136 patents
Page 7 of 7
Utility
Compounds and methods for modulation of DUX4
20 Jan 20
The present disclosure pertains generally to double-stranded small interfering RNAs that modulate gene expression for use in research, diagnostics, and/or therapeutics.
Frank Rigo, Stephen J. Tapscott
Filed: 14 Jan 16
Utility
Compositions and methods for modulating Ataxin 3 expression
13 Jan 20
Disclosed are methods for modulating splicing of Ataxin 3 mRNA in an animal with modified oligonucleotides.
Frank Rigo, Thazha P. Prakash, Wilhelmina Maria Clasina Van Roon-Mom, Lodewijk Julius Anton Toonen
Filed: 22 Sep 16
Utility
Methods for modulating Ataxin 2 expression
13 Jan 20
Disclosed herein are methods for decreasing Ataxin 2 mRNA and protein expression.
C. Frank Bennett, Susan M. Freier, Stefan M. Pulst, Daniel R. Scoles, Gene Hung
Filed: 6 May 18
Utility
Modulation of nuclear-retained RNA
6 Jan 20
Provided herein are methods, compounds, and compositions for reducing expression of a nrRNA in an animal.
C. Frank Bennett, Huynh-Hoa Bui, Kenneth W. Dobie
Filed: 5 Jul 17
Utility
Modulators of SMAD7 expression
30 Dec 19
The present embodiments provide methods, compounds, and compositions useful for inhibiting SMAD7 expression, which may be useful for treating, preventing, or ameliorating a disease associated with SMAD7.
Susan M. Freier, Huynh-Hoa Bui
Filed: 6 Sep 18
Utility
Modulators of PCSK9 expression
30 Dec 19
The present embodiments provide methods, compounds, and compositions useful for inhibiting PCSK9 expression, which may be useful for treating, preventing, or ameliorating a disease associated with PCSK9.
Eric E. Swayze, Susan M. Freier
Filed: 22 Mar 18
Utility
Antisense compounds
2 Dec 19
Provided herein are gapmer oligomeric compounds for reduction of target RNA in vivo comprising different nucleotide modifications within one or both wing regions.
Eric E. Swayze
Filed: 8 Dec 16
Utility
Methods and compositions for modulating apolipoprotein (a) expression
18 Nov 19
Disclosed herein are antisense compounds and methods for decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) or Lp(a).
Rosanne M. Crooke, Mark J. Graham, Susan M. Freier, Marc Lim, Andrew Dibble
Filed: 26 Dec 17
Utility
Modulation of signal transducer and activator of transcription 3 (STAT3) expression
18 Nov 19
Disclosed herein are antisense compounds and methods for decreasing STAT3 mRNA and protein expression.
Eric E. Swayze, Susan M. Freier, Robert A. MacLeod, Youngsoo Kim
Filed: 8 May 16
Utility
2′-methoxy substituted oligomeric compounds and compositions for use in gene modulations
18 Nov 19
Compositions comprising first and second oligomers are provided wherein at least a portion of the first oligomer is capable of hybridizing with at least a portion of the second oligomer, at least a portion of the first oligomer is complementary to and capable of hybridizing to a selected target nucleic acid, and at least one of the first or second oligomers includes a modified sugar and/or backbone modification.
Stanley T. Crooke
Filed: 11 Oct 17
Utility
Methods and compositions for modlating apolipoprotein (A) expression
11 Nov 19
Disclosed herein are antisense compounds and methods for decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) or Lp(a).
Rosanne M. Crooke, Mark J. Graham, Susan M. Freier
Filed: 19 Dec 17
Utility
Antisense compounds targeting apolipoprotein E receptor 2
11 Nov 19
The present invention provides compounds comprising oligonucleotides complementary to an LRP8 transcript.
Frank Rigo, Michelle L. Hastings
Filed: 3 Jun 15
Utility
Method for solution phase detritylation of oligomeric compounds
21 Oct 19
Provided herein are methods for removal of monomethoxytrityl groups from oligomeric compounds comprising terminally linked monomethoxytrityl protected amino groups.
Dana Chreng, Michael T. Migawa
Filed: 22 Feb 16
Utility
Compositions for modulating C9ORF72 expression
14 Oct 19
Disclosed herein are compositions and methods for reducing expression of C90RF72 mRNA and protein in an animal with C90RF72 specific inhibitors.
Susan M. Freier
Filed: 14 Oct 13
Utility
Methods and compounds useful in conditions related to repeat expansion
7 Oct 19
Described are compounds and methods useful for the treatment and investigation of diseases and disorders associated with expanded repeat-containing RNA molecules.
Thazha P. Prakash, Jiaxin Hu, Jing Liu, Dongbo Yu, David Corey, Eric E. Swayze
Filed: 30 Jul 14
Utility
Allele specific modulators of P23H rhodopsin
30 Sep 19
The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP) by administering a P23H rhodopsin specific inhibitor to a subject.
Susan F. Murray, Punit P. Seth, Michael L. McCaleb, Susan M. Freier, Priyam Singh
Filed: 25 Feb 16