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：US20180233059A1

公开(公告)日：2018-08-16

申请号：US15433703

申请日：2017-02-15

Applicant: Rosalind Franklin University of Medicine and Science

Inventor： Tamzin J. Batteson ,  Sarah S. Garber

IPC: G09B9/00 ,  G09B23/30 ,  G09B7/02 ,  G09B7/10 ,  G09B19/00

CPC classification number: G09B9/00 ,  G09B7/02 ,  G09B7/10 ,  G09B19/00 ,  G09B23/30

Abstract: In one aspect, a method for improving patient outcome is disclosed. The method includes (i) observing a first interview of a simulated patient, wherein, during the first interview, the simulated patient is in a first form; (ii) participating in a second interview of the simulated patient, wherein, during the second interview, the simulated patient is in a second form, and wherein participating in the second interview comprises interacting with the simulated patient; (iii) participating in a third interview of the simulated patient, wherein, during the third interview, the simulated patient is in the first form, and wherein participating in the third interview comprises interacting with the simulated patient; and (iv) based at least in part on the first, second, and third interviews, creating a plan of care for the simulated patient.

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：US20180022722A1

公开(公告)日：2018-01-25

申请号：US15671833

申请日：2017-08-08

Applicant: Rosalind Franklin University of Medicine and Science

Inventor： Grace E. Stutzmann ,  Russell Dahl ,  Christopher H. Kaiho

IPC: C07D307/54 ,  C07D307/52 ,  C07D407/12 ,  C07D405/12

CPC classification number: C07D307/54 ,  C07D307/52 ,  C07D405/12 ,  C07D407/12

Abstract: Disclosed herein are methods and compositions comprising compounds capable of normalizing neuronal calcium dyshomeostasis. Also disclosed are methods comprising these compounds for treating neuronal or neurological disorders, including Alzheimer's disease, Parkinson's disease, Huntington's disease, fronto-temporal dementia, Pick's disease, chronic traumatic encepholopathy, traumatic brain injury, stroke, cerebellar ataxia, multiple sclerosis, Down syndrome, and aging-related CNS disorders.

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：US20170295793A1

公开(公告)日：2017-10-19

申请号：US15483987

申请日：2017-04-10

Applicant: ROSALIND FRANKLIN UNIVERSITY OF MEDICINE AND SCIENCE

Inventor： Kwang Poo Chang ,  Bala Krishna Kolli ,  Shin-Hong Shiao

IPC: A01N55/02 ,  A01N55/00 ,  A01N43/90

CPC classification number: A01N55/02 ,  A01N43/90 ,  A01N55/00

Abstract: Disclosed herein are photodynamic insecticide methods and compositions for the control or reduction of insect populations comprising the use of photosensitizer compounds in combination with light.

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.