

公开(公告)日：2018-05-03

申请号：US15835698

申请日：2017-12-08

Applicant: Rosalind Franklin University of Medicine and Science

Inventor： Michelle L. Hastings

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 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：US09840710B2

公开(公告)日：2017-12-12

申请号：US15349840

申请日：2016-11-11

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

Inventor： Michelle L. Hastings ,  Ole Isacson ,  Joanna A. Korecka-Roet

IPC: C07H21/02 ,  C07H21/04 ,  A61K48/00 ,  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：US20170191066A1

公开(公告)日：2017-07-06

申请号：US15315828

申请日：2015-06-04

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

Inventor： Frank Rigo ,  Michelle L. Hastings

IPC: C12N15/113

CPC classification number: C12N15/1138 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/351 ,  C12N2310/3521 ,  C12N2310/3525 ,  C12N2310/3533 ,  C12N2320/33

Abstract: The present invention provides compounds comprising oligonucleotides complementary to an LRP8 transcript. Certain such compounds are useful for hybridizing to an LRP8 transcript, including but not limited, to an LRP8 transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the LRP8 transcript. In certain embodiments, such hybridization results in an increase in inclusion of exon 19 in the LRP8 mRNA transcript. In certain embodiments, such compounds are used to treat Alzheimer's Disease.