Patents Assigned to Sirna Therapeutics, Inc.
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Patent number: 11952573Abstract: The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of PHD2 gene expression and/or activity, and/or modulate a beta-catenin gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against PHD2 gene expression.Type: GrantFiled: April 1, 2022Date of Patent: April 9, 2024Assignee: Sirna Therapeutics, Inc.Inventors: Brandon Ason, Duncan Brown, Walter R. Strapps
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RNA interference mediated inhibition of gene expression using short interfering nucleic acids (siNA)
Patent number: 11932854Abstract: The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.Type: GrantFiled: October 25, 2021Date of Patent: March 19, 2024Assignee: Sirna Therapeutics, Inc.Inventors: Mark Cancilla, James John Cunningham, William Michael Flanagan, Henry J. Haringsma, Denise M. Kenski, Matthew G. Stanton, Steven M. Stirdivant, Aarron T. Willingham -
Patent number: 11911475Abstract: The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.Type: GrantFiled: June 24, 2022Date of Patent: February 27, 2024Assignee: SIRNA THERAPEUTICS, INC.Inventors: Matthew G. Stanton, Brian W. Budzik, Gregory L. Beutner, Hongbiao Liao
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Publication number: 20230000989Abstract: The instant invention provides for novel catiomc lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.Type: ApplicationFiled: June 24, 2022Publication date: January 5, 2023Applicant: SIRNA THERAPEUTICS, INC.Inventors: Matthew G. Stanton, Brian W. Budzik, Gregory L. Beutner, Hongbiao Liao
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Patent number: 11530407Abstract: The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.Type: GrantFiled: February 18, 2020Date of Patent: December 20, 2022Assignee: Sirna Therapeutics, Inc.Inventors: Brian Allen Carr, Vasant R. Jadhav, Denise M. Kenski, David M. Tellers, Aarron T. Willingham
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RNA interference mediated inhibition of gene expression using short interfering nucleic acids (siNA)
Patent number: 11193126Abstract: The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.Type: GrantFiled: April 6, 2018Date of Patent: December 7, 2021Assignee: SIRNA THERAPEUTICS, INC.Inventors: Mark Cancilla, James John Cunningham, William Michael Flanagan, Henry J. Haringsma, Denise M. Kenski, Matthew G. Stanton, Steven M. Stirdivant, Aarron T. Willingham -
Patent number: 11117917Abstract: Disclosed herein is a modular composition comprising 1) an oligonucleotide; 2) one or more tetraGalNAc ligands of Formula (I), which may be the same or different; optionally, 3) one or more linkers, which may be the same or different; 4) one or more peptides independently selected from Table 3, which may be the same or different; and optionally, 5) one or more targeting ligands, solubilizing agents, pharmacokinetics enhancing agents, lipids, and/or masking agents.Type: GrantFiled: February 5, 2019Date of Patent: September 14, 2021Assignee: Sirna Therapeutics, Inc.Inventors: David Tellers, Steven L. Colletti, Vadim Dudkin, Jeffrey Aaronson, Aaron Momose, Thomas Joseph Tucker, Yu Yuan, Kathleen B. Calati, Lu Tian, Rubina G. Parmar, Anthony W. Shaw, Weimin Wang, Rachel Anne Storr, Marina Busuek, Robert A. Kowtoniuk
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Patent number: 10889815Abstract: The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.Type: GrantFiled: November 7, 2018Date of Patent: January 12, 2021Assignee: SIRNA THERAPEUTICS, INC.Inventors: Leonid Beigelman, James McSwiggen, Chandra Vargeese
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Patent number: 10793860Abstract: The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of HBV gene expression and/or activity, and/or modulate a HBV gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against HBV gene expression.Type: GrantFiled: August 5, 2019Date of Patent: October 6, 2020Assignee: Sirna Therapeutics, Inc.Inventors: Steven Bartz, Duncan Brown, Michael Robinson
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Patent number: 10738308Abstract: The present invention relates to RNAi molecules, and compositions thereof, comprising a 2? internucleoside linkage connecting the nucleotide at position 1 and the nucleotide at position 2 at the 5? end of the antisense strand. Specifically, the invention relates to single- and double-stranded short interfering nucleic acid (siNA) molecules that are capable of mediating RNA interference comprising 5? modified nucleotides that comprise, among other potential modifications, a 2? internucleoside linkage. The invention further relates to 5? modified nucleotides used as reagents to generate the RNAi molecules of the invention and methods of using the disclosed RNAi molecules.Type: GrantFiled: January 5, 2018Date of Patent: August 11, 2020Assignee: SIRNA THERAPEUTICS, INC.Inventors: Wonsuk Chang, Erin N. Guidry, Matthew G. Stanton, Daniel Zewge
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Patent number: 10729787Abstract: The present invention provides methods comprising the in vivo delivery of small nucleic acid molecules capable of mediating RNA interference and reducing the expression of myostatin, wherein the small nucleic acid molecules are introduced to a subject by systemic administration. Specifically, the invention relates to methods comprising the in vivo delivery of short interfering nucleic acid (siNA) molecules that target a myostatin gene expressed by a subject, wherein the siNA molecule is conjugated to a lipophilic moiety, such as cholesterol. The myostatin siNA conjugates that are delivered as per the methods disclosed are useful to modulate the in vivo expression of myostatin, increase muscle mass and/or enhance muscle performance. Use of the disclosed methods is further indicated for treating musculoskeletal diseases or disorders and/or diseases or disorders that result in conditions in which muscle is adversely affected.Type: GrantFiled: May 31, 2018Date of Patent: August 4, 2020Assignee: SIRNA THERAPEUTICS, INC.Inventors: Marija Tadin-strapps, Tayeba Khan, Walter Richard Strapps, Laura Sepp-Lorenzino, Vasant R. Jadhav, Duncan Brown
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Publication number: 20200222540Abstract: The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.Type: ApplicationFiled: January 13, 2020Publication date: July 16, 2020Applicant: SIRNA THERAPEUTICS, INC.Inventors: Matthew G. Stanton, Brian W. Budzik, Gregory L. Beutner, Hongbiao Liao
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Patent number: 10689648Abstract: The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.Type: GrantFiled: August 23, 2017Date of Patent: June 23, 2020Assignee: SIRNA THERAPEUTICS, INC.Inventors: Brian Allen Carr, Vasant R. Jadhav, Denise M. Kenski, David M. Tellers, Aarron T. Willingham
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Patent number: 10662428Abstract: The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.Type: GrantFiled: April 11, 2019Date of Patent: May 26, 2020Assignee: SIRNA THERAPEUTICS, INC.Inventors: Leonid Beigelman, James McSwiggen, Chandra Vargeese
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Patent number: 10584335Abstract: The instant disclosure features single-stranded RNA molecules comprising one or more internal, non-nucleotide spacers. A non-nucleotide spacer covalently links nucleotide portions of the molecule. The single-stranded RNA molecules function as guide or antisense strands that are capable of inhibiting gene expression via an RNA interference mechanism and, thus, represent single-stranded RNAi agents. The single-stranded RNAi molecules can be used in methods for a variety of therapeutic, diagnostic, target validation, genomic discovery, genetic engineering, and pharmacogenomic applications.Type: GrantFiled: November 10, 2017Date of Patent: March 10, 2020Assignee: SIRNA THERAPEUTICS, INC.Inventors: Lee Lim, Guillaume Chorn, Aarron T. Willingham, Lihong Zhao
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Patent number: 10577606Abstract: The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of PHD2 gene expression and/or activity, and/or modulate a beta-catenin gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against PHD2 gene expression.Type: GrantFiled: August 2, 2017Date of Patent: March 3, 2020Assignee: Sirna Therapeutics, Inc.Inventors: Brandon Ason, Duncan Brown, Walter R. Strapps
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Patent number: 10550385Abstract: This invention relates to a process for introducing two or more 2?-modifications into an RNA, wherein the RNA has a 2?-O substituent containing an alkyl ester functional group on one or more ribose rings of a strand and a 2?-O substituent containing an alkyne functional groups on one or more ribose rings on the same strand. The process comprises a) adding an amine compound to the RNA to form amidation reactions with the alkyl ester functional groups; b) dissolving the modified RNA from step (a) in a solvent to form a solution; and c) adding an organic azide and a copper or ruthenium catalyst to the solution obtained in step (b) to form 2?-azide-alkyne cycloaddition reaction products with the alkyne functional groups.Type: GrantFiled: December 18, 2013Date of Patent: February 4, 2020Assignee: SIRNA THERAPEUTICS, INC.Inventors: Daniel Zewge, Gregory T. Copeland, Zhen Li, Joseph D. Armstrong
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Patent number: 10526602Abstract: This invention relates generally to segmented oligonucleotides capable of modulating gene expression. Specifically, the instant invention relates to segmented microRNA (miRNA) oligonucleotides, including segmented miRNA precursors and segmented pre-microRNAs. The invention also relates to compositions comprising such segmented oligonucleotides, as well as to methods of making and using such oligonucleotides for diagnosis and treatment of diseases associated or causally linked to aberrant levels or activities of gene expression, including aberrant levels of coding and/or non-coding RNA.Type: GrantFiled: July 12, 2017Date of Patent: January 7, 2020Assignee: SIRNA THERAPEUTICS, INC.Inventors: Guillaume Chorn, Lee Lim, Lihong Zhao
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Patent number: 10508277Abstract: The present invention relates to a multifunctional short interfering nucleic acid (siNA) having a structure of Formula MF-III: wherein each X, X?, Y, and Y? is independently an oligonucleotide of length about 15 nucleotides to about 50 nucleotides; X comprises a nucleotide sequence that is complementary to a nucleotide sequence present in region Y?; X? comprises a nucleotide sequence that is complementary to a nucleotide sequence present in region Y; one or more of X, X?, Y, and Y? is independently complementary to a first, second, third, or fourth target sequence, respectively, or a portion thereof; and W represents a nucleotide or non-nucleotide linker that connects sequences Y? and Y, wherein the siNA directs cleavage of the first, second, third, and/or fourth target sequence via RNA interference.Type: GrantFiled: May 11, 2018Date of Patent: December 17, 2019Assignee: SIRNA THERAPEUTICS, INC.Inventor: Vasant R. Jadhav
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Patent number: 10407682Abstract: The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of HBV gene expression and/or activity, and/or modulate a HBV gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against HBV gene expression.Type: GrantFiled: December 18, 2017Date of Patent: September 10, 2019Assignee: Sirna Therapeutics, Inc.Inventors: Steven Bartz, Duncan Brown, Michael Robinson