Patents Assigned to RaNA Therapeutics, Inc.
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Publication number: 20180223282Abstract: Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of MECP2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of MECP2. Methods for modulating expression of MECP2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of MECP2.Type: ApplicationFiled: October 19, 2017Publication date: August 9, 2018Applicants: RaNA Therapeutics, Inc., The General Hospital Corporation d/b/a Massachusetts General HospitalInventors: Arthur M. Krieg, Romesh Subramanian, James McSwiggen, Jeannie T. Lee
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Patent number: 9717690Abstract: Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.Type: GrantFiled: June 8, 2012Date of Patent: August 1, 2017Assignee: RaNA Therapeutics, Inc.Inventors: Braydon Charles Guild, Michael Heartlein, Frank DeRosa, Jerry Chi Zhang
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Publication number: 20170211065Abstract: The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties.Type: ApplicationFiled: January 26, 2017Publication date: July 27, 2017Applicant: RaNA Therapeutics, Inc.Inventors: Eugen Uhlmann, Markus Weber, Romesh Subramanian, Thomas Dino Rockel, Arthur M. Krieg
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Patent number: 9713626Abstract: Pharmaceutical compositions comprising an mRNA-loaded nanoparticle, wherein the mRNA is an in vitro transcribed mRNA and has a coding sequence at least 80% identical to SEQ ID NO: 3, and wherein the mRNA encodes a human cystic fibrosis transmembrane conductance regulator (CFTR) protein comprising the amino acid sequence of SEQ ID NO:1 are provided. The present invention is particularly useful for treating cystic fibrosis.Type: GrantFiled: October 6, 2015Date of Patent: July 25, 2017Assignees: RaNA Therapeutics, Inc., Ethris GmbHInventors: Michael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith
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Patent number: 9668980Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a mRNA solution and a lipid solution, wherein the mRNA solution and/or the lipid solution are at a pre-determined temperature greater than ambient temperature.Type: GrantFiled: July 2, 2015Date of Patent: June 6, 2017Assignee: RaNA Therapeutics, Inc.Inventors: Frank DeRosa, Shrirang Karve, Michael Heartlein
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Publication number: 20170152511Abstract: Aspects of the invention relate to methods for increasing gene expression in a targeted manner. In some embodiments, methods and compositions are provided that are useful for posttranscriptionally altering protein and/or RNA levels in a targeted manner. Aspects of the invention disclosed herein provide methods and compositions that are useful for protecting RNAs from degradation (e.g., exonuclease mediated degradation).Type: ApplicationFiled: April 30, 2015Publication date: June 1, 2017Applicant: RaNA Therapeutics, Inc.Inventor: FATIH OZSOLAK
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Patent number: 9580708Abstract: The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided.Type: GrantFiled: September 14, 2012Date of Patent: February 28, 2017Assignee: RaNA Therapeutics, Inc.Inventors: Eugen Uhlmann, Markus Weber, Romesh Subramanian, Thomas Dino Rockel, Arthur M. Krieg
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Publication number: 20160222391Abstract: Methods for modulating expression of SMN1 and/or SMN2 in cells obtained from subjects having ALS or in subjects having ALS using single stranded oligonucleotides are provided. Methods for treating ALS using single stranded oligonucleotides are also provided.Type: ApplicationFiled: October 3, 2014Publication date: August 4, 2016Applicant: RaNA Therapeutics, Inc.Inventors: Arthur M. Krieg, James Barsoum
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Publication number: 20160201064Abstract: Provided herein are compositions and methods for increasing Frataxin (FXN) expression. Compositions and methods for treating Friedrich's ataxia are also provided.Type: ApplicationFiled: August 15, 2014Publication date: July 14, 2016Applicant: RaNA Therapeutics, Inc.Inventor: Fatih Ozsolak
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Publication number: 20160201063Abstract: Provided herein are methods for increasing Frataxin (FXN) expression that involve targeting or expressing regulatory factors that modulate the epigenetic state of FXN genes. Also provided herein are methods for increasing FXN expression using inhibitors of a negative epigenetic regulator of FXN. Compositions and methods for treating Friedrich's ataxia are also provided.Type: ApplicationFiled: August 15, 2014Publication date: July 14, 2016Applicant: RaNA Therapeutics, Inc.Inventor: Fatih Ozsolak
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Publication number: 20160122760Abstract: Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of FOXP3. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of FOXP3. Methods for modulating expression of FOXP3 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of FOXP3.Type: ApplicationFiled: June 6, 2014Publication date: May 5, 2016Applicant: RaNA Therapeutics, Inc.Inventors: James Barsoum, Mariano Severgnini, James McSwiggen
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Publication number: 20150315587Abstract: The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties.Type: ApplicationFiled: April 28, 2015Publication date: November 5, 2015Applicant: RaNa Therapeutics, Inc.Inventors: Eugen Uhlmann, Markus Weber, Romesh Subramanian, Thomas Dino Rockel, Arthur M. Krieg
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Publication number: 20150315588Abstract: The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties.Type: ApplicationFiled: April 28, 2015Publication date: November 5, 2015Applicant: RaNA Therapeutics, Inc.Inventors: Eugen Uhlmann, Markus Weber, Romesh Subramanian, Thomas Dino Rockel, Arthur M. Krieg
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Publication number: 20150315585Abstract: The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties.Type: ApplicationFiled: April 28, 2015Publication date: November 5, 2015Applicant: RaNA Therapeutics, Inc.Inventors: Eugen Uhlmann, Romesh Subramanian, Arthur M. Krieg
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Publication number: 20150315586Abstract: The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties.Type: ApplicationFiled: April 28, 2015Publication date: November 5, 2015Applicant: RaNA Therapeutics, Inc.Inventors: Eugen Uhlmann, Romesh Subramanian, Arthur M. Krieg
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Publication number: 20150299695Abstract: The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided.Type: ApplicationFiled: September 14, 2012Publication date: October 22, 2015Applicant: RaNA Therapeutics, Inc.Inventors: Eugen Uhlmann, Markus Weber, Romesh Subramanian, Thomas Dino Rockel, Arthur M. Krieg
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Publication number: 20150252364Abstract: Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2 that comprises exon 7. Methods for modulating expression of SMN1 or SMN2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of SMN1 or SMN2.Type: ApplicationFiled: May 16, 2013Publication date: September 10, 2015Applicants: RaNA Therapeutics, Inc., The General Hospital Corporation d/b/a Massachusetts General HospitalInventors: Arthur M. Krieg, Romesh Subramanian, James McSwiggen, Jeannie T. Lee
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Publication number: 20150247141Abstract: The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties.Type: ApplicationFiled: September 13, 2013Publication date: September 3, 2015Applicant: RaNA Therapeutics, Inc.Inventors: Eugen Uhlmann, Romesh Subramanian, Arthur M. Krieg
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Publication number: 20150247144Abstract: Aspects of the invention relate to methods for increasing gene expression in a targeted manner. In some embodiments, methods and compositions are provided that are useful for posttranscriptionally altering protein and/or RNA levels in a targeted manner. Aspects of the invention disclosed herein provide methods and compositions that are useful for protecting RNAs from degradation (e.g., exonuclease mediated degradation).Type: ApplicationFiled: April 30, 2015Publication date: September 3, 2015Applicant: RaNA Therapeutics, Inc.Inventor: FATIH OZSOLAK
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Publication number: 20150247145Abstract: Aspects of the invention relate to methods for increasing gene expression in a targeted manner. In some embodiments, methods and compositions are provided that are useful for posttranscriptionally altering protein and/or RNA levels in a targeted manner. Aspects of the invention disclosed herein provide methods and compositions that are useful for protecting RNAs from degradation (e.g., exonuclease mediated degradation).Type: ApplicationFiled: April 30, 2015Publication date: September 3, 2015Applicant: RaNA Therapeutics, Inc.Inventor: FATIH OZSOLAK