Patents Assigned to Translate Bio MA, Inc.
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Publication number: 20230062603Abstract: The present disclosure provides a method of pulmonarily administering an oligonucleotide to lung cells. In some embodiments, the disclosure provides formulations that comprise an oligonucleotide that is complementary to a target gene in lung cells and a lipid nanoparticle. Methods of the present disclosure are useful in modifying expression of target genes associated with chronic obstructive pulmonary disease, asthma, and pulmonary fibrosis.Type: ApplicationFiled: January 6, 2021Publication date: March 2, 2023Applicant: Translate Bio MA, Inc.Inventors: Balkrishen Bhat, Brian Bettencourt, Saswata Karmakar, Caroline Woo, John Androsavich, Shrirang Karve
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Publication number: 20220218829Abstract: Provided herein are conjugates comprising targeting moieties such as sugars, folates and cell-penetrating peptides, which can be used for the improved delivery of agents (e.g., nucleic acids, such as oligonucleotides or mRNAs, or other agents) to cells. The invention provides conjugates and compounds comprising targeting moieties, methods for preparing the same, and intermediates useful in their preparation. In another aspect, the present invention provides formulations (e.g., pharmaceutical compositions) comprising the targeting moiety-containing conjugates and compounds. The present invention also provides methods for delivering agents (e.g., nucleic acids such as oligonucleotides or mRNAs) to a cell, methods for treating and/or preventing a disease or condition in a subject, and methods for modulating gene expression in a cell or a subject. Further, provided herein are kits comprising the conjugates, or formulations thereof; and kits for the preparation of conjugates described herein.Type: ApplicationFiled: December 16, 2021Publication date: July 14, 2022Applicant: Translate Bio MA, Inc.Inventors: Balkrishen Bhat, Saswata Karmakar, Debatosh Majumdar, Jia Tay, Nelson Chau
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Patent number: 11253601Abstract: Provided herein are conjugates comprising targeting moieties such as sugars, folates and cell-penetrating peptides, which can be used for the improved delivery of agents (e.g., nucleic acids, such as oligonucleotides or mRNAs, or other agents) to cells. The invention provides conjugates and compounds comprising targeting moieties, methods for preparing the same, and intermediates useful in their preparation. In another aspect, the present invention provides formulations (e.g., pharmaceutical compositions) comprising the targeting moiety-containing conjugates and compounds. The present invention also provides methods for delivering agents (e.g., nucleic acids such as oligonucleotides or mRNAs) to a cell, methods for treating and/or preventing a disease or condition in a subject, and methods for modulating gene expression in a cell or a subject. Further, provided herein are kits comprising the conjugates, or formulations thereof; and kits for the preparation of conjugates described herein.Type: GrantFiled: July 11, 2017Date of Patent: February 22, 2022Assignee: Translate Bio MA, Inc.Inventors: Balkrishen Bhat, Saswata Karmakar, Debatosh Majumdar, Jia Tay, Nelson Chau
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Publication number: 20210102203Abstract: 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: May 18, 2020Publication date: April 8, 2021Applicants: Translate Bio MA, Inc., The General Hospital Corporation d/b/a Massachusetts General HospitalInventors: Arthur M. Krieg, Romesh R. Subramanian, James McSwiggen, Jeannie T. Lee
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Publication number: 20210052706Abstract: Provided herein are compositions and methods for facilitating or enhancing delivery of nucleic acids, such as synthetic mRNAs, into cells or tissues. Such compositions and methods may include use of a targeting moiety-conjugated, such as an N-acetylgalactosamine (GalNAc)-conjugated, oligonucleotide to facilitate or enhance delivery.Type: ApplicationFiled: January 10, 2019Publication date: February 25, 2021Applicant: Translate Bio MA, Inc.Inventors: Balkrishen Bhat, Jia Tay, Saswata Karmakar, Nelson Chau
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Patent number: 10844375Abstract: 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 8, 2017Date of Patent: November 24, 2020Assignee: Translate Bio MA, Inc.Inventors: Eugen Uhlmann, Romesh Subramanian, Arthur M. Krieg
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Patent number: 10837014Abstract: 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: GrantFiled: January 16, 2018Date of Patent: November 17, 2020Assignee: Translate Bio MA, Inc.Inventors: Arthur M. Krieg, Romesh Subramanian, James McSwiggen, Jeannie T. Lee
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Patent number: 10758558Abstract: Provided herein are hybrid oligonucleotides comprising a region that promotes cleavage of a nucleic acid and a region that protects a nucleic acid from exonuclease activity. Such hybrid oligonucleotides are useful for modulating the expression of genes. Related compositions and methods are also provided. In some embodiments, methods are provided for treating a disease, such as by administering a hybrid oligonucleotide.Type: GrantFiled: February 12, 2016Date of Patent: September 1, 2020Assignee: Translate Bio MA, Inc.Inventor: Fatih Ozsolak
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Patent number: 10704046Abstract: 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: August 30, 2018Date of Patent: July 7, 2020Assignee: Translate Bio MA, Inc.Inventors: Eugen Uhlmann, Markus Weber, Romesh R. Subramanian, Thomas Dino Rockel, Arthur M. Krieg
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Patent number: 10655128Abstract: 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: GrantFiled: October 19, 2017Date of Patent: May 19, 2020Assignees: Translate Bio MA, Inc., The General Hospital CorporationInventors: Arthur M. Krieg, Romesh Subramanian, James McSwiggen, Jeannie T. Lee
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Publication number: 20200040333Abstract: 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: January 16, 2018Publication date: February 6, 2020Applicants: Translate Bio MA, Inc., The General Hospital CorporationInventors: Arthur M. Krieg, Romesh Subramanian, James McSwiggen, Jeannie T. Lee
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Publication number: 20190224326Abstract: Provided herein are conjugates comprising targeting moieties such as sugars, folates and cell-penetrating peptides, which can be used for the improved delivery of agents (e.g., nucleic acids, such as oligonucleotides or mRNAs, or other agents) to cells. The invention provides conjugates and compounds comprising targeting moieties, methods for preparing the same, and intermediates useful in their preparation. In another aspect, the present invention provides formulations (e.g., pharmaceutical compositions) comprising the targeting moiety-containing conjugates and compounds. The present invention also provides methods for delivering agents (e.g., nucleic acids such as oligonucleotides or mRNAs) to a cell, methods for treating and/or preventing a disease or condition in a subject, and methods for modulating gene expression in a cell or a subject. Further, provided herein are kits comprising the conjugates, or formulations thereof; and kits for the preparation of conjugates described herein.Type: ApplicationFiled: July 11, 2017Publication date: July 25, 2019Applicant: Translate Bio MA, Inc.Inventors: Balkrishen Bhat, Saswata Karmakar, Debatosh Majumdar, Jia Tay, Nelson Chau
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Publication number: 20190062743Abstract: 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: August 30, 2018Publication date: February 28, 2019Applicant: Translate Bio MA, Inc.Inventors: Eugen Uhlmann, Markus Weber, Romesh R. Subramanian, Thomas Dino Rockel, Arthur M. Krieg
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Publication number: 20190055553Abstract: Aspects of the disclosure provide steric-blocking oligonucleotide-based methods of modulating expression of target genes, e.g., by targeting non-coding RNA scaffolds.Type: ApplicationFiled: October 14, 2016Publication date: February 21, 2019Applicant: TRANSLATE BIO MA, INC.Inventors: James BARSOUM, Caroline WOO
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Patent number: 10174323Abstract: Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of ATP2A2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of ATP2A2. Methods for modulating expression of ATP2A2 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 ATP2A2.Type: GrantFiled: May 16, 2013Date of Patent: January 8, 2019Assignees: The General Hospital Corporation, Translate Bio MA, Inc.Inventors: Arthur M. Krieg, Romesh Subramanian, James McSwiggen, Jeannie T. Lee
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Patent number: 10174328Abstract: 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: GrantFiled: October 3, 2014Date of Patent: January 8, 2019Assignee: Translate Bio MA, Inc.Inventors: Arthur M. Krieg, James Barsoum
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Patent number: 10174315Abstract: Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of hemoglobin genes (HBB, HBD, HBE1, HBG1 or HBG2). Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of hemoglobin genes. Methods for modulating expression of hemoglobin genes 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 hemoglobin genes.Type: GrantFiled: May 16, 2013Date of Patent: January 8, 2019Assignees: The General Hospital Corporation, Translate Bio MA, Inc.Inventors: Arthur M. Krieg, Romesh Subramanian, James McSwiggen, Jeannie T. Lee
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Publication number: 20180312839Abstract: Aspects of the disclosure provide compositions or compounds for activating or enhancing expression of SMN. Further aspects provide compositions and kits, e.g., comprising single stranded oligonucleotides, for activating or enhancing expression of SMN that comprises exon 7. Methods for modulating expression of SMN are also provided.Type: ApplicationFiled: October 26, 2016Publication date: November 1, 2018Applicant: Translate Bio MA, Inc.Inventors: Balkrishen Bhat, David Bullough
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Publication number: 20180311176Abstract: Aspects of the disclosure relate to particle formulations, e.g., nanoparticle formulations, methods of making particle formulations, and methods for delivery of oligonucleotides and/or synthetic RNA, e.g., for increasing gene expression in a targeted manner. In some embodiments, compositions and methods are provided that are useful for posttranscriptionally altering protein and/or RNA levels in a targeted manner. Aspects of the disclosure described herein provide compositions and methods that are useful for protecting RNAs from degradation (e.g., exonuclease mediated degradation).Type: ApplicationFiled: October 26, 2016Publication date: November 1, 2018Applicant: Translate Bio MA, Inc.Inventors: Fatih Ozsolak, Balkrishen Bhat
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Publication number: 20180298384Abstract: 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: January 16, 2018Publication date: October 18, 2018Applicants: Translate Bio MA, Inc., The General Hospital CorporationInventors: Arthur M. Krieg, Romesh Subramanian, James McSwiggen, Jeannie T. Lee