Abstract: The present disclosure provides oligomeric compound comprising a modified oligonucleotide having a central region comprising one or more modifications. In certain embodiments, the present disclosure provides oligomeric compounds having an improved therapeutic index or an increased maximum tolerated dose.
Type:
Grant
Filed:
February 12, 2019
Date of Patent:
May 17, 2022
Assignee:
lonis Pharmaceuticals, Inc.
Inventors:
Punit P. Seth, Michael Oestergaard, Michael T. Migawa, Xue-hai Liang, Wen Shen, Stanley T. Crooke, Eric E. Swayze
Abstract: Oligonucleotides, chemically-modified oligonucleotides, and oligonucleotide-conjugate complexes for use in research, diagnostics, and/or therapeutics are described herein. In some embodiments, oligonucleotides comprising a stabilized phosphate moiety covalently attached to the 5?-terminal nucleoside are provided.
Type:
Grant
Filed:
June 18, 2018
Date of Patent:
August 31, 2021
Assignee:
lonis Pharmaceuticals, Inc.
Inventors:
Thazha P. Prakash, Eric E. Swayze, Walter F. Lima, Garth A. Kinberger
Abstract: Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.
Abstract: Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.
Abstract: Certain embodiments are directed to compounds and compositions targeted to human androgen receptor (AR) for inhibiting androgen receptor levels in a cell, which can be useful for methods of treating cancer and inhibiting cancer cell growth or proliferation.
Type:
Grant
Filed:
February 13, 2019
Date of Patent:
March 9, 2021
Assignee:
lonis Pharmaceuticals, Inc.
Inventors:
Robert A. Macleod, Youngsoo Kim, Tianyuan Zhou, Susan M. Freier, Punit Seth, Eric Swayze, Brett Monia